Key Takeaways:
Novartis AG (NVS) received a positive opinion from the European Medicines Agency’s (EMA) key committee for its gene therapy Itvisma, recommending its approval for treating a broad range of spinal muscular atrophy (SMA) patients. The decision paves the way for a new treatment in a market projected to reach $7.34 billion by 2030.
The Committee for Medicinal Products for Human Use (CHMP) backed Itvisma for children aged two and older, teenagers, and adults with 5q spinal muscular atrophy. The recommendation is based on a Phase III study showing a statistically significant 2.39-point improvement in motor function, offering a potential one-time alternative to chronic therapies.
"Even a 1-point difference in the HFSME can translate into tangible functional gains for individuals with SMA, such as the ability to grasp a pen," Professor Tim Hagenacker, from the Department of Neurology at University Hospital Essen, said. "Preserving existing capabilities is critical, as maintaining independence and autonomy is a central goal of care for patients living with a progressive neuromuscular disease.”
The positive opinion stems from the registrational STEER study, where 75 patients receiving Itvisma showed a 2.39-point improvement on the Hammersmith Functional Motor Scale (HFMSE) compared to a 0.51-point gain in the 51-patient sham group. The result was statistically significant with a p-value of 0.0074, and the effects were sustained over 52 weeks. The recommendation was also supported by data from the STRENGTH and STRONG studies.
A final decision on marketing authorization from the European Commission is expected within the next two months. If approved, Itvisma will become the first gene replacement therapy in the EU for SMA patients two years and older. Itvisma is a one-time intrathecal injection designed to replace the missing or defective SMN1 gene, the root cause of the disease.
This positions Novartis to compete more broadly with existing SMA treatments from Biogen Inc. and Roche Holding AG. The company already markets Zolgensma, a gene therapy for infants and young children with SMA. The expansion into an older patient population with Itvisma could significantly increase Novartis's footprint in the SMA market, which is growing at a compound annual rate of 12.9 percent, according to data from The Business Research Company.
"A positive CHMP opinion for Itvisma is an important step toward addressing this gap," Nicole Gusset, CEO of patient advocacy group SMA Europe, said, highlighting the limited innovative therapies for older children and adults.
The approval would solidify Novartis’s position in the gene therapy space and provide a significant new revenue stream. For patients, it offers the potential for a one-time treatment to replace the burden of chronic, regularly administered therapies. Investors will now watch for the final European Commission decision in approximately two months, which will be the key catalyst for market access in the EU.
This article is for informational purposes only and does not constitute investment advice.
