Annuncio • Jan 26
Amicus Therapeutics, Inc. Provides Earnings Guidance for the Year Ended December 31, 2025 Amicus Therapeutics, Inc. provided earnings guidance for the year ended December 31, 2025. for the year, the company expects its total net product revenues amounted to approximately $634 million. Board Change • Dec 30
Insufficient new directors No new directors have joined the board in the last 3 years. The company's board is composed of: No new directors. 5 experienced directors. 4 highly experienced directors. Independent Director Eiry Roberts was the last director to join the board, commencing their role in 2021. The company’s insufficient board refreshment is considered a risk according to the Simply Wall St Risk Model. Annuncio • Dec 19
BioMarin Pharmaceutical Inc. (NasdaqGS:BMRN) agreed to acquire Amicus Therapeutics, Inc. (NasdaqGM:FOLD) for $4.7 billion. BioMarin Pharmaceutical Inc. (NasdaqGS:BMRN) agreed to acquire Amicus Therapeutics, Inc. (NasdaqGM:FOLD) for $4.7 billion on December 19, 2025. A cash consideration of $4.7 billion valued at $14.5 per share will be paid by BioMarin Pharmaceutical Inc. As part of consideration, $4.7 billion is paid towards common equity of Amicus Therapeutics, Inc. The transaction will be financed through cash on hand and non-convertible debt financing of $3.7 billion.
The transaction is subject to approval by regulatory board / committee, approval of merger agreement by target board, approval of offer by acquirer board, approval of offer by target shareholders and subject to antitrust regulations. The deal has been unanimously approved by the board. The transaction is expected to close in the second quarter of 2026.
Morgan Stanley & Co. LLC acted as financial advisor for BioMarin Pharmaceutical Inc. J.P. Morgan Securities LLC acted as financial advisor for BioMarin Pharmaceutical Inc. Jones Day acted as legal advisor for BioMarin Pharmaceutical Inc. Cooley LLP acted as legal advisor for BioMarin Pharmaceutical Inc. Centerview Partners LLC acted as financial advisor for Amicus Therapeutics, Inc. Goldman Sachs & Co. LLC acted as financial advisor for Amicus Therapeutics, Inc. Kirkland & Ellis LLP acted as legal advisor for Amicus Therapeutics, Inc. Morgan Stanley Senior Funding, Inc. is acting as sole lead arranger and has provided a bridge commitment for this amount. Annuncio • Nov 04
Amicus Therapeutics, Inc. Reiterates Earnings Guidance for the Year 2025 Amicus Therapeutics, Inc. reiterated earnings guidance for the year 2025. For the year, the company expects Total Revenue Growth in the range of 15% to 22%, GAAP Net Income of Positive during second half 2025. Annuncio • Oct 23
Amicus Therapeutics, Inc. to Report Q3, 2025 Results on Nov 04, 2025 Amicus Therapeutics, Inc. announced that they will report Q3, 2025 results at 9:30 AM, US Eastern Standard Time on Nov 04, 2025 Annuncio • Sep 08
Amicus Therapeutics Announces the Presentation of New 4-Year Data for Pombiliti (Cipaglucosidase alfa-atga) + Opfolda®? (miglustat) Amicus Therapeutics announced the presentation of new 4-year muscle function, muscle strength and biomarker endpoints from the PROPEL open-label extension (OLE) study of cipaglucosidase alfa-atga + miglustat (cipa+mig) in adults with late-onset Pompe disease (LOPD) at the International Congress of Inborn Errors of Metabolism (ICIEM) in Kyoto, Japan. The new analysis, "208-week efficacy and safety of cipaglucOSidase alfa plus miglustat in patients with late-onset Pompe Disease treated from PROPEL baseline: muscle function and biomarkers," was conducted on the 82 patients (62 ERT-experienced and 20 ERT-naive) who were randomized to cipa+mig in PROPEL and who enrolled in the open label extension (OLE). Pombiliti + Opfolda is indicated in the U.S. for the treatment of adult patients with LOPD weighing 40 kg and who are not improving on their current ERT. The study enrolled 123 adult LOPD patients who still had the ability to walk and to breathe without mechanical ventilation. Patients enrolled in PROPEL were randomized 2:1 so that for every two patients randomized to be treated with cipaglucosIDase alfa-atga+ miglustat, one was randomized to be treated with the comparator. Of the patients enrolled in PROPEL, 77% were being treated with alglucosidase alfa (n=95) for at least 2 years at study entry and 23% had never been treated with any ERT (n=28). 117 of the 123 patients (>95%) completed the PROPEL study. Efficacy endpoints of the study included primary endpoint of change from baseline to week 52 in 6MWD for comparison of superiority and key secondary endpoint of change from baseline toweek 52 in 6MWD in the overall population. After 52 weeks, ERT-experienced patients treated with cipaglUCosidase alfa- atga + miglustat (n=61) walk an estimated 17 meters (95% CI, 0.2, 33) farther than the comparator group (n=29). After 52 weeks, ERT -experienced patients treated with cIPaglucosidase Alfa-atga + mig Purpose (n=55) showed an estimated treatment difference of 3.5% (95% CI, 1.0, 6.0) in FVC compared with the comparator group (n<29). The Phase 3 open-label extension (Ole) study (ATB200-07) is a multicenter, international study designed to evaluate the long-term safety and efficacy of cipaglucasidase alfa-atgas + miglustat in adult subjects with LOPD who completed the Phase 3 PROPEL Study (ATB200-03). Of the 118 patients treated in the OLE, 81 continued cipa+mig treatment from PROPEL (cipa+mIG group; 20 ERT-naive); 20 ERT-naive), and 37 switched from alg+pbo to cip a+mig (switch group; 29 ERT-experienced; 8 ERT-naive). Efficacy endpoints of The study included primary endpoint of change of change from PROPEL baseline to approximately 4 years in 6-minute walk distance (6MWD), forced vital capacity (FVC), creatine kinase (CK) and hexosetetrasaccharide (Hex4) levels, patient-reported outcomes and safety. Annuncio • Jul 31
Amicus Therapeutics, Inc. Re-Iterates Financial Guidance for the Full Year 2025 Amicus Therapeutics, Inc. re-iterated financial guidance for the full year 2025. For the year, the company expects total revenue of 15% to 22%. GAAP Net income is Positive during second half of 2025. Annuncio • Jul 21
Amicus Therapeutics, Inc. to Report Q2, 2025 Results on Jul 31, 2025 Amicus Therapeutics, Inc. announced that they will report Q2, 2025 results on Jul 31, 2025 Annuncio • Jun 25
