SMA gene therapy now approved in Europe to treat patients of all ages
Itvisma 'can make a meaningful difference' for teens, adults, doctor says
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- Itvisma, a one-time gene therapy for SMA, is now EU-approved for patients ages 2 and older.
- The expanded approval in Europe gives gene therapy access to older children, teens, and adults with SMA, complementing existing treatments.
- Administered via spinal injection, Itvisma was shown to significantly improve or maintain motor function in trials.
The one-time gene therapy Itvisma (onasemnogene abeparvovec-brve) is now approved in the European Union for people with spinal muscular atrophy (SMA) ages 2 and older — a regulatory decision that will make the injection therapy available to SMA patients of all ages.
According to therapy developer Novartis, this expanded approval, which comes on the heels of a positive recommendation from an arm of the European Medicines Agency (EMA), makes Itvisma the first and only gene therapy approved for this broad population in the EU. The approval, applicable in all EU member nations, comes less than a year after Itvisma was approved for the same indication in the U.S.
“This approval marks a major milestone for people living with SMA,” Patrick Horber, MD, president, international at Novartis, said in a company press release announcing the decision.
“With Itvisma, we are going further to expand access to a one-time gene replacement therapy for older children, teens and adults – potentially addressing long-standing unmet needs for patients,” Horber said.
Zolgensma (onasemnogene abeparvovec-xioi), an older gene therapy from Novartis, was already approved in Europe to treat babies and young children — weighing up to 21 kg (about 46 pounds) — with SMA. The approval of Itvisma means that a one-time gene therapy will now be available to people of all ages with the rare genetic disease.
“Together with Zolgensma, we can now offer gene replacement therapy options across different stages of SMA in Europe, from newborns to adults,” Horber said.
Nicole Gusset, CEO of SMA Europe, a nonprofit umbrella organization, said the EU approval of Itvisma “is an important milestone for the SMA community.”
“Beyond the scientific achievement, it brings the prospect of a new treatment option closer to people and families who are looking for choices that reflect their individual needs and circumstances,” Gusset said. “We welcome today’s decision and hope it translates into timely and equitable access across Europe.”
New gene therapy approval may give options to older SMA patients
SMA is chiefly caused by mutations in the SMN1 gene, which lead to the death and degeneration of nerve cells that control movement. Both Itvisma and Zolgensma use the same viral construct to deliver a healthy version of this gene to these nerve cells.
The key difference is in how the treatments are administered: Zolgensma is given intravenously, by infusion into the bloodstream at a dose determined by a patient’s weight, whereas Itvisma is injected directly into the spinal canal at a fixed dose, called an intrathecal injection.
EU approval of Itvisma was mainly based on data from a Phase 3 clinical trial called STEER (NCT05089656), which tested the gene therapy versus a sham procedure in children and teenagers with SMA who had not previously received other SMA treatments.
The results showed that Itvisma significantly improved motor function compared with the sham procedure, and long-term data indicated that patients given the therapy experienced sustained gains in motor function.
The approval of Itvisma in Europe is an important advance because it brings a new gene replacement therapy option to a broader patient population.
Novartis’ application to the EU also included data from another Phase 3 study, STRENGTH (NCT05386680), in which patients who had previously been on other SMA treatments switched to Itvisma. Those results showed that motor function remained generally stable after the switch, with some individuals achieving new motor milestones after gene therapy.
“Maintaining or improving motor function can make a meaningful difference for older children, teens and adults living with SMA,” said Jana Haberlová, MD, PhD, of Motol and Homolka University Hospital in Czechia. “The approval of Itvisma in Europe is an important advance because it brings a new gene replacement therapy option to a broader patient population and gives clinicians an additional way to support patients across the course of the disease.”
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