공고 • Dec 21
Calliditas Therapeutics AB Announces Full FDA Approval of TARPEYO
Calliditas Therapeutics AB (publ) announced that the U.S. Food and Drug Administration (FDA) has approved TARPEYO (budesonide) delayed release capsules to reduce the loss of kidney function in adults with primary immunoglobulin A nephropathy (IgAN) at risk for disease progression. TARPEYO was first approved in December 2021 under accelerated approval, based on the surrogate marker of proteinuria. Marking a significant milestone, TARPEYO is now the first fully FDA-approved treatment for IgAN based on a measure of kidney function. The approval is based on data from the Company's Phase 3 NefIgArd clinical trial, a randomized, double-blind, multicenter, study that assessed the efficacy and safety of TARPEYO dosed at 16 mg once daily versus placebo on a background of optimized RASi therapy in adult patients with primary IgAN. TARPEYO was generally well-tolerated in the Phase 3 NefIgArd clinical trial. The most common adverse reactions (5%) in this study were peripheral edema, hypertension, muscle spasms, acne, headache, URT infection, face edema, weight increased, dyspepsia, dermatitis, arthralgia, and white blood cell count increased. TARPEYO is available exclusively through Calliditas specialty pharmacy, Biologics by McKesson. To get started with TARPEYO, prescribers must fill out a TARPEYO Touchpoints Enrollment Form, which serves as a prescription. This Enrollment Form will connect patients with all the benefits provided by TARPEYO Touchpoints, including financial aid programs that can eliminate or reduce out-of-pocket costs, assistance from team of care navigators, pharmacists, and nurse educators; and the convenience of at-home, next-day delivery. At Calliditas, they believe that the cost of treatment should never be a barrier to care. With TARPEYO Touchpoints, 97% of patients taking TARPEYO have paid less than $10 per prescription, and 88% have paid nothing at all. Indication: TARPEYO is indicated to reduce the loss of kidney function in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk for disease progression. Important Safety Information: Contraindications: TARPEYO is contraindicated in patients with hypersensitivity to budesonide or any of the ingredients of TARPEYO. Serious hypersensitivity reactions, including anaphylaxis, have occurred with other budesonide formulations. Warnings and Precautions: Hypercorticism and adrenal axis suppression: When corticosteroids are used chronically, systemic effects such as hypercorticism and adrenal suppression may occur. Corticosteroids can reduce the response of the hypothalamus-pituitary-adrenal (HPA) axis to stress. In situations where patients are subject to surgery or other stress situations, supplementation with a systemic corticosteroid is recommended. When discontinuing therapy or switching between corticosteroids, monitor for signs of adrenal axis suppression. Patients with moderate to severe hepatic impairment (Child-Pugh Class B and C respectively) could be at an increased risk of hypercorticism and adrenal axis suppression due to an increased systemic exposure to oral budesonide. Avoid use in patients with severe hepatic impairment (Child-Pugh Class C). Monitor for increased signs and/or symptoms of hypercorticism in patients with moderate hepatic impairment (Child-Pugh Class B). Risks of immunosuppression: Patients who are on drugs that suppress the immune system are more susceptible to infection than healthy individuals. Chickenpox and measles, for example, can have a more serious or even fatal course in susceptible patients or patients on immunosuppressive doses of corticosteroids. Avoid corticosteroid therapy in patients with active or quiescent tuberculosis infection; untreated fungal, bacterial, systemic viral, or parasitic infections, or ocular herpes simplex. Avoid exposure to active, easily-transmitted infections (e.g., chicken pox, measles). Corticosteroid therapy may decrease the immune response to some vaccines. Other corticosteroid effects: TARPEYO is a systemically available corticosteroid and is expected to cause related adverse reactions. Monitor patients with hypertension, prediabetes, diabetes mellitus, osteoporosis, peptic ulcer, glaucoma or cataracts, or with a family history of diabetes or glaucoma, or with any other condition where corticosteroids may have unwanted effects. Adverse reactions: In clinical studies, the most common adverse reactions with TARPEYO (occurring in 5% of TARPEYO treated patients, and 2% higher than placebo) were peripheral edema (17%), hypertension (12%), muscle spasms (12%), acne (11%), headache (10%), upper respiratory tract infection (8%), face edema (8%), weight increased (7%), dyspepsia (7%), dermatitis (6%), arthralgia (6%), and white blood cell count increased (6%). Drug interactions: Budesonide is a substrate for CYP3A4. Avoid use with potent CYP3A4 inhibitors, such as ketoconazole, itraconazole, ritonavir, indinavir, saquinavir, erythromycin, and cyclosporine. Avoid ingestion of grapefruit juice with TARPEYO. Intake of grapefruit juice, which inhibits CYP3A4 activity, can increase the systemic exposure to budesonide. Use in specific populations: Pregnancy: The available data from published case series, epidemiological studies, and reviews with oral budesonide use in pregnant women have not identified a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. There are risks to the mother and fetus associated with IgAN. Infants exposed to in-utero corticosteroids, including budesonide, are at risk for hypoadrenalism.