Novartis reports positive Phase III results for SMA gene therapy OAV101 IT

19 March 2025

Swiss pharma major Novartis (NOVN: VX) has announced positive results from its Phase III STEER study, evaluating the efficacy and safety of intrathecal onasemnogene abeparvovec (OAV101 IT) in patients aged 2 to under 18 with spinal muscular atrophy (SMA) Type 2.

The study demonstrated a statistically-significant improvement in motor function among participants. Patients receiving OAV101 IT showed a 2.39-point increase on the Hammersmith Functional Motor Scale Expanded (HFMSE), compared to a 0.51-point improvement in the sham control group.

The HFMSE is a standard measure for assessing motor abilities and disease progression in SMA patients.

This article is accessible to registered users, to continue reading please register for free. A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here.

Try before you buy

Free

7 day trial access

Take a Free Trial

All the news that moves the needle in pharma and biotech
Exclusive features, podcasts, interviews, data analyses and commentary from our global network of life sciences reporters.
Receive The Pharma Letter daily news bulletin, free forever.

Become a subscriber

£820

Or £77 per month

Subscribe Now

Unfettered access to industry-leading news, commentary and analysis in pharma and biotech.
Updates from clinical trials, conferences, M&A, licensing, financing, regulation, patents & legal, executive appointments, commercial strategy and financial results.
Daily roundup of key events in pharma and biotech.
Monthly in-depth briefings on Boardroom appointments and M&A news.
Choose from a cost-effective annual package or a flexible monthly subscription

The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It’s part of the key information for keeping me informed

Chairman, Sanofi Aventis UK