Novartis received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) on its intrathecal gene‑therapy Itvisma (onasemnogene abeparvovec). The opinion recommends marketing authorization for patients with 5q spinal muscular atrophy (SMA) who are two years of age or older, including children, teens and adults with a bi‑allelic SMN1 mutation.
The recommendation is grounded in data from the STEER, STRENGTH and STRONG studies. In the STEER trial, patients treated with Itvisma achieved a 2.39‑point improvement in the Hammersmith Functional Motor Scale (HFMSE) after 52 weeks, compared with a 0.51‑point gain in the sham group, demonstrating sustained motor‑function benefits for the older SMA population.
Strategically, the approval expands Novartis’s rare‑disease portfolio beyond its intravenous Zolgensma therapy, which is limited to infants. Itvisma’s intrathecal, weight‑independent dosing makes it a one‑time treatment for a broader patient group, positioning the company to capture a multibillion‑dollar sales opportunity in Europe. The therapy will compete with established SMA treatments such as Biogen’s Spinraza and Roche’s Evrysdi, but its gene‑therapy approach offers a distinct clinical advantage for older patients.
The CHMP positive opinion follows Novartis’s U.S. FDA approval of Itvisma in November 2025, reinforcing the company’s commitment to rare‑disease gene therapies. The European Commission is expected to issue a final marketing‑authorization decision within approximately two months, after which market uptake will determine the therapy’s commercial success.
The event signals a significant regulatory win for Novartis, potentially accelerating its rare‑disease strategy and providing a new revenue stream that could reshape the company’s long‑term growth trajectory.
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