Amicus Therapeutics Announces Approval of Pombiliti® (Cipaglucosidase Alfa) + Opfolda® (miglustat) in Japan Amicus Therapeutics announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has approved Pombiliti (cipaglucosidase alfa) + Opfolda (miglustat) for the treatment of adult patients with late-onset Pompe disease (LOPD). Pombiliti + Opfolda is a two-component therapy. Pombiliti is a recombinant human GAA enzyme (rhGAA) naturally expressed with high levels of bis-M6P (Mannose 6-Phosphate), designed for increased uptake into muscle cells. Opfolda is an enzyme stabilizer designed to stabilize the enzyme in the blood. The MHLW approval for Pombiliti + Op folda was based on clinical data from the Phase 3 pivotal study (PROPEL). PROPEL is the only trial in LOPD to study the real-world population of both ERT-naive and ERT-experienced participants in a controlled setting. With this approval, Pombiliti + Op foldinga is now approved in the U.S., E.U., U.K., Canada, Australia, Switzerland and Japan. Pombiliti® + Opfolda®, is a two-component therapy that consists of cipaglucosidases alfa-atga, a bis-M6P-enriched rgAA that facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that's designed to reduce loss of enzyme activity in the blood. U.S. INDICATIONSANDUSAGE POMBILITI in combination with OPFOLDA is indicated for the treatment of adult patients With late-onset Pompe disease ("lysosomal acid alpha-glucosidase [GAA] deficiency) weighing 40 kg and who are not improving on their current enzyme replacement therapy (ERT). SFETY INFORMATION including ANAPHYLAXIS: Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for at least 60 days after the last dose. Adverse Reactions: Most common adverse reactions < 5% are headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia. Annuncio • Jun 03
Amicus Therapeutics Announces New Analysis of Pombiliti (Cipaglucosidase Alfa-atga) + Opfolda®? (miglustat) Published in Muscle and Nerve Amicus Therapeutics announced the publication of a post-hoc analysis of data from the ERT-experienced cohort of the PROPEL study of cipaglucosidase alfa-atga + miglustat (cipa+mig) in adults with late-onset Pompe disease (LOPD) in Muscle and Nerve. In this new publication, based on a within group effect-size analysis, subjects who switched from cipa+mig to cipa+mig achieved improvements or stability in most of the outcomes measured. In PROPEL, 77% of patients (n=95) received enzyme replacement therapy (ERT) with alglucosidase alfa before study entry, with a median ERT duration of 7.4 years. In this new publication of post-hoc analysis from PROPEL,ERT-experienced patients switched to cipa+mIG (n=65), showed improvement (d0.2) or stability (0.2. About the PROPEL Study: PROPEL was a 52-week, double-blind randomized global study designed to assess the efficacy, safety, and tolerability of cipaglucOSidase alfa-atgas + miglustat compared to non-U.S. approved alglucosidase Alfa + miglustat (the comparator). The study enrolled 123 adult LOPD patients who still had the ability to walk and to breathe without mechanical ventilation. Patients enrolled in PROPEL were randomized 2:1 so that for every two patients randomized to be treated with cipaglucosIDase alfa-atga+ miglustat, one was randomized to be treated with the comparator. Of the patients enrolled in PROPEL, 77% were being treated with alglucosidases alfa (n=95) for at least 2 years at study entry and 23% had never been treated with any ERT (n=28). 117 of the 123 patients (>95%) completed the PROPEL study. Efficacy endpoints of the study included primary endpoint of change from baseline to week 52 in 6-minute walk distance (6MWD) for comparison of superiority and key secondary endpoint of change from baseline toweek 52 in 6MWD in the overall population. After 52 weeks, ERT-experienced patients treated with cipaglUCosidase alfa- atga + miglustat (n=55) showed an estimated treatment difference of 3.5% (95% CI, 1.0, 6.0) in FVC compared with the comparator group (n=29). Adverse Reactions: Most common adverse reactions < 5% are headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia. Annuncio • Apr 28
Amicus Therapeutics, Inc., Annual General Meeting, Jun 05, 2025 Amicus Therapeutics, Inc., Annual General Meeting, Jun 05, 2025. Annuncio • Apr 21
Amicus Therapeutics, Inc. to Report Q1, 2025 Results on May 01, 2025 Amicus Therapeutics, Inc. announced that they will report Q1, 2025 results on May 01, 2025 Annuncio • Feb 21
Amicus Therapeutics, Inc. Provides Earnings Guidance for the Year 2025 Amicus Therapeutics, Inc. provided earnings guidance for the year 2025. For the period, the company expects total revenue growth of 17% to 24%. The company expects GAAP Net Income positive during second half of 2025. Annuncio • Feb 10
Amicus Therapeutics, Inc. to Report Fiscal Year 2024 Results on Feb 19, 2025 Amicus Therapeutics, Inc. announced that they will report fiscal year 2024 results on Feb 19, 2025 Reported Earnings • Nov 07
Third quarter 2024 earnings released: US$0.022 loss per share (vs US$0.073 loss in 3Q 2023) Third quarter 2024 results: US$0.022 loss per share (improved from US$0.073 loss in 3Q 2023). Revenue: US$141.5m (up 37% from 3Q 2023). Net loss: US$6.73m (loss narrowed 69% from 3Q 2023). Revenue is forecast to grow 19% p.a. on average during the next 3 years, compared to a 22% growth forecast for the Biotechs industry in Europe. Over the last 3 years on average, earnings per share has increased by 31% per year but the company’s share price has only increased by 1% per year, which means it is significantly lagging earnings growth. Annuncio • Nov 06
Amicus Therapeutics, Inc. Raises Earnings Guidance for the Year 2024 Amicus Therapeutics, Inc. raised earnings guidance for the year 2024. For the period, the company expects Total Revenue Growth of 30% to 32% from previous guidance of 26% to 31%. Annuncio • Oct 28
Amicus Therapeutics, Inc. to Report Q3, 2024 Results on Nov 06, 2024 Amicus Therapeutics, Inc. announced that they will report Q3, 2024 results on Nov 06, 2024 Breakeven Date Change • Sep 17
Forecast to breakeven in 2025 The 13 analysts covering Amicus Therapeutics expect the company to break even for the first time. New consensus forecast suggests the company will make a profit of US$60.1m in 2025. Average annual earnings growth of 62% is required to achieve expected profit on schedule. Reported Earnings • Aug 09
Second quarter 2024 earnings released: US$0.052 loss per share (vs US$0.15 loss in 2Q 2023) Second quarter 2024 results: US$0.052 loss per share (improved from US$0.15 loss in 2Q 2023). Revenue: US$126.7m (up 34% from 2Q 2023). Net loss: US$15.7m (loss narrowed 64% from 2Q 2023). Revenue is forecast to grow 21% p.a. on average during the next 3 years, compared to a 20% growth forecast for the Biotechs industry in Europe. Over the last 3 years on average, earnings per share has increased by 27% per year but the company’s share price has remained flat, which means it is significantly lagging earnings. Annuncio • Aug 09
Amicus Therapeutics, Inc. Raises Earnings Guidance for the Year 2024 Amicus Therapeutics, Inc. raised earnings guidance for the year 2024. For the year, the company expects total Revenue Growth of 26% to 31% from previous guidance of 25% to 30%. Annuncio • Jul 30
Amicus Therapeutics, Inc. to Report Q2, 2024 Results on Aug 08, 2024 Amicus Therapeutics, Inc. announced that they will report Q2, 2024 results on Aug 08, 2024 Annuncio • May 10
