Ankündigung • Jun 30

Sarepta Therapeutics Announces FDA Acceptance Of Supplemental New Drug Applications For Amondys 45 And Vyondys 53 For Duchenne Muscular Dystrophy

Sarepta Therapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the supplemental New Drug Applications (sNDAs) for AMONDYS 45 (casimersen) and VYONDYS 53 (golodirsen) for the treatment of Duchenne muscular dystrophy (DMD). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027. The sNDA submissions seek conversion of the accelerated approvals of AMONDYS 45 and VYONDYS 53 to traditional approvals. The applications are supported by data from the ESSENCE confirmatory study, as well as substantial published real-world evidence and the favorable and consistent safety profiles of both exon-skipping therapies. EXONDYS 51 (eteplirsen), AMONDYS 45 (casimersen) and VYONDYS 53 (golodirsen) are exon-skipping therapies approved under the FDA’s accelerated approval pathway for patients with DMD who have mutations amenable to exon 51, exon 45 and exon 53 skipping, respectively. For more than a decade, Sarepta’s exon-skipping therapies have been used to treat over 1,800 amenable patients worldwide, from infants as young as 7 months to adults well into their 30s. In the Phase 3 ESSENCE study evaluating casimersen and golodirsen versus placebo, the primary endpoint was not met; however, numerical trends favored treatment. Additional post-hoc analyses were performed to evaluate heterogeneity challenges related to DMD progression and the impact of conducting ESSENCE during COVID-19. At week 96, treatment was associated with increased dystrophin expression and a consistent reduction in 4-step ascend decline across multiple analyses. The therapies were well tolerated over 144 weeks with no new safety signals observed, consistent with established clinical and real-world experience. Results from ESSENCE add to the available evidence for VYONDYS 53 and AMONDYS 45, including real-world studies demonstrating that treatment with VYONDYS 53 is associated with a 7.5 year delay in the need for nighttime ventilation and treatment with AMONDYS 45 is associated with a statistically significant slowing of lung function decline and a potentially meaningful benefit in the predicted time to use of a cough assist device. Across our PMO portfolio, real-world evidence indicates a multi-year benefit on survival, delays in time to loss of ambulation of 3 and 4 years, a substantial reduction in risk of reaching a left ventricular ejection fraction (LVEF) of less than 55%, and a significant reduction in emergency room and other hospital visits. EXONDYS 51 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion, or “skipping”, of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. EXONDYS 51 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental. Hypersensitivity reactions, including bronchospasm, chest pain, cough, tachycardia, and urticaria have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy. Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 mg or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended. The most common adverse reactions from observational clinical studies (N=163) seen in greater than 10% of patients were headache, cough, rash, and vomiting. Other adverse events may occur. VYONDYS 53 (golodirsen) uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 53 of dystrophin pre-mRNA, resulting in exclusion, or “skipping,” of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. Because of the effect of reduced skeletal muscle mass on creatinine measurements, creatinine may not be a reliable measure of kidney function in DMD patients. Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting AMONDYS 45. Consider also measuring glomerular filtration rate using an exogenous filtration marker before starting AMONDYS 45. During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to-creatinine ratio (UPCR) every three months. Only urine expected to be free of excreted AMONDYS 45 should be used for monitoring of urine protein. Urine obtained on the day of AMONDYS 45 infusion prior to the infusion, or urine obtained at least 48 hours after the most recent infusion, may be used. Alternatively, use a laboratory test that does not use the reagent pyrogallol red, as this reagent has the potential to cross react with any AMONDYS 45 that is excreted in the urine and thus lead to a false positive result for urine protein. If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation. Adverse reactions occurring in at least 20% of patients treated with AMONDYS 45 and at least 5% more frequently than in the placebo group were (AMONDYS 45, placebo): upper respiratory infections (65%, 55%), cough (33%, 26%), pyrexia (33%, 23%), headache (32%, 19%), arthralgia (21%, 10%), and oropharyngeal pain (21%, 7%). Other adverse reactions that occurred in at least 10% of patients treated with AMONDYS 45 and at least 5% more frequently than in the placebo group were: ear pain, nausea, ear infection, post-traumatic pain, and dizziness and light-headedness. Other adverse events may occur.

