The Food and Drug Administration on Friday approved the first gene therapy for a type of spinal muscular atrophy, an inherited neuromuscular disease that causes progressive loss of muscle function.
The most severe form of SMA causes infants to die or rely on permanent breathing support by the age of 2. Zolgensma uses a re-engineered virus to deliver a functional copy of the defective gene.It’s a one-time injection. In its pivotal clinical trial and an ongoing clinical trial, a majority of the infants and young children injected once with Zolgensma remained alive, could breathe on their own, and showed improvements in motor milestones. But the new drug, will also be the most expensive drug in the world. Novartis is pricing Zolgensma at $2.125 million, or an annualized cost of $425,000 per year for five years.
Company executives said that financial assistance will be available. Complex payment and insurance reimbursement arrangements required for expensive gene therapies need to be handled. The terapy will be available within two weeks. In 1983 Congress passed the Orphan Drug Act, a well-meaning law designed to encourage research into rare diseases. It offered drug makers tax breaks and other incentives for such work. Orphan treatments rarely face the competitive pressures that check the prices of medicines for common conditions.