SMA treatment Itvisma gets European regulatory panel OK
CHMP positive opinion of gene therapy follows successful trials
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A European Medicines Agency (EMA) panel recommended that the one-time gene therapy Itvisma (onasemnogene abeparvovec-brve) be approved for patients with spinal muscular atrophy (SMA) ages 2 and older.
The Committee for Medicinal Products for Human Use (CHMP) opinion will be reviewed by the European Commission, which has final say over therapy approvals in Europe. The commission doesn’t have to follow the CHMP’s recommendation, but it almost always does. Novartis, the company developing Itvisma, said it expects a final decision from the commission in about two months.
“Today’s positive CHMP opinion is a significant step towards potentially reducing the long-term burden of chronic treatment administration for patients living with SMA in Europe,” Patrick Horber, MD, president of the international division at Novartis, said in a company press release.
SMA is caused by mutations in the SMN1 gene. Lacking a healthy version of this gene, motor neurons (the nerve cells that control movement) sicken and die, resulting in SMA symptoms such as progressive muscle weakness and wasting.
Itvisma is a gene therapy designed to deliver a working version of the SMN1 gene to motor neurons, addressing the root cause of SMA to slow the disease’s progression. The therapy contains the same construct as Zolgensma (onasemnogene abeparvovec-xioi), another gene therapy from Novartis that’s widely approved to treat infants and toddlers with SMA. A key difference between the two gene therapies is that Itvisma is given by injection into the spinal canal at a fixed dose, while Zolgensma is infused into the bloodstream at a dose adjusted by weight.
Expanding options
Novartis has been developing Itvisma with the goal of making one-time gene therapy an option for all people with SMA, regardless of age. Itvisma was approved in the U.S. a few months ago, a move advocates heralded as ushering in a new era of therapeutic choice.
“Building on the established role of Zolgensma for babies and young children with SMA, [the positive CHMP] opinion for Itvisma reflects our ambition to expand treatment options for a broader patient population in SMA,” Horber said. “It also underscores Novartis’ commitment to pushing scientific boundaries to address unmet needs and improve long-term outcomes across the SMA and rare disease community.”
Nicole Gusset, CEO of SMA Europe, said, “Living with SMA affects every stage of life, including education, employment, and independence. Older children, teenagers, and adults face fewer opportunities to benefit from innovative therapies. A positive CHMP opinion for Itvisma is an important step toward addressing this gap.”
Novartis’ application seeking approval of Itvisma in Europe was based mainly on data from STEER (NCT05089656), a Phase 3 clinical trial that tested the gene therapy against a sham procedure in children and teenagers with SMA who had not previously received other treatments. The trial met its goal, showing that Itvisma significantly improved motor function relative to the sham. Long-term data showed that patients treated with Itvisma showed sustained improvements in motor function.
“Itvisma met the primary endpoint showing motor function improvement versus placebo,” said Tim Hagenacker, MD, a neurologist at University Hospital Essen in Germany. “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 application also includes data from a study called STRENGTH (NCT05386680) in which young people with SMA who had previously been on other SMA treatments switched to one-time Itvisma treatment. Results showed that motor function remained stable after the switch, and some participants achieved new motor milestones following treatment.
The CHMP’s recommendation applies to 5q SMA, the main form of the disease.
