Novartis wins U.S. approval for gene therapy for spinal muscular atrophy

Swiss drugmaker Novartis won U.S. approval for its gene therapy Zolgensma for spinal muscular atrophy (SMA), the leading genetic cause of death in infants, the company said on Friday, a one-time treatment expected to push the pricing boundary for rare diseases to new heights.

The company said the drug was approved for babies less than 2 years of age with Type 1 SMA. The company said the wholesale acquisition cost of the therapy is $2.125 million. 

(With inputs from agencies.)