Biogen’s NDA for Nusinersen Accepted by FDA
Biogen today announced that its New Drug Application (NDA) for nusinersen, an investigational treatment for spinal muscular atrophy (SMA), has been accepted by the U.S. Food and Drug Administration (FDA) for Priority Review, and that the company’s Marketing Authorization Application (MAA) has been validated by the European Medicines Agency (EMA). Nusinersen had previously been granted Accelerated Assessment status by the EMA’s Committee for Medicinal Products for Human Use (CHMP). The regulatory review process for these applications has now been initiated in the U.S. and EU. Both the Priority Review and Accelerated Assessment designations can reduce the standard review time. If approved, nusinersen would be the first therapy for SMA, a leading genetic cause of infant mortality.
Starting in 2003, Cure SMA provided the seed funding needed to begin investigation into this therapeutic approach. The intellectual property generated with our funding was then licensed to Ionis Pharmaceuticals to create nusinersen. Biogen intends to market nusinersen under the brand name SPINRAZATM. This name has been conditionally accepted by the FDA and the CHMP and will be confirmed upon approval.
“The FDA and EMA have acknowledged the potential for nusinersen to address the urgent need for an effective SMA treatment by granting special status to the applications, and FDA has shared that they plan to act early on our NDA under an expedited review,” said Michael Ehlers, M.D., Ph.D., executive vice president, head of Research and Development at Biogen. “We are now focused on working with the agencies to hopefully bring this investigational treatment to the SMA community as quickly as possible.”
The regulatory filing packages in the U.S. and EU are based on data that demonstrate the clinically meaningful efficacy and favorable safety profile of nusinersen from multiple studies. These include the results from the interim analysis of ENDEAR, the Phase 3 study evaluating nusinersen in infantile-onset (most likely to develop Type 1) SMA, as well as open-label data in other patient populations. The ENDEAR interim analysis demonstrated that infants receiving nusinersen experienced a statistically significant improvement in the achievement of motor milestones compared to those who did not receive nusinersen. Data from the other endpoints analyzed were also consistently in favor of the treated infants. Nusinersen was generally well-tolerated, with a favorable safety profile. No adverse events (AEs) were considered related to nusinersen.
Leading the Way on Regulatory Issues
When the FDA evaluates a drug for approval, they must weigh many different factors, including the quantity and quality of evidence of safety and effectiveness, potential benefits of a treatment versus the potential risks, and the impact the treatment will have on the patient community. While we wait for the NDA to be reviewed, we won’t sit back. We are actively working to continue our ongoing efforts to make sure that our community’s voice is heard during this entire process.
This includes our upcoming Patient-Focused Drug Development Meeting with the FDA, newborn screening initiatives, coverage and payment projects, and many more initiatives designed to make certain that regulators, payers, and industry partners understand the impact of SMA, and create solutions that will address the areas most important to us.
For More Information
For more information on how Cure SMA is working on the clinical and regulatory issues that are important to our community, please see these recent articles from our news section.
- 6th Annual "Hope on the Hill" Congressional Dinner
- Patient-Focused Drug Development Meeting
- Cure SMA Announces $2.5 Million in New Planned Research Funding
- Cure SMA Posts Keynote Presentations on the NDA Process
- 2016 Update on SMA-FDA Interactions
This is an historic moment that many in our community have worked for tirelessly. We extend our deepest gratitude to all our families, supporters, donors, and partners who have contributed to this milestone.
Cure SMA would like to thank and acknowledge Cold Spring Harbor Laboratory (CSHL) and the University of Massachusetts Medical School for generating critical intellectual property for the program that was licensed to Ionis Pharmaceuticals. We specifically thank Dr. Adrian Krainer and his colleagues at CSHL for years of dedication to and hard work on the preclinical development of nusinersen for SMA, and Drs. Ravindra Singh and Elliot Androphy for their work funded by Cure SMA in originally identifying the ISSN1 gene sequence, which is the sequence targeted in nusinersen. In addition, Drs. Krainer and Androphy are long standing members of the Cure SMA Scientific Advisory Board.