Novartis says it is ready to launch Zolgensma (onasemnogene abeparvovec) within the next few weeks after receiving FDA approval for its spinal muscular atrophy (SMA) single-dose gene therapy.

Zolgensma, a single-dose, one-time gene therapy has been approved to treat children less than two years of age with spinal muscular atrophy (SMA).

“Today’s approval marks another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases,” acting US Food and Drug Administration (FDA) commissioner Ned Sharpless said Friday.

Image: iStock/Michail_Petrov-96

The gene therapy was developed by AveXis, acquired by Novartis in May 2018 for $8.7 billion. Since the acquisition, the Swiss pharma giant has continued investing into and expanding Zolgensma’s manufacturing network and boasts of over one million square-feet of manufacturing space. This is comprised of AveXis plants in Illinois and California, along with investments of $115 million  in Durham, North Carolina, and the acquisition of a 700,000 square-foot production plant from AstraZeneca in Longmont, Colorado.

“We are ready to launch within the next weeks and have ample supply,” a Novartis spokesperson told Bioprocess Insider.

$2.1 million cost

Zolgensma has a wholesale acquisition cost (WAC) of $2.125 million.

This is substantially lower than the $4-5 million estimates made by both management and commentators over the past year, but it still makes Zolgensma the most expensive therapy in the world and a potential burden for healthcare systems and payors.

However, Novartis has laid down plans in partnership with Accredo to offer a pay-over-time option, representing five payments of $425,000.

It also claims the total cost for the patient is half the 10-year cost of “current chronic SMA treatment,” without naming Biogen and Ionis’ oligonucleotide Spinraza (nusinersen), approved in 2016.

“It’s very clear that Novartis has spent extensive time in payor outreach well ahead of this launch – and even issued a separate press release going through payor progress,” said EvercoreISI analyst Umer Raffat said in an investor note.