- Biogen plans to study whether treatment with its therapy for spinal muscular atrophy, Spinraza, could provide additional benefits to infants and young children who have already received Novartis gene therapy Zolgensma for their disease.
- The clinical trial, which Biogen expects to begin early next year and run for two years, is suggestive of the growing role Zolgensma has as an early treatment for the degenerative neuromuscular condition. Sales numbers released by Novartis on Tuesday show the gene therapy earned $205 million in the months between April and June, even as hospitals in the U.S. struggled to manage rising numbers of COVID-19 cases.
- In the U.S., Zolgensma is approved for infants with SMA who are up to two years of age. Novartis aims to expand that approval into older children, but has been slowed by a regulatory hold on the study meant to support that use. The Swiss drugmaker said Tuesday it hopes to submit an application in 2021.
Spinraza has been widely used since its U.S. approval in 2016 and is cleared to treat both pediatric and adult patients with SMA.
Yet Zolgensma, with its promise of long-lasting benefit, has given families of the 450 to 500 newborns and young infants diagnosed with SMA in the U.S. each year another choice. About 100 patients each quarter are now being treated with Zolgensma, which is designed to correct the genetic defect that leads to the disease.
With its new study, Biogen is looking to test whether those patients who receive Zolgensma early on could still benefit from treatment with Spinraza later in life.
Successive treatment with the two drugs has not been formally studied before, but there have been some instances in which doctors have prescribed the therapies in sequence. Four of 10 patients in a long-term study of Zolgensma later received Spinraza, Biogen said in its statement on the planned trial.
Novartis CEO Vas Narasimhan, asked Tuesday by an analyst about Biogen's study, noted that "in our clinical trial data as well as in the real world, we see patients maintaining the milestones that they gain with Zolgensma."
Biogen aims to enroll 60 patients, including 40 aged 9 months or younger who received Zolgensma treatment before 6 months of age.
The biotech plans to submit its study plan to regulators in the coming months and begin enrolling patients in the first quarter of next year.
Even if Biogen is able to measure some additional benefit, a key hurdle to Spinraza use following Zolgensma would be cost.
Zolgensma is priced at $2.1 million per patient, a cost that is meant to reflect the potential for substantial and long-lasting benefit without further treatment. Spinraza, meanwhile, costs $750,000 in the first year of treatment, and $375,000 thereafter. Stacking the two treatments on top of each other would be an exceptionally expensive approach outside of a clinical trial setting.
Novartis, meanwhile, is hoping to expand use of Zolgensma into older patients, principally through testing of an spinal injection dosing regimen. Study of that intrathecal administration, however, was placed on partial clinical hold by the FDA last year over signs of inflammation observed in a small animal study.
The Swiss pharmaceutical company plans to monitor for one year non-human primates given the intrathecal dose in hopes of allaying the FDA's safety concerns.
Novartis will meet with the FDA this year ahead of a planned application to the agency next year, Narasimhan said Tuesday.