Babies born with a severe form of a rare genetic condition known as spinal muscular atrophy almost always die before their second birthday. A gene therapy approved Fridayby the Food and Drug Administration promises to change that, offering a potential one-time fix for the genetic deficiency behind the neuromuscular disease.
Zolgensma, as the treatment is called, is only the second gene therapy for an inherited disorder to be cleared for commercial use in the U.S. Its keenly awaited approval marks another milestone for a fast-moving field, and validates the decision by Novartis last year to acquire its developer, the biotech company AveXis, for $8.7 billion.
The therapy is also notable for its price. At $2.125 million per dose, Zolgensma is the most expensive treatment ever brought to market. Novartis intends for that price to be paid in installments of $425,000 a year for five years.
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