FDA’s NME approvals in September
Agency approved two new cancer and one new neuro drug; plus Amicus’ Pompe therapy among pair of approvals for rare metabolic disorders
The FDA approved five new molecular entities in September: two for oncology indications, two for rare metabolic diseases, and one for major depressive disorder.
The $1.9 billion takeout of Sierra Oncology Inc. by GSK plc (LSE:GSK; NYSE:GSK) produced an FDA approval in September, with the JAK-1, JAK-2 and ALK2 inhibitor Ojjaara momelotinib receiving authorization to treat myelofibrosis patients with anemia. In Phase III MOMENTUM trial, which read out 11 weeks before the acquisition, Ojjaara met the primary endpoint measuring total symptom scores and showed statistical significance in secondary endpoints evaluating transfusion independence and splenic response rate. Sierra had gained rights to the drug from Gilead Sciences Inc. (NASDAQ:GILD), which is eligible for royalties...