Biogen and Ionis have taken a crucial step in the pathway to approval of nusinersen. Biogen has completed the submission of a New Drug Application (NDA) to the Food and Drug Administration (FDA) and will submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in the coming weeks. Biogen has also applied for Priority Review which, if granted, would shorten the review period of nusinersen following the Agency’s acceptance of the NDA.
The regulatory submissions are comprised of results from the pre-specified interim analysis of ENDEAR, the controlled Phase 3 study evaluating nusinersen in infantile-onset (most likely to develop Type 1) SMA, as well as all other clinical and preclinical data currently available, which includes open-label data in other patients types. 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 treatment.
The FDA has collaborated closely with Biogen and Ionis throughout the development program, and most recently, allowed a “rolling submission” of the application. This rolling submission provided Biogen and Ionis with the opportunity for increased dialogue with the FDA over the past two months and the ability to make the documents available to the FDA as soon as possible. Additionally, the EMA’s Committee for Medicinal Products for Human Use (CHMP) recently granted Accelerated Assessment to nusinersen, which can reduce the standard review time in the EU. Biogen will initiate regulatory filings in other countries in the coming months.