Global Blood Therapeutics Inc.
Stopping the cycle of sickle cell disease
This article was originally published in Start Up
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.
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GBT440 is not efficacious enough in studies of idiopathic pulmonary fibrosis, but the drug was well-tolerated at twice the exposure in the treatment of sickle cell disease, the drug's lead indication.
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.
Part 2: High costs are built into the rare disease business model. Participants at a roundtable discussion led by Scrip at the J.P. Morgan Healthcare Conference talked about how much they are feeling the pressure on pricing.