Sickle Cell Disease Orphan Status Lures Pharma Interest
As interest in drugs with orphan indications picks up and the science advances, sickle cell disease is attracting the attention of big pharma, with deals inked for two promising therapeutics and a variety of approaches in the clinic with partnership potential.
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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.
Global Blood Therapeutic Inc.'s homegrown lead candidate is an oral, once-daily drug aimed at sickle cell disease, a genetic condition that causes red blood cells to become deformed or “sickled." The goal with GBT440 is to correct the disease, so that patients would actually have no evidence of it following treatment.
With enrollment finally ramping up in trial open to older patients, Mast Therapeutics envisions a broad labeling horizon for MST-188, after a very long development road.