Renewable Energy

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

Credit: REUTERS/Arnd Wiegmann

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.

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)

((J.Miller@thomsonreuters.com; +41 58 306 7734; Reuters Messaging: j.miller.thomsonreuters.com@reuters.net))

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