- The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) adopts a positive opinion recommending approval for Alexion Pharmaceuticals' (NASDAQ:ALXN) Strensiq and Kanuma. A final decision from the European Commission usually takes ~60 days.
- Strensiq (asfotase alfa) is long-term enzyme replacement therapy for patients with pediatric-onset hypophosphatasia (HPP) to treat the bone manifestations of the disease. HPP is an ultra-rare inherited metabolic disease characterized by defective bone mineralization that can lead to their deformity and destruction, profound muscle weakness, seizures, respiratory failure and premature death. Infants who exhibit HPP symptoms with the first six months of life have an overall mortality rate of 73% at five years. It affects ~1 in 100,000 newborns.
- Kanuma (sebelipase alfa) is long-term enzyme replacement therapy for patients with lysosomal acid lipase deficiency (LAL-d), an inherited ultra-rare metabolic disorder in which patients experience chronic lipid accumulation which leads to multi-systemic organ damage and premature death. It affects 1 in 350,000 newborns.
- There are no approved therapies for either disorder.
Europe's CHMP adopts positive opinion supporting approval for two Alexion enzyme replacement therapies
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