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Durchschnittliche Amtszeit des Managements	1.1yrs
Durchschnittliche Amtszeit der Vorstandsmitglieder	10.2yrs

Name	Position	Amtszeit	Vergütung	Eigentümerschaft
Ian Estepan	President & COO	1.1yrs	US$4.44m	0.030% € 453.1k
Ryan Wong	Executive VP & CFO	1.1yrs	US$3.21m	0.011% € 167.1k
Cristin Rothfuss	Executive VP	1.8yrs	US$4.23m	keine Daten
Douglas Ingram	Advisor	less than a year	US$7.84m	0.37% € 5.7m
Louise Rodino-Klapac	President of Research & Development and Technical Operations	1.1yrs	US$4.46m	0.052% € 791.8k
Michael Severino	CEO & Director	less than a year	keine Daten	keine Daten
Rachael Potter	Chief Scientific Officer	1.1yrs	keine Daten	keine Daten
Tamara Thornton	Director of Finance	no data	keine Daten	keine Daten
Alison Nasisi	Executive VP & Chief People Officer	4.8yrs	keine Daten	keine Daten
Diane Berry	Executive VP and Chief of Global Policy & Advocacy Officer	6.9yrs	keine Daten	keine Daten
Patrick Moss	Executive VP & Chief Commercial Officer	1.1yrs	keine Daten	keine Daten
James Richardson	Executive VP & Chief Medical Officer	less than a year	keine Daten	keine Daten

Name	Position	Amtszeit	Vergütung	Eigentümerschaft
Michael Severino	CEO & Director	less than a year	keine Daten	keine Daten
M. Behrens Wilsey	Independent Chairwoman	17.4yrs	US$603.92k	0.20% € 3.0m
Hans Wigzell	Chairman of Corporate Strategy Board & Independent Non-Employee Director	16.2yrs	US$562.47k	0.024% € 366.0k
Richard Barry	Independent Director	11.2yrs	US$577.28k	3.05% € 46.7m
Beverly Davidson	Member of Strategic & Scientific Advisory Board	11yrs	keine Daten	keine Daten
Deirdre Connelly	Independent Director	1.9yrs	US$558.15k	0.0032% € 48.3k
Joy Cavagnaro	Member of Scientific Advisory Board	6.8yrs	keine Daten	keine Daten
Louis Kunkel	Member of Strategic & Scientific Advisory Board	11yrs	keine Daten	keine Daten
Claude Nicaise	Independent Non-Employee Director	11.2yrs	US$554.47k	0.030% € 457.5k
Kenneth Fischbeck	Member of Strategic & Scientific Advisory Board	9.4yrs	keine Daten	keine Daten
Carsten Bonnemann	Member of Scientific Advisory Board	6.7yrs	keine Daten	keine Daten
Matthew Wood	Member of Strategic & Scientific Advisory Board	9.4yrs	keine Daten	keine Daten

Daten	Zuletzt aktualisiert (UTC-Zeit)
Unternehmensanalyse	2026/08/09 00:39
Aktienkurs zum Tagesende	2026/08/07 00:00
Gewinne	2026/06/30
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Marktpreise	30 Jahre	Aktienkurse Dividenden, Splits und Aktionen	ICE-Marktdaten SEC-Formular S-1
Eigentümerschaft	10 Jahre	Top-Aktionäre Insiderhandel	SEC-Formular 4 SEC-Formular 13D
Verwaltung	10 Jahre	Das Führungsteam Direktorium	SEC-Formular 10-K SEC-Formular DEF 14A
Wichtige Entwicklungen	10 Jahre	Ankündigungen des Unternehmens	SEC-Formular 8-K

Analyst	Einrichtung
Brian Skorney	Baird
Huidong Wang	Barclays
Eliana Merle	Barclays

Sarepta Therapeutics Management

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