Nusinersen for Spinal Muscular Atrophy

Nusinersen, one of several recently approved drugs intended for use in a rare disease, boasts an eye-popping price tag. Biogen Pharmaceuticals announced that nusinersen will cost $750  000 for the first year of treatment and $375 000 each year thereafter (prescribed indefinitely) for patients with spinal muscular atrophy (SMA). Other recently approved costly therapies for rare diseases include ivacaftor, costing $311 000 per year, for responsive patients with cystic fibrosi s; combination lumacaftor-ivacaftor, costing $272 000 per year, for patients with cystic fibrosis; eteplirsen, costing $300 000 per year, for a 25-kg child with Duchenne muscular dystrophy; and asfotase alfa, costing $285 000 per year, for patients with infantile and juvenile-onset hypophospha tasia. Many more such drugs are poised to enter the market, particularly in fields such as cancer medicine.
Source: JAMA Pediatrics - Category: Pediatrics Source Type: research