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Sickle Cell Innovators Required: Emmaus' Pending Approval Highlights Rare Disease Void

25 May 2017

FDA approval is likely for Emmaus' sickle cell drug L-glutamine this summer, representing the first new product for the disease since 1998 – a decision FDA advisory committee panelists hope will spur more innovation.

Sickel cell anemia — First sickle cell drug in almost two decades is set for US approval this summer • Source: Shutterstock

As potentially the first new drug to approach the sickle cell disease market in nearly 20 years and with a limited late-stage pipeline, reception this summer for Emmaus Life Sciences Inc.’s L-glutamine is likely to be strong despite the drug showing only modest benefit in a Phase III trial.

Despite quibbles over the efficacy analysis of the Phase III data, L-glutamine looks set for a US approval following a...