Blueprint Medicines’ Ayvakit (avapritinib) is headed to the FDA for a new indication after the drug hit the mark in Part 2 of the PIONEER trial for non-advanced systemic mastocytosis.
Although the treatment was once seen to have strong commercial potential in breast cancer, this second trial failure deals a major blow to the French healthcare company’s development prospects.
In what has been a long time coming, Tel Aviv and New York-based BrainStorm announced Monday that it would submit a Biologics License Application (BLA) to the FDA for NurOwn as a potential treatment for ALS. Simultaneously, BrainStorm announced corrected analyses it says strengthen its original conclusions.
MaaT Pharma announced that the U.S. FDA has maintained the clinical hold on MaaT013, its candidate for patients with steroid-resistant acute graft-versus-host disease.
The company’s original plan was to not apply to regulators until 2023, but now, it plans to submit the drug to the FDA in the next few months, with plans to launch the drug in mid-2023.
One of the first trials aimed at tackling long COVID helped some patients recover from lingering physical and mental fatigue, although the drug developed by Axcella Health Inc failed on the small study’s main goal of restoring the normal function of mitochondria – the energy factories of cells.
Laboratory Corp of America Holdings