Amicus Therapeutics, Inc. Provides Earnings Guidance for the Year 2024 Amicus Therapeutics, Inc. provides earnings guidance for the year 2024. For the period, the company expects Total Revenue Growth 25% to 30%. Reported Earnings • May 10
First quarter 2024 earnings released: US$0.16 loss per share (vs US$0.18 loss in 1Q 2023) First quarter 2024 results: US$0.16 loss per share (improved from US$0.18 loss in 1Q 2023). Revenue: US$110.4m (up 28% from 1Q 2023). Net loss: US$48.4m (loss narrowed 8.5% from 1Q 2023). Revenue is forecast to grow 23% p.a. on average during the next 3 years, compared to a 18% growth forecast for the Biotechs industry in Europe. Over the last 3 years on average, earnings per share has increased by 22% per year but the company’s share price has only increased by 7% per year, which means it is significantly lagging earnings growth. Annuncio • May 03
Amicus Therapeutics, Inc. to Report Q1, 2024 Results on May 09, 2024 Amicus Therapeutics, Inc. announced that they will report Q1, 2024 results on May 09, 2024 Annuncio • Apr 26
Amicus Therapeutics, Inc., Annual General Meeting, Jun 06, 2024 Amicus Therapeutics, Inc., Annual General Meeting, Jun 06, 2024, at 09:00 Eastern Daylight. Agenda: To elect three Class II directors as nominated by the Board of Directors each to serve a three-year term expiring at the 2027 Annual Meeting or until their respective successors have been elected; to approve the Amended and Restated 2007 Equity Incentive Plan to add 7,000,000 shares to the equity pool; to ratify the appointment of Ernst & Young LLP as the Company's independent registered public accounting firm for the fiscal year ending December 31,2024; to approve, on an advisory basis, the Company's executive compensation; and to Consider and act upon any other business that is properly presented at the meeting. Breakeven Date Change • Apr 12
Forecast breakeven date pushed back to 2025 The 11 analysts covering Amicus Therapeutics previously expected the company to break even in 2024. New consensus forecast suggests losses will reduce by 100% to 2024. The company is expected to make a profit of US$120.8m in 2025. Average annual earnings growth of 55% is required to achieve expected profit on schedule. Breakeven Date Change • Mar 18
Forecast breakeven date moved forward to 2024 The 11 analysts covering Amicus Therapeutics previously expected the company to break even in 2026. New consensus forecast suggests the company will make a profit of US$15.8m in 2024. Earnings growth of 53% is required to achieve expected profit on schedule. Annuncio • Feb 29
Amicus Therapeutics, Inc. Provides Earnings Guidance for the Year 2024 Amicus Therapeutics, Inc. provided earnings guidance for the year 2024. For the full-year 2024, the company anticipates total Galafold revenue growth between 11% and 16% at CER. Reported Earnings • Feb 29
Full year 2023 earnings released: US$0.51 loss per share (vs US$0.82 loss in FY 2022) Full year 2023 results: US$0.51 loss per share (improved from US$0.82 loss in FY 2022). Revenue: US$399.4m (up 21% from FY 2022). Net loss: US$151.6m (loss narrowed 36% from FY 2022). Revenue is forecast to grow 22% p.a. on average during the next 3 years, compared to a 13% growth forecast for the Biotechs industry in Germany. Over the last 3 years on average, earnings per share has increased by 19% per year whereas the company’s share price has increased by 15% per year. Annuncio • Feb 16
Amicus Therapeutics, Inc. to Report Fiscal Year 2023 Results on Feb 28, 2024 Amicus Therapeutics, Inc. announced that they will report fiscal year 2023 results on Feb 28, 2024 Annuncio • Jan 08
Amicus Therapeutics, Inc. Provides Revenue Guidance for the Year 2024 Amicus Therapeutics, Inc. provided revenue guidance for the year 2024. Amicus is focused on the following four key strategic priorities in 2024: Delivering double-digit Galafold revenue growth (11%-16% at CER). Board Change • Jan 01
Insufficient new directors There is 1 new director who has joined the board in the last 3 years. The company's board is composed of: 1 new director. 5 experienced directors. 4 highly experienced directors. Independent Director Eiry Roberts was the last director to join the board, commencing their role in 2021. The company’s insufficient board refreshment is considered a risk according to the Simply Wall St Risk Model. Annuncio • Dec 05
Amicus Therapeutics Announces Executive Changes Amicus Therapeutics announced that John F. Crowley, Executive Chairman of Amicus Therapeutics, will step down from the Amicus Board of Directors and assume the role of President and Chief Executive Officer of BIO, the Biotechnology Innovation Organization, effective March 4, 2024. Michael Raab, Lead Independent Director, will become the Chairman of the Amicus Board of Directors. BIO is based in Washington, DC and is the world’s largest biotechnology policy and advocacy association, representing the entire biotechnology industry, with over 1,200 member organizations worldwide. Mr. Crowley joined Amicus in 2005 and served as Chairman and Chief Executive Officer until 2022, when he transitioned to his current role as Executive Chairman. Under Mr. Crowley’s leadership, Amicus has grown from a five-person early-stage start-up to a multi-billion dollar, publicly traded, commercial organization with two marketed therapies and over 500 employees in 21 countries around the world, treating thousands of people living with rare and devastating diseases. Reported Earnings • Nov 09
Third quarter 2023 earnings released: US$0.073 loss per share (vs US$0.12 loss in 3Q 2022) Third quarter 2023 results: US$0.073 loss per share (improved from US$0.12 loss in 3Q 2022). Revenue: US$103.5m (up 27% from 3Q 2022). Net loss: US$21.6m (loss narrowed 35% from 3Q 2022). Revenue is forecast to grow 25% p.a. on average during the next 3 years, compared to a 15% growth forecast for the Biotechs industry in Germany. Over the last 3 years on average, earnings per share has increased by 17% per year but the company’s share price has fallen by 16% per year, which means it is significantly lagging earnings. Annuncio • Oct 28
Amicus Therapeutics, Inc. to Report Q3, 2023 Results on Nov 08, 2023 Amicus Therapeutics, Inc. announced that they will report Q3, 2023 results on Nov 08, 2023 Annuncio • Oct 17
Amicus Therapeutics, Inc. announced that it has received $29.999985 million in funding On October 16, 2023, Amicus Therapeutics, Inc. closed the transaction. The transaction included participation from two investors. Annuncio • Sep 29
