Global Blood's sickle cell therapy heading for NDA

Global Blood Therapeutics Inc. (NASDAQ:GBT) added almost $800 million in market cap on Monday after it said FDA agreed with a proposed regulatory path by which the company could obtain accelerated approval of voxelotor (formerly GBT-440) to treat sickle cell disease. Global Blood hopes to meet with FDA next quarter; a submission timeline would follow.

New and prior data from the Phase III HOPE trial have shown that voxelotor led to significant improvements in hemoglobin levels among patients with sickle cell disease. On a conference call Monday, Global Blood President and CEO Ted Love said FDA agreed that by increasing hemoglobin, voxelotor is reasonably likely to lead to clinical benefit in sickle cell patients, including a reduced risk of stroke, and would thus meet the standard for the therapy's evaluation under the accelerated approval pathway...