FDA Approves Gene Therapy for Spinal Muscular Atrophy

July 23, 2019

Impact: High

• Zolgensma is approved for the treatment of pediatric patients less than 2 years of age with SMA including those who are pre-symptomatic at diagnosis.
• Zolgensma is designed to address the genetic root cause of SMA by replacing the defective or missing SMN1 gene to halt disease progression with a single, one-time infusion.
• Data from the Phase 3 STR1VE trial show prolonged event-free survival, increases in motor function and significant milestone achievement in patients with SMA Type 1, consistent with the Phase 1 START trial.
• In the START trial, patients treated with Zolgensma achieved motor milestones never seen in the natural history of the disease, including sitting, talking and some patients walking, with no waning of effect nearly four years post-dosing.
• AveXis has an exclusive, worldwide license with Nationwide Children’s Hospital to both the intravenous and intrathecal delivery of AAV9 gene therapy for the treatment of all types of SMA; has an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for the in vivo gene therapy treatment of SMA in humans.

inThought Analysis

Zolgensma is both the first gene therapy ever developed to treat a neuromuscular disease, and it’s by far the world’s most expensive drug. Now that Zolgensma is approved, the debate will likely shift towards pricing and availability.

Novartis had previously said it could price the treatment between $1.5 million to $5 million, and has confirmed the treatment will cost $2.125 million — or $425,000 a year spread out over five years. The company said it’s “working closely with insurers to create 5-year agreements based on the success of the treatment as well as other novel pay-over-time options.” It’s currently in “advanced discussions” with more than 15 insurers on payment options.

While SMA is rare, there are more gene therapies to come, and an immutable drug pricing truth is that once one drug sets the pricing level in a category, future drugs will be priced in the same range.

Source: Novartis press release