Amicus Therapeutics Announces FDA Approval and Launch of New Treatment for Pompe Disease Amicus Therapeutics announced that the U.S. Food and Drug Administration (FDA) has approved Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules. This two-component therapy is indicated for adults living with late-onset Pompe disease (LOPD) weighing =40 kg and who are not improving on their current enzyme replacement therapy (ERT). Late-onset Pompe disease is a rare, debilitating, and life-threatening lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced levels of GAA lead to the accumulation of the substrate glycogen in the lysosomes of muscle cells and glycogen buildup causes muscle damage. Disease severity ranges across a spectrum, with predominant manifestations such as skeletal muscle weakness and progressive respiratory involvement. Pombiliti + Opfolda is a unique two-component therapy. Pombiliti is a recombinant human GAA enzyme (rhGAA) naturally expressed with high levels of bis-M6P (Mannose 6-Phosphate), designed for increased uptake into muscle cells. Once in the cell, Pombiliti can be properly processed into its most active and mature form to break down glycogen. Opfolda is an enzyme stabilizer designed to stabilize the enzyme in the blood. The FDA approval was based on clinical data observed from the Phase 3 pivotal study (PROPEL), the only trial in LOPD to study ERT-experienced participants in a controlled setting. Amicus Therapeutics will launch Pombiliti + Opfolda immediately in the U.S. The FDA previously granted Breakthrough Therapy designation for Pombiliti + Opfolda. Pombiliti + Opfolda has also been approved for the treatment of adults with LOPD in the European Union and the United Kingdom. Annuncio • Aug 24
New Paradigm Therapeutics, Inc. acquired worldwide rights of SD-101 from Amicus Therapeutics, Inc. (NasdaqGM:FOLD). New Paradigm Therapeutics, Inc. acquired worldwide rights of SD-101 from Amicus Therapeutics, Inc. (NasdaqGM:FOLD) on August 22, 2023. The Paradigm Therapeutics team will be working passionately to provide patients with a new therapeutic alternative, in this disease with no effective whole-body treatments.New Paradigm Therapeutics, Inc. completed the acquisition of worldwide rights of SD-101 from Amicus Therapeutics, Inc. (NasdaqGM:FOLD) on August 22, 2023. Annuncio • Aug 22
Amicus Therapeutics, Inc. Announces Chief Financial Officer Changes Amicus Therapeutics, Inc. announced that Simon Harford has been appointed Chief Financial Officer, effective as of August 21, 2023. Simon brings extensive financial leadership experience, having served in significant finance roles in two global pharmaceutical companies as well as most recently as a public company CFO in the biotechnology industry. Mr. Harford will take the place of Daphne Quimi, who, as previously disclosed, informed the Company of her intention to retire before the end of 2023. Ms. Quimi will remain employed by the Company as Senior Vice President, Finance, for a transition period to ensure an orderly transition of duties and responsibilities. Prior to joining the Company, Mr. Harford, 63, served as Chief Financial Officer of Albireo Pharma Inc., from October 2018. Prior to that, Mr. Harford served as Chief Financial Officer of Parexel International, Inc., from May 2017 to October 2018. Prior to that, Mr. Harford served as Senior Vice President, Finance of GlaxoSmithKline plc, Group Vice President and Controller of Avon Products, Inc. and a variety of financial-oriented roles at Eli Lilly and Company. Mr. Harford holds a Master of Business Administration from Darden Graduate School of Business Administration at the University of Virginia. Annuncio • Aug 17
Amicus Therapeutics Announces Approval and Launch of New Pompe Disease Therapy in the United Kingdom Amicus Therapeutics announced that the Medicines and Healthcare products Regulatory Agency (MHRA) of the United Kingdom (U.K.) has granted marketing authorizations for Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) for adults living with late-onset Pompe disease (acid a-glucosidase [GAA] deficiency). The indication for Pombiliti is a long-term enzyme replacement therapy (ERT) used in combination with miglustat for adults with late-onset Pompe disease (LOPD). The indication for Opfolda is an enzyme stabilizer of cipaglucosidase alfa long-term enzyme replacement therapy for adults with LOPD. In addition, the National Institute for Health and Care Excellence (NICE) issued final guidance recommending reimbursement of Pombiliti + Opfolda for use within the National Health Service (NHS) in England and Wales. As stated in the guidance, NICE concluded that the cost-effectiveness estimates for Pombiliti + Opfolda showed a positive net health benefit and recommended Pombiliti + Opfolda for adults with LOPD as first line and later lines of therapy. Prior to Great Britain (GB) approval, Pombiliti + Opfolda was granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP), a Priority Innovative Medicines designation, as well as a positive scientific opinion under the Early Access to Medicines Scheme (EAMS). This accelerated the time to market and enabled healthcare professionals to prescribe the treatment prior to marketing authorization based on clinical factors for patients with a clear unmet need. Late-onset Pompe disease (LOPD) is a rare, debilitating, and life-threatening lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced levels of GAA lead to the accumulation of the substrate glycogen in the lysosomes of muscles and other tissues. Disease severity ranges on a spectrum, but predominant manifestations are skeletal muscle weakness and progressive respiratory involvement. The MHRA and NICE decisions were based on clinical data from the Phase 3 pivotal study (PROPEL), the only trial in LOPD to study both ERT-naïve and ERT-experienced participants in a controlled setting. Pombiliti + Opfolda has demonstrated fulfilment of the Great Britain orphan designation criteria and will be added to the Orphan Register held by the MHRA. Both will benefit from 10 years of market exclusivity in respect of similar medicinal products in the approved orphan indication. Pombiliti + Opfolda, is a two-component therapy that consists of cipaglucosidase alfa, a bis-M6P-enriched rhGAA that facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that’s designed to reduce loss of enzyme activity in the blood. In clinical studies, Pombiliti + Opfolda was associated with demonstrated improvements in both musculoskeletal and respiratory measures. New Risk • Aug 10
New minor risk - Shareholder dilution The company's shareholders have been diluted in the past year. Increase in shares outstanding: 2.4% This is considered a minor risk. Shareholder dilution occurs when there is an increase in the number of shares on issue that is not proportionally distributed between all shareholders. Often due to the company raising equity capital or some options being converted into stock. All else being equal, if there are more shares outstanding then each existing share will be entitled to a lower proportion of the company's total earnings, thus reducing earnings per share (EPS). While dilution might not always result in lower EPS (like if the company is using the capital to fund an EPS accretive acquisition) in a lot cases it does, along with lower dividends per share and less voting power at shareholder meetings. This is currently the only risk that has been identified for the company. Reported Earnings • Aug 09
