Novartis’ (Avexis) received FDA approval for Zolgensma, the first and only therapy for pediatric patients with spinal muscular atrophy (SMA). The therapy will be made available in the U.S. and marketed by Avexis. In Europe, Zolgensma has prime (priority medicines) designation under accelerated assessment procedure.

TrialSite News has written about Novartis’ pivot to gene therapies and the original estimate of $4 million per treatment for Zolgensma.  The price point is now being set at $2.125, or an annualized cost of $425,000 per annum for five years reports the company.

Zolgensma is a gene therapy designed to treat SMA patients under 2 years of age.  The inherited neuromuscular disease causes progressive loss of muscle function. In the severe cases, the infant dies or relies on permanent breathing support as they approach 2 years old.  A genetic defect, Zolgensma utilizes a re-engineered virus to deliver a copy of the defective gene so that SMN protein can be produced. With  SMA, the defective gene produces SMN, a protein necessary for the survival of motor neurons.

Source: Reuters

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