Biogen and Ionis Pharmaceuticals announced that SPINRAZA (nusinersen), an investigational treatment for spinal muscular atrophy (SMA), met the primary endpoint at the interim analysis of CHERISH, the Phase 3 study evaluating SPINRAZA in later-onset (consistent with Type 2) SMA. The analysis found that children receiving SPINRAZA experienced a highly statistically significant improvement in motor function compared to those who did not receive treatment. SPINRAZA demonstrated a favorable safety profile in the study.
“These results, along with our successful trial in infantile-onset SMA, reinforce the potential of SPINRAZA to benefit a broad range of SMA patients,” said Michael Ehlers, M.D., Ph.D., executive vice president, head of Research and Development at Biogen. “We will make regulators around the globe aware of this data and will continue working closely with them to bring SPINRAZA to families affected by SMA as quickly as possible.”
Biogen is preparing for the potential launch of SPINRAZA in the U.S. possibly as early as the end of 2016 or the first quarter of 2017.
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 SPINRAZA (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.