Biogen closes in on bringing muscular atrophy preventing drug to market

pharmafile | September 26, 2016 | News story | Manufacturing and Production, Research and Development |  Biogen, EMA, FDA, Nusinersen 

Biogen has applied for Priority Review, which, if granted, would shorten the review period necessary for nusinersen. They also plan to submit a Marketing Authorization Application for nusinersen to the European Medicines Agency (EMA) in the coming weeks, after having recently been granted Accelerated Assessment, which would, again, shorten the review period.

Nusinersen is an investigational, potentially disease-modifying therapy for the treatment of Spinal Muscular Atrophy (SMA). SMA is characterized by the loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy and weakness. Nusinersen is an antisense oligonucleotide (ASO) that is designed to alter the splicing of pre-mRNA from the SMN2 gene in order to increase production of fully functional SMN protein. SMN2 is a gene that is nearly identical to SMN1.

Biogen commented on their application to the FDA by releasing a statement: “We appreciate the FDA’s collaboration with us during the application process, and we look forward to continuing this productive dialogue, with the goal of rapidly bringing the first treatment for SMA to as many patients as possible.”

Ben Hargreaves