Biogen Spinraza Approval: The Perfect Antidote To Sarepta Headlines?
Approval of Biogen/Ionis’ Spinraza gives the drug development community a successful, collaborative model for a rapid pathway to full approval for a rare disease therapy; it also provides a timely, compelling rebuttal to claims that US FDA’s drug review operations are in dire need of reform.
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The firm has stumped up $60m for Ionis’s preclinical antisense asset in spinal muscular atrophy, a potential successor to the slowing Spinraza, which also faces patent expiry in a few years.
Expanded access program for rare pediatric disease treatment did not enroll many patients due to clinician community overwhelmed by patient demand and questions over who would pay ancillary drug costs.
Filing supporting Brineura was supported by a tiny, single-arm study and had no advisory committee review, the latest sign of the US regulatory agency's willingness to speed orphan drugs for serious pediatric diseases.