Novartis Wins FDA Nod for SMA Gene Therapy in Older Patients
Approval for Itvisma, a new formulation of Zolgensma, positions the one-time treatment to directly challenge chronic therapies from Biogen and Roche.
Novartis AG (NVS) has secured a pivotal U.S. Food and Drug Administration (FDA) approval for Itvisma, its gene replacement therapy for spinal muscular atrophy (SMA), significantly expanding its reach to include older children, teens, and adults. The decision opens a major new front in the multi-billion dollar SMA market, positioning the one-time treatment as a direct competitor to established, chronic therapies from rivals Biogen and Roche.
Shares of Novartis traded near their 52-week high at $126.54 on Monday following the announcement. The Swiss pharmaceutical giant, with a market capitalization of approximately $244 billion, is betting that the convenience of a single dose will appeal to a patient population currently reliant on repeated treatments.
Itvisma uses the same active substance (onasemnogene abeparvovec) as Zolgensma, Novartis’s groundbreaking gene therapy first approved for infants under two. This new approval is for an intrathecal administration, allowing the therapy to be delivered directly to the spinal fluid of patients two years and older. The approval is a “game-changing advance,” according to a statement from Dr. John W. Day, Director of Neuromuscular Medicine at Stanford University School of Medicine, who noted it expands the use of “transformational gene replacement therapy for SMA across age groups.”
The strategic importance of this label expansion cannot be overstated. Until now, the market for older SMA patients has been dominated by two key players: Biogen’s Spinraza (nusinersen) and Roche’s Evrysdi (risdiplam). Biogen’s Spinraza, an antisense oligonucleotide, is administered via spinal injection multiple times per year for life. Roche’s Evrysdi offers the convenience of a daily oral liquid. Both are highly successful products, generating billions in annual revenue by providing continuous treatment.
Novartis’s Itvisma disrupts this model by offering the potential for a one-and-done treatment. “After redefining SMA care with the first gene replacement therapy for this challenging disease, we can now help address unmet needs across an even broader SMA population,” said Victor Bultó, President of Novartis US, in the company's announcement. The therapy offers “the potential to reduce the burden that comes with chronic treatment.”
Spinal muscular atrophy is a progressive and debilitating genetic disease that leads to the loss of motor neurons and subsequent muscle wasting. The approval of a single-dose therapy that can preserve motor function offers a significant new option for patients and physicians.
Patient advocacy groups hailed the decision. “This new route of administration for a single dose of gene replacement therapy can mean so much more than what is measured by numbers on a functional motor scale – it could mean greater independence and freedom in activities of daily life,” commented Kenneth Hobby, President of Cure SMA. “This is another welcome advancement, and it represents real progress in expanding access for many older patients.”
For Novartis, the commercial challenge will now shift to market access and reimbursement. Gene therapies are known for their high upfront costs, often running into the millions of dollars per dose. The company will need to convince payers that the long-term value of a one-time treatment outweighs the recurring, albeit lower, annual costs of its competitors' drugs.
While Wall Street’s consensus analyst price target for NVS stood at $131.28 prior to the news, this approval could prompt a wave of revised forecasts as analysts model the potential market share capture in the lucrative older SMA patient segment. The successful rollout of Itvisma could create a durable, high-margin revenue stream for Novartis and fundamentally alter the standard of care for thousands of SMA patients.