Second quarter 2023 earnings released: US$0.15 loss per share (vs US$0.21 loss in 2Q 2022) Second quarter 2023 results: US$0.15 loss per share (improved from US$0.21 loss in 2Q 2022). Revenue: US$94.5m (up 17% from 2Q 2022). Net loss: US$43.2m (loss narrowed 30% from 2Q 2022). Revenue is forecast to grow 25% p.a. on average during the next 3 years, compared to a 14% growth forecast for the Biotechs industry in Germany. Over the last 3 years on average, earnings per share has increased by 15% per year but the company’s share price has only increased by 1% per year, which means it is significantly lagging earnings growth. Annuncio • Aug 01
Amicus Therapeutics, Inc. to Report Q2, 2023 Results on Aug 08, 2023 Amicus Therapeutics, Inc. announced that they will report Q2, 2023 results on Aug 08, 2023 Annuncio • Jul 14
Amicus Therapeutics, Inc. Appoints Samantha Prout as Chief Accounting Officer and Controller On July 10, 2023, Amicus Therapeutics, Inc. (the “Company”) appointed Samantha Prout, 45, as the Company’s Chief Accounting Officer and Controller. Ms. Prout had previously been the Company’s principal accounting officer from June 2018 to September 2021. Ms. Prout joined the Company in March 2018 and has held the positions of Senior Director & Global Controller, Executive Director & Global Controller, and Vice President & Global Controller prior to her promotion to Senior Vice President, Global Controller in 2021. Prior to joining the Company, Ms. Prout spent nearly three years at NRG Energy (“NRG”), first as Controller for its Home Solar division, then as the Director of Financial Reporting, following 15 years at KPMG LLP. Ms. Prout received a Bachelor of Arts from Gettysburg College and is a certified public accountant in New Jersey. Annuncio • Jun 28
Amicus Therapeutics Inc. Announces Approval and Launch of New Pompe Disease Therapy in the European Union Amicus Therapeutics, Inc. announced that the European Commission (EC) has granted approval for Opfolda(R) (miglustat) 65mg capsules, an enzyme stabilizer of cipaglucosidase alfa, a long-term enzyme replacement therapy for adults with late-onset Pompe disease. Pombiliti (cipaglucosidases alfa), is a bis-M6P-enriched rhGAA enzyme, designed for increased uptake into muscle cells. Once in the cell, Pombiliti can be processed into its most active and mature form to break down glycogen. Opfolda (miglustat) is an enzyme stabilizer designed to stabilize the enzyme in the blood. The EC approval was based on clinical data from the Phase 3 pivotal study (PROPEL), the only trial in LOPD to study the real-world population of both ERT-nave and ERT-experienced participants in a controlled setting. In the U.K., the Medicines and Healthcare products Regulatory Agency (MHRA) regulatory approval is expected in the third quarter of 2023. The U.S. Food and Drug Administration's review is ongoing, and the Company expects approval in the third quarter of 20 23. The FDA previously granted Breakthrough Therapy designation for Pombiliti + Opfolda. Pombiliti + Op folda, is a two-component therapy that consists of cipaglucOSidase alfa, a bis-M6P -enriched rhGAA that facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that's designed to reduce loss of enzyme activity in the blood. In clinical studies, Pombiliti + Op foldinga was associated with demonstrated improvements in both muscleoskeletal and respiratory measures. Refer to SmPC for full list. Annuncio • May 11
Amicus Therapeutics, Inc. Provides Earnings Guidance for the Year 2023 Amicus Therapeutics, Inc. provided earnings guidance for the year 2023. For the full-year 2023, the company anticipates total Galafold revenue growth between 12% and 17% at CER driven by continued underlying demand from both switch and treatment-naïve patients, geographic expansion, label extensions, the continued diagnosis of new Fabry patients, and commercial execution across all major markets, including the U.S., EU, U.K., and Japan. Reported Earnings • May 10
First quarter 2023 earnings released: US$0.18 loss per share (vs US$0.30 loss in 1Q 2022) First quarter 2023 results: US$0.18 loss per share (improved from US$0.30 loss in 1Q 2022). Revenue: US$86.3m (up 9.6% from 1Q 2022). Net loss: US$52.9m (loss narrowed 38% from 1Q 2022). Revenue is forecast to grow 28% p.a. on average during the next 3 years, compared to a 20% growth forecast for the Biotechs industry in Germany. Over the last 3 years on average, earnings per share has increased by 14% per year but the company’s share price has remained flat, which means it is significantly lagging earnings. Reported Earnings • Mar 02
Full year 2022 earnings released: US$0.82 loss per share (vs US$0.92 loss in FY 2021) Full year 2022 results: US$0.82 loss per share (improved from US$0.92 loss in FY 2021). Revenue: US$329.2m (up 7.8% from FY 2021). Net loss: US$236.6m (loss narrowed 5.5% from FY 2021). Revenue is forecast to grow 23% p.a. on average during the next 3 years, compared to a 21% growth forecast for the Biotechs industry in Europe. Over the last 3 years on average, earnings per share has increased by 15% per year whereas the company’s share price has increased by 13% per year. Annuncio • Feb 15
Amicus Therapeutics, Inc. to Report Q4, 2022 Results on Mar 01, 2023 Amicus Therapeutics, Inc. announced that they will report Q4, 2022 results at 9:30 AM, US Eastern Standard Time on Mar 01, 2023 Annuncio • Jan 10
Amicus Therapeutics, Inc. Provides Revenue Guidance for the Year 2023 Amicus Therapeutics, Inc. provided revenue guidance for the year 2023. For the year, the company anticipates double-digit Galafold revenue growth of 12% to 17% at constant exchange rates. Growth is expected to be driven by continued underlying demand from both switch and treatment-naïve patients, geographic expansion, continued diagnosis of new Fabry patients, and commercial execution across all major markets, including the U.S., EU, U.K., and Japan. Annuncio • Dec 17
