Biogen + Ionis have completed the rolling submission of a New Drug Application (NDA) to the FDA for nusinersen, a treatment for spinal muscular atrophy (SMA).

Biogen and Ionis announced that Biogen has completed the rolling submission of a New Drug Application (NDA) to the FDA for the approval of nusinersen, an investigational treatment for spinal muscular atrophy (SMA). 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 III 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. Biogen anticipates hearing from regulatory authorities regarding the acceptance and validation of these submissions within the next couple of months.

In addition to the NDA filing with FDA, Biogen plans to submit a Marketing Authorization Application (MAA) for nusinersen to the European Medicines Agency (EMA) in the coming weeks. The EMA�s Committee for Medicinal Products for Human Use (CHMP) recently granted Accelerated Assessment to nusinersen, which can reduce the standard review time. Biogen will initiate regulatory filings in other countries in the coming months.