Novartis Expands SMA Treatment Options with FDA Approval of Itvisma Gene Therapy

Novartis has secured a significant victory in the treatment of spinal muscular atrophy (SMA) with the U.S. Food and Drug Administration's approval of Itvisma, an intrathecal version of its gene therapy Zolgensma. This groundbreaking development expands treatment options for older SMA patients, marking a pivotal moment in the field of genetic medicine.

Broadening Access to Gene Therapy

Itvisma, formerly known as onasemnogene abeparvovec-brve or OAV101 IT, is now approved for children two years and older, teens, and adults with SMA who have a confirmed mutation in the survival motor neuron 1 (SMN1) gene. This approval significantly broadens the patient population that can benefit from gene replacement therapy, as the original Zolgensma was limited to children under two years of age.

The new formulation addresses the genetic root cause of SMA by providing a functional copy of the SMN1 gene, which is absent in these patients. John Day, professor at Stanford University School of Medicine, called the approval a "game-changing advance" that "signals new possibilities for the broader field of neurological disorders and genetic medicine."

Clinical Efficacy and Safety Profile

The FDA's decision was based on data from the Phase III STEER study and supported by the Phase IIIb STRENGTH study. These trials demonstrated statistically significant improvements in motor function and stabilization of motor abilities not typically observed in the natural progression of the disease. The effects were sustained over one year of follow-up.

In the STEER study, patients receiving Itvisma showed an average improvement of 2.39 points on the Hammersmith Functional Motor Scale Expanded (HFMSE), compared to a 0.51-point improvement in the control group. The STRENGTH trial further validated the therapy's efficacy in patients who had discontinued treatment with other SMA drugs.

Adverse events were consistent across both studies, with the most common being upper respiratory tract infections, fever, and vomiting.

Pricing and Market Implications

Novartis has set the wholesale acquisition cost of Itvisma at $2.59 million, positioning it as a cost-effective option compared to existing chronic treatments. The company claims that over a 10-year span, Itvisma costs 35% to 46% less than current alternatives. Novartis is also offering a patient support program to enhance accessibility.

With approximately 9,000 people living with SMA in the U.S., the majority being older children, teens, and adults, Novartis projects multibillion-dollar peak sales for Itvisma. This approval solidifies Novartis's position in the SMA market, where Zolgensma has already treated over 5,000 patients globally and generated $1.2 billion in sales last year.

The SMA treatment landscape now includes multiple options, with Biogen's Spinraza and Roche's Evrysdi also available. Biogen is developing a higher-dose version of Spinraza, with regulatory decisions expected in Europe and the U.S. in the near future.

As the pharmaceutical industry continues to advance in genetic medicine, Novartis's success with Itvisma represents a significant milestone in expanding treatment options for patients with SMA and potentially paving the way for similar approaches in other neurological disorders.