Biogen and Ionis Pharmaceuticals have provided the following community statement regarding the the completion of their NDA filing for nusinersen.
Dear members of the SMA community,
Today we have achieved a crucial step in the pathway to approval of nusinersen. We have completed the submission of our New Drug Application (NDA) to the Food and Drug Administration (FDA) and we will submit our application to the European Medicines Agency (EMA) in the coming weeks.
Once the regulatory agencies receive an application, a validation or review period begins, in which they will review the submission to ensure the application is complete and sufficient to proceed. We are grateful to the FDA for their close collaboration throughout our development program, and most recently, their willingness to work with us on a “rolling submission” of our application. This rolling submission provided us 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. We appreciate both regulatory agencies’ collaboration with us during this important time of application submission and review.
We are also incredibly thankful to the entire SMA community for your continued support. We still have additional milestones to complete before a potential approval and the final approved label, but we are getting closer. A product label will instruct physicians on the use of an approved treatment and the final product label is ultimately decided by each regulatory authority representing a country or region where approval is being sought. For each regulatory submission we are providing all of the data we have to date, which includes data from patients with pre-symptomatic, Type 1, Type 2 and Type 3, and we are seeking a broad label for the treatment of SMA. Again, if approved the final label is decided by the regulatory agencies and is based on their assessment of the data we provide to them. We know there will continue to be many questions such as the timelines of the approval process, the potential label if nusinersen is approved, and about our ongoing plans for expanded access. We remain committed to transparent and timely communications and will continue to provide updates as the program moves forward.
Sincerely,
Biogen and Ionis
Please click here to listen to Biogen’s podcast of frequently asked questions and answers on the Nusinersen program, or download the transcript of that podcast.