Sickle Cell Disease Treatment Gets Panel Nod Despite Efficacy Concerns

US FDA's Oncologic Drugs Advisory Committee votes 10-3 in favor of approving Emmaus' L- glutamine, touting its strong safety profile and the unmet clinical need in spite of agency's questions about efficacy results.

The US FDA's Oncologic Drugs Advisory Committee's 10-3 recommendation for the approval of Emmaus Life Sciences Inc.'s sickle cell disease candidate L-glutamine shows that a strong safety profile and unmet clinical need can overcome questionable imputation methods and a modest showing of efficacy.

This is a strong endorsement for the company's product, as FDA had previously put forth several concerns about the clinical data. The sponsor saw a higher than expected dropout rate in the treatment arm of its main Phase III trial, and used an extrapolation method to fill those holes deemed "inappropriate" by the agency

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