Update for November 26th: Two approvals, one delayed decision and only four developmental updates.

The FDA approved emapalumab (Gamifant from Novimmune) on 11/20/18 for the treatment of refractory, recurrent, or progressive primary hemophagocytic lymphohistiocytosis (HLH) or intolerance to conventional HLH therapy. 

The FDA approved glasdegib (Daurismo from Pfizer) on 11/21/18 for the treatment of newly diagnosed acute myeloid leukemia (AML) in patients aged 75 years or older or patients that are not candidates for intensive chemotherapy.

The FDA delayed a decision on Sage Therapeutics’ brexanolone by three months to work on a REMS program for the drug. The FDA did not ask for any additional safety or efficacy data. The new PDUFA Date is May 19, 2019, this drug has Orphan Drug and Breakthrough Priority Designations.

In a Phase III trial, 67% of patients that received Aimmune Therapeutics’ AR101 for peanut allergy were able to tolerate a 600mg dose of peanut protein compared to 4% with placebo and 50% of patients tolerated 1000 mg of peanut protein compared to 2.4% with placebo. 90% of patients were < 18. There was no improvement in peanut protein tolerance in patients > 17. Severe reactions were seen in 4.3% of the AR101 group vs. 0.8% with placebo and 11.6% of AR101 patients withdrew from the trial due to ADR compared to 2.4% with placebo with 14% of AR101 patients requiring epinephrine during the study. 

Novo announced that in a Phase III trial, semaglutidereduced a combined endpoint of cardiovascular death, non-fatal myocardial infarction or non-fatal stroke compared to placebo when added to standard care in patients with type 2 diabetics. Novo announced that in another Phase III trial, daily oral semaglutide HbA1c was lowered by 1.7% with 14mg, 1.5% with 7mg and 1.7% with 3 mg compared to a 1.4% reduction with daily liraglutide 0.9 mg in Japanese type 2 diabetic patients. Only the 14 mg dose was statistically superior to liraglutide.

 Blueprint Medicines announced interim results from an open label Phase I trial where treatment with avapritinibresulted in an 84% objective response rate (ORR) in patients with gastrointestinal stromal tumors and a PDGFRA mutation compared to a 25% ORR in patients without the mutation.

Northwest Biotherapeutics’ DCVax-L is a cancer vaccine in development intended to prevent Glioblastoma multiforme. In a phase I/II trial 33% of patients had reached or exceeded the 4-year survival. Interim results from a 331 patient, Phase III trial suggested a survival benefit in patients treated with DCVax-L. Experts question if a conclusion can be drawn from the trial when completed, because patients were allowed to crossover if there was tumor progression. Almost 90% of patients received the vaccine. Enrollment was completed in 2015, but the trial will remain ongoing until the specified number of events have occurred. At two years, overall survival was 23.1 months with 46.4% of patients still alive after 24 months.