Amicus Therapeutics Receives Positive CHMP Opinion for Pombiliti™ (cipaglucosidase alfa) for Late-Onset Pompe Disease Amicus Therapeutics announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending marketing authorization of cipaglucosidase alfa, a long-term enzyme replacement therapy (ERT) used in combination with miglustat for adults with late-onset Pompe disease (LOPD). A decision from the European Commission (EC) on cipaglucosidase alfa, the enzyme replacement therapy component of AT-GAA, is expected in the first quarter of 2023. Cipaglucosidase alfa will be commercialized under the brand name POMBILITI™. The Company expects a CHMP opinion of miglustat, the enzyme stabilizer component of AT-GAA, in the second quarter of 2023. Late-onset Pompe disease is a rare, debilitating, and life-threatening lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to the accumulation of the substrate glycogen in the lysosomes of muscles and other tissues. Disease severity ranges on a spectrum, but predominant manifestations are skeletal muscle weakness and progressive respiratory involvement. AT-GAA is designed as a two-component therapy consisting of cipaglucosidase alfa, a long-term enzyme replacement therapy, administered in combination with miglustat, an oral enzyme stabilizer, for the treatment of adults with late-onset Pompe disease. Cipaglucosidase alfa is a recombinant human acid alpha-glucosidase enzyme (rhGAA) enriched with bis-mannose-6-phosphate designed to facilitate high-affinity uptake with retained capacity for processing into the most active form of the enzyme to break down glycogen. The CHMP based its positive opinion on clinical data from the Phase 3 pivotal study (PROPEL), the only randomized, controlled trial in LOPD to include patients in the high unmet need ERT-experienced population, in addition to ERT-naïve patients. As anticipated and consistent with a recent opinion of another ERT in this disease space, the CHMP also determined cipaglucosidase alfa does not qualify as a New Active Substance (NAS). Reported Earnings • Nov 08
Third quarter 2022 earnings released: US$0.12 loss per share (vs US$0.19 loss in 3Q 2021) Third quarter 2022 results: US$0.12 loss per share (improved from US$0.19 loss in 3Q 2021). Revenue: US$81.7m (up 2.7% from 3Q 2021). Net loss: US$33.3m (loss narrowed 34% from 3Q 2021). Revenue is forecast to grow 26% p.a. on average during the next 3 years, compared to a 21% growth forecast for the Biotechs industry in Germany. Over the last 3 years on average, earnings per share has increased by 17% per year but the company’s share price has only increased by 7% per year, which means it is significantly lagging earnings growth. Annuncio • Nov 08
Amicus Therapeutics, Inc. Provides Revenue Guidance for the Full-Year of 2022 Amicus Therapeutics, Inc. provided revenue guidance for the full-year of 2022. For the year the company anticipates total Galafold revenue growth between 15% and 20% at CER driven by continued underlying demand from both switch and treatment-naive patients, geographic expansion, the continued diagnosis of new Fabry patients and commercial execution across all major markets, including the U.S., EU, U.K., and Japan. Applying average October 2022 exchange rates, the negative currency impact on full-year 2022 Galafold reported sales would be approximately 9%. Annuncio • Oct 29
Amicus Therapeutics, Inc. Announces U.S. FDA Defers Action on Filing for AT-GAA in Late-onset Pompe Disease Amicus Therapeutics announced that the U.S. Food and Drug Administration (FDA) has deferred action on the Biologics License Application (BLA) for cipaglucosidase alfa, the biologic component of AT-GAA. Due to restrictions on travel related to COVID-19, the FDA was unable to conduct the required inspection of the WuXi Biologics manufacturing site in China during the review cycle. As a result, the FDA is deferring action on the application until the manufacturing site inspection is complete. The Company continues to expect the FDA to approve the two components of AT-GAA, including the BLA and New Drug Application (NDA) for miglustat, together. The sole reason cited in the FDA-issued letter for the deferred action was the Agency’s inability to complete the manufacturing facility inspection. While both applications remain under review, the FDA has not provided anticipated action date(s) as they continue to monitor the public health situation and travel restrictions in China. However, the Company is now actively engaged with the FDA on developing plans and logistics for a pre-approval inspection plan. Under FDA guidance relating to pre-approval inspections during the COVID-19 pandemic, the Agency may defer action on a pending application when a facility inspection is necessary but cannot be completed by the PDUFA goal date due to travel restrictions, provided that no deficiencies have been identified and the application otherwise satisfies the requirements for approval. Annuncio • Oct 28
Amicus Therapeutics, Inc. to Report Q3, 2022 Results on Nov 07, 2022 Amicus Therapeutics, Inc. announced that they will report Q3, 2022 results on Nov 07, 2022 Recent Insider Transactions • Sep 20
Executive Chairman recently sold €112k worth of stock On the 15th of September, John Crowley sold around 10k shares on-market at roughly €10.67 per share. This transaction amounted to 1.1% of their direct individual holding at the time of the trade. This was the largest sale by an insider in the last 3 months. This was John's only on-market trade for the last 12 months. Reported Earnings • Aug 05
Second quarter 2022 earnings released: US$0.21 loss per share (vs US$0.19 loss in 2Q 2021) Second quarter 2022 results: US$0.21 loss per share (down from US$0.19 loss in 2Q 2021). Revenue: US$80.7m (up 4.3% from 2Q 2021). Net loss: US$62.2m (loss widened 21% from 2Q 2021). Over the next year, revenue is forecast to grow 30%, compared to a 31% growth forecast for the industry in Germany. Over the last 3 years on average, earnings per share has increased by 25% per year but the company’s share price has remained flat, which means it is significantly lagging earnings. Annuncio • Aug 05
Amicus Therapeutics, Inc. Provides Guidance for 2022 Amicus Therapeutics, Inc. provided guidance for 2022. For the year the company anticipates total Galafold revenue of $350 million to $365 million at constant exchange rates. Double-digit revenue growth between 15 and 20% at CER1 in 2022 is expected to be driven by continued underlying demand from both switch and treatment-naïve patients, geographic expansion, the continued diagnosis of new Fabry patients and commercial execution across all major markets, including the U.S., EU, U.K., and Japan. Applying average July 2022 exchange rates, the negative currency impact on full-year 2022 Galafold reported sales would be approximately 9%. Annuncio • Aug 02
