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

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 10-3 vote in favor of the drug's benefit/risk profile from the FDA's Oncologic Drugs Advisory Committee (ODAC) on May 24