U.S. approves Novartis gene therapy for rare, deadly muscle disorder
NEW YORK/ZURICH, May 24 - Swiss drugmaker Novartis NOVN.S 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.
(Reporting by John Miller and Caroline Humer Editing by Michele Gershberg and Bill Berkrot)