Amylyx’s Relyvrio: US FDA Review Shows Regulatory Flexibility Can Come In Many Forms
Pink Sheet Drug Review Profile looks at how the agency determined that Amylyx’s ‘conceptual basis’ for use of the drug’s two active ingredients to treat ALS was enough to satisfy the combination drug rule; the sponsor also was allowed to submit certain animal studies and drug-drug interaction data during the course of the NDA review.
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Too Big To Succeed? Cytokinetics Heart Failure Trial Proves Too Much For FDA Committee
US FDA asked a very typical question of its advisory committee about Cytokinetics’ omecamtiv mecarbil: is there ‘substantial evidence’ of efficacy? But in this case the issue wasn’t a trial that was too small or clearly flawed – it was whether such a large, rigorous trial should have shown more than it did.
Accelerated Approval Decline? Too Early To Overinterpret US FDA Trend
The US FDA is likely to end 2022 with noticeably fewer Accelerated Approvals granted than in recent years. But it is far too early to conclude that the agency is backing off the pathway – at least in spirit.
BrainStorm Hoping For Advisory Committee After FDA Refuses To File BLA For Its ALS Cell Therapy
BrainStorm, which has a history of antagonizing FDA with direct appeals to patients for NurOwn, tells investors that public elements of an adcomm could be ‘persuasive,’ but first step will be formal meeting request to try to overcome the RTF. Firm sees Relyvrio as template for success.