ZURICH (Reuters) – Novartis will do a new study of its $2.1 million-per-patient gene therapy Zolgensma on older spinal muscular atrophy patients getting the medicine via a spinal infusion, the Swiss drugmaker said on Wednesday, after the U.S. Food and Drug Administration said it wanted more data.
Novartis said the FDA’s request is not related to an ongoing hold on a clinical trial over safety concerns, and that it also will not affect younger patients with the rare genetic condition for whom the treatment is already approved.
“The FDA has acknowledged the potential of (Zolgensma) in this patient population and recommends a pivotal confirmatory study to supplement” existing data and support filing for approval, Novartis said, adding it “remains confident in the overall benefit-risk profile” for patients.
(Reporting by John Miller; Editing by Michael Shields)
