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Emmaus Sickle Cell NDA Facing FDA Concerns About Clinical Meaningfulness

 
• By Bridget Silverman

PGLG's application only has one Phase III trial, and Emmaus is hoping statistical re-analyses can address FDA skepticism about clinical meaningfulness of the primary endpoint data.

Emmaus Life Sciences Inc. is counting on new statistical analyses and secondary endpoints to resolve FDA's concerns about the single Phase III trial submitted with the new drug application for the company's pharmaceutical-grade L-glutamine (PGLG) to treat sickle cell disease.

The product could be a landmark in a disease with significant unmet need – Emmaus emphasizes that PGLG would be the first new SCD treatment in 20 years, and the first ever approved for pediatric patients – but the company's descriptions of

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