U.S. approves Novartis gene therapy for rare, deadly muscle disorder

by Reuters
Friday, 24 May 2019 17:33 GMT

(Corrects 2nd paragraph to show all types)

NEW YORK/ZURICH, May 24 - 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 all types of 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)

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