Amicus Therapeutics, Inc. Appoints Bradley L. Campbell as Chief Executive Officer As previously announced by Amicus Therapeutics, Inc. on February 24, 2022, Bradley L. Campbell, who has served as a member of the Company’s board of directors since June 2018 and as President and Chief Operating Officer since January 2015, and Chief Operating Officer since December 2013, has been appointed Chief Executive Officer of the Company. Mr. Campbell, 47, joined Amicus in 2006. Mr. Campbell currently serves on a number of boards of directors, including Gennao Bio, the Alliance for Regenerative Medicine (ARM), and the Corporate Advisory Board for the National Tay-Sachs and Allied Diseases Association. He previously served on the Board of ARYA Sciences Acquisition Corp. III, a healthcare focused special purpose acquisition vehicle, as well as Progenics Pharmaceuticals from 2016 until its successful acquisition by Lantheus Holdings in 2020. Prior to working at the Company, Mr. Campbell spent time in various commercial and business development roles at Genzyme and Bristol-Myers Squibb and as a strategy consultant for Marakon Associates. He received a B.A. in Public Policy Studies from Duke University and an M.B.A. from Harvard Business School. On August 1, 2022, Mr. Campbell entered into an employment agreement with the Company pursuant to which Mr. Campbell will serve as the Chief Executive Officer of the Company beginning on August 1, 2022 until Mr. Campbell’s employment is terminated by either Mr. Campbell or the Company. The CEO Employment Agreement supersedes Mr. Campbell’s prior employment agreement with the Company. Annuncio • May 12
Amicus Therapeutics Receives Notification of PDUFA Date Extensions for AT-GAA Amicus Therapeutics announced that the U.S. Food and Drug Administration (FDA) has extended the review period by 90 days for the Biologics License Application (BLA) for cipaglucosidase alfa and the New Drug Application (NDA) for miglustat, the two components of AT-GAA. The revised PDUFA action dates for miglustat and cipaglucosidase alfa are August 29, 2022 and October 29, 2022, respectively. The Company continues to expect the FDA to approve the applications together. The FDA extended the PDUFA dates to allow additional time to review information submitted by the Company as part of its ongoing reviews. The extension of the review timeline was not related to requests for any additional clinical data. The company also expects that the additional time will allow for the completion of the pre-license approval inspections necessary at the WuXi Biologics manufacturing site in China. Previously, the FDA granted Breakthrough Therapy Designation to AT-GAA for the treatment of late-onset Pompe disease based on clinical efficacy results from the Phase 1/2 clinical study. In the European Union, the Marketing Authorization Applications were validated in the fourth quarter of 2021 and the Committee for Medicinal Products for Human Use (CHMP) opinion is expected in late 2022. AT-GAA is an investigational two-component therapy that consists of cipaglucosidase alfa (ATB200), a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly bis-phosphorylated mannose-6 phosphate (bis-M6P) glycans, to enhance uptake into cells, administered in conjunction with miglustat (AT2221), a stabilizer of cipaglucosidase alfa. In preclinical studies, AT-GAA was associated with increased levels of the mature lysosomal form of GAA and reduced glycogen levels in muscle, alleviation of the autophagic defect and improvements in muscle strength. In addition, Amicus is enrolling an open-label, uncontrolled, multicenter study to evaluate the PK, safety, efficacy, and PD of AT-GAA in pediatric patients aged 0 to 18 years with LOPD (ATB200-04). More information, including a list of participating sites, is available at www.clinicaltrials.gov: NCT03911505. Reported Earnings • May 10
First quarter 2022 earnings released: US$0.30 loss per share (vs US$0.25 loss in 1Q 2021) First quarter 2022 results: US$0.30 loss per share (down from US$0.25 loss in 1Q 2021). Revenue: US$78.7m (up 19% from 1Q 2021). Net loss: US$85.3m (loss widened 30% from 1Q 2021). Over the next year, revenue is forecast to grow 24%, compared to a 37% growth forecast for the industry in Germany. Over the last 3 years on average, earnings per share has increased by 31% per year but the company’s share price has fallen by 17% per year, which means it is significantly lagging earnings. Annuncio • May 10
Amicus Therapeutics, Inc. Provides Revenue Guidance for the Full Year of 2022 Amicus Therapeutics, Inc. provided revenue guidance for the full year of 2022. For the year, the company expects total Galafold revenue of $350 million to $365 million at constant exchange rates. Double-digit revenue growth between 15 and 20% at CER1 in 2022 is expected to be driven by continued underlying demand from both switch and naïve patients, geographic expansion, the continued diagnosis of new Fabry patients and commercial execution across all major markets, including the U.S., EU, U.K., and Japan. Applying average April 2022 exchange rates, the negative currency impact on full-year 2022 Galafold reported sales would be approximately 6%. Breakeven Date Change • Apr 13
Forecast to breakeven in 2024 The 11 analysts covering Amicus Therapeutics expect the company to break even for the first time. New consensus forecast suggests the company will make a profit of US$137.1m in 2024. Average annual earnings growth of 64% is required to achieve expected profit on schedule. Breakeven Date Change • Feb 26
Forecast breakeven date pushed back to 2024 The 10 analysts covering Amicus Therapeutics previously expected the company to break even in 2023. New consensus forecast suggests losses will reduce by 78% per year to 2023. The company is expected to make a profit of US$141.5m in 2024. Average annual earnings growth of 64% is required to achieve expected profit on schedule. Reported Earnings • Feb 25
Full year 2021 earnings: Revenues and EPS in line with analyst expectations Full year 2021 results: US$0.92 loss per share (up from US$1.07 loss in FY 2020). Revenue: US$305.5m (up 17% from FY 2020). Net loss: US$250.5m (loss narrowed 9.5% from FY 2020). Revenue was in line with analyst estimates. Over the next year, revenue is forecast to grow 19%, compared to a 61% growth forecast for the pharmaceuticals industry in Germany. Over the last 3 years on average, earnings per share has increased by 31% per year but the company’s share price has fallen by 13% per year, which means it is significantly lagging earnings. Annuncio • Feb 11
Amicus Therapeutics, Inc. to Report Fiscal Year 2021 Results on Feb 24, 2022 Amicus Therapeutics, Inc. announced that they will report fiscal year 2021 results on Feb 24, 2022 Annuncio • Jan 12
Amicus Therapeutics, Inc. Provides Unaudited Revenue Guidance for the Year 2021 and 2022 Amicus Therapeutics, Inc. provided unaudited revenue guidance for the year 2021 and 2022. Global revenue for Galafold in full-year 2021 was approximately $306 million, preliminary and unaudited, representing a year-over-year increase of 17% from total revenue of $260.9 million in 2020. Full-year revenue benefited from a positive currency impact of approximately $7 million. Fourth quarter Galafold revenue was approximately $84 million, preliminary and unaudited.For the full-year 2022, the Company anticipates total Galafold revenue of $350 million to $365 million. Double-digit revenue growth (15-20%) in 2022 is expected to be driven by continued underlying demand from both switch and naïve patients, geographic expansion, the continued diagnosis of new Fabry patients and commercial execution across all major markets, including the U.S., EU, U.K., and Japan. Annuncio • Dec 05
European Medicines Agency Validates Amicus Therapeutics Marketing Authorization Applications for AT-GAA for the Treatment of Pompe Disease Amicus Therapeutics, Inc. announced that the European Medicines Agency (EMA) validated the Marketing Authorization Applications (MAA) for AT-GAA, the Company’s investigational two-component therapy for the treatment of Pompe disease. Validation of the application confirms the submission is accepted, and the EMA’s centralized procedure with Committee for Medicinal Products for Human Use (CHMP)’s assessment begins. The MAAs were submitted to the EMA based on the evaluation of the effects of AT-GAA in adults living with Pompe disease and its safety profile, which include data from the Phase 1/2 and Phase 3 PROPEL studies, as well as data from the long-term open-label extension study. Reported Earnings • Nov 10
Third quarter 2021 earnings released: US$0.19 loss per share (vs US$0.25 loss in 3Q 2020) The company reported a solid third quarter result with reduced losses, improved revenues and improved control over expenses. Third quarter 2021 results: Revenue: US$79.5m (up 18% from 3Q 2020). Net loss: US$50.3m (loss narrowed 21% from 3Q 2020). Over the last 3 years on average, earnings per share has increased by 30% per year but the company’s share price has only increased by 3% per year, which means it is significantly lagging earnings growth. Breakeven Date Change • Oct 01
Forecast to breakeven in 2023 The 11 analysts covering Amicus Therapeutics expect the company to break even for the first time. New consensus forecast suggests the company will make a profit of US$29.4m in 2023. Average annual earnings growth of 73% is required to achieve expected profit on schedule. Reported Earnings • Aug 06
Second quarter 2021 earnings released: US$0.19 loss per share (vs US$0.20 loss in 2Q 2020) The company reported a solid second quarter result with reduced losses, improved revenues and improved control over expenses. Second quarter 2021 results: Revenue: US$77.4m (up 24% from 2Q 2020). Net loss: US$51.2m (loss narrowed 2.4% from 2Q 2020). Over the last 3 years on average, earnings per share has increased by 26% per year but the company’s share price has fallen by 15% per year, which means it is significantly lagging earnings. Annuncio • Aug 04
Amicus Therapeutics Announces European Commission Approval of Galafold® (migalastat) for Adolescents with Fabry Disease Amicus Therapeutics announced the European Commission has approved Galafold® (migalastat) for use in adolescents aged 12 to <16 years weighing = 45 kg with a confirmed diagnosis of Fabry disease and who have an amenable mutation. Galafold is already approved in multiple geographies around the world, including the U.S., EU, and Japan, for adults who have an amenable variant, or mutation. The extension of the indication was supported by 1-month interim safety and pharmacokinetics data from Study AT1001-020 which was a 2-stage, open-label, multicenter study to evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of migalastat treatment in pediatric subjects aged 12 to <18 years and weighing = 45 kg with Fabry disease and with amenable mutations to the gene encoding a-galactosidase A (GLA). The company will work closely with all relevant government authorities to secure access for eligible patients as quickly as possible. Galafold is not approved for adolescents outside of Europe. Annuncio • Jun 09
Amicus Therapeutics Announces United Kingdom’s MHRA Grants Early Access to AT-GAA Amicus Therapeutics announced that the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted a positive scientific opinion through the Early Access to Medicines Scheme (EAMS) to AT-GAA, the Company’s investigational two-component therapy for the treatment of Pompe disease. This positive opinion means that eligible adults living with late-onset Pompe disease (LOPD) who have received alglucosidase alfa for at least 2 years can now switch and have access to AT-GAA prior to marketing authorization in the UK. The EAMS mechanism is intended to provide individuals in the UK who live with a life threatening or seriously debilitating condition early access to innovative and unlicensed medicines for which there is not yet marketing authorization and where there is a clear unmet medical need. The MHRA’s decision is based on the evaluation of the effects of AT-GAA in LOPD patients and its safety profile, including data from both the Phase 1/2 and Phase 3 PROPEL study. Results from the global Phase 3 PROPEL clinical study of AT-GAA were presented at the 17th Annual WORLDSymposium™ 2021, held virtually February 8-12, 2021. Marketing Authorization Applications for AT-GAA are expected to be submitted with the UK and EU, respectively, in the second half of 2021. For the full approval in the UK, Amicus will seek inclusion of the treatment population to all ERT-switch patients regardless of length of time on prior ERT, as well as the treatment naïve population. Future studies will also investigate the treatment of patients with Infantile Onset Pompe Disease. Previously, the MHRA has granted the PIM (Priority Innovative Medicines) designation to AT-GAA. Also, the U.S. FDA granted Breakthrough Therapy Designation to AT-GAA for the treatment of LOPD based on clinical efficacy results from the Phase 1/2 clinical study. A rolling Biologics License Application (BLA) for AT-GAA was initiated with the U.S. FDA in the fourth quarter of 2020 and is on track with submission of the final modules in the second quarter of 2021. Recent Insider Transactions • May 29
Independent Director recently bought €204k worth of stock On the 25th of May, Lynn Bleil bought around 29k shares on-market at roughly €7.14 per share. This was the largest purchase by an insider in the last 3 months. Insiders have collectively bought €227k more in shares than they have sold in the last 12 months. Reported Earnings • May 11
First quarter 2021 earnings released: US$0.25 loss per share (vs US$0.35 loss in 1Q 2020) The company reported a solid first quarter result with reduced losses, improved revenues and improved control over expenses. First quarter 2021 results: Revenue: US$66.4m (up 9.7% from 1Q 2020). Net loss: US$65.7m (loss narrowed 26% from 1Q 2020). Over the last 3 years on average, earnings per share has increased by 22% per year but the company’s share price has fallen by 13% per year, which means it is significantly lagging earnings. 
