Spark Therapeutics’ FDA approved gene therapy, Luxturna, has a price tag – $850,000 (U.S.). Or $425,00 per eye for a rare type of blindness. The therapy is approved for persons with confirmed biallelic RPE65 mutation-associated retinal dystrophy.
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Gene therapy is now a reality. At least in the United States. Today, the U.S. Food and Drug Administration (FDA) approved the gene therapy Luxturna (voretigene neparvovec-rzyl) to treat patients with a rare form of blindness – biallelic RPE65 mutation-associated retinal dystrophy.
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Canadian pharmaceutical company, Prometic Life Sciences Inc. announced they have received an orphan drug designation for Ryplazim (plasminogen) to treat idiopathic pulmonary fibrosis (IP”) by the US Food and Drug Administration (FDA).
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The U.S. Food and Drug Administration (FDA) has approved the asthma drug, Nucala (mepolizumab) to also treat adults with eosinophilic granulomatosis with polyangiitis (also known as Churg-Strauss syndrome), a rare autoimmune disease that inflames blood vessels in the blood.
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