Zolgensma is a gene therapy for spinal muscular atrophy (SMA) that was approved by the US FDA in May 2019, at a price of $2.1 million per patient. Zolgensma was licensed from a children’s hospital where it was developed on NIH grants. Zolgensma’s FDA application was also granted Fast Track, Breakthrough Therapy, Priority Review, and Orphan Drug designations, and also garnered the manufacturer a Rare Pediatric Disease Priority Review Voucher. The company that manufactures Zolgensma, AveXis, was bought by Novartis in 2018.

KEI Pages on Zolgensma:

June 14, 2019. Charity and NIH funding related to Zolgensma

August 2, 2019. Request to Dr. Jerry Mendell to disclose costs of clinical trial for Zolgensma

June 19, 2019. UPenn, Nationwide Children’s Hospital refuse to disclose which patents they licensed to Novartis for Zolgensma

FDA Approval:

May 24, 2019. FDA Press Announcement. “FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality.

FDA Page on Zolgensma (including package insert, approval letter, reviews, and other documents).

Press Links:

May 24, 2019. The New York Times. Katie Thomas. “This New Treatment Could Save the Lives of Babies. But It Costs $2.1 Million.

May 24, 2019. Stat. Adam Feuerstein. “At $2.1 million, newly approved Novartis gene therapy will be world’s most expensive drug.

May 24, 2019. NPR. Rob Stein. “At $2.1 Million, New Gene Therapy Is The Most Expensive Drug Ever.”