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Emmaus Needs US FDA Advisory Cmte. To Rescue Sickle Cell Disease Candidate

 
• By Michael Cipriano

FDA reviewers disagree with Emmaus' extrapolation methods for holes in efficacy data for its L-glutamine ahead of May 24 Oncologic Drugs Advisory Committee meeting.

Emmaus Life Sciences Inc. will likely need the US FDA's Oncologic Drugs Advisory Committee to save the day for the company's sickle cell disease candidate L-glutamine, as the agency itself appears deeply skeptical of the Emmaus' method for extrapolating efficacy data to substitute for a high discontinuation rate.

The Torrance, Calif.-based drugmaker saw a higher than expected dropout rate in the treatment arm of its main Phase III trial, and used an imputation method deemed by FDA as "inappropriate" to fill in the missing data holes, according to FDA's briefing documents released in advance of the May 24 meeting

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