Global Blood Makes Case For Accelerated Approval Of Sickle Cell Drug Voxelotor
Plan to pursue accelerated approval carries risk that US FDA will ask for more data, which could significantly delay the launch, some analysts warn.
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Company revealed that US FDA agreed to accelerated approval pathway for voxelotor in sickle cell disease, with focus on stroke prevention. Updated data from a key trial was also released at ASH.
With an eye on a patient population mainly covered by Medicare and Medicaid, Emmaus Life Sciences CEO Yutaka Niihara says the company wants to ensure its pharmaceutical grade L-glutamine product is accessible in the US.
Part 2: High costs are built into the rare disease business model. Participants at a roundtable discussion led by Scrip at the J.P. Morgan Healthcare Conference talked about how much they are feeling the pressure on pricing.