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Emflaza's 505(b)(2) Pathway Review: Rare, But With Precedent

 
• By Bridget Silverman

US FDA has relied on 505(b)(2) pathway to approve a small group of 'orphaned orphan' drugs – products without patent protection being used on an informal 'gray market' of personal importation and compassionate use protocols.

The pricing controversy surrounding Marathon Pharmaceuticals LLC's aborted launch of Emflaza (deflazacort) for Duchenne muscular dystrophy has also drawn critical attention to what Sen. Bernie Sanders and Rep. Elijah Cummings call the "unusual circumstances" of the drug's approval by FDA.

But analysis of FDA drug review documents shows that Emflaza's path to approval was not unprecedented. The corticosteroid for Duchenne...

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• By Sue Sutter

The FDA's Oncologic Drugs Advisory Committee will consider whether appropriate dosages of belantamab mafodotin have been identified for two second-line, multiple myeloma indications. Ocular toxicities and large number of dose modifications in trials raised FDA concerns about benefit-risk.

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US FDA To Advisory Committee Industry Reps: Don’t Talk Too Much

 
• By Sue Sutter

Nonvoting industry representative’s presence at Pediatric Advisory Committee meeting was required by statute, the agency said, a de facto acknowledgement that an April directive precluding industry reps from sitting on advisory committees has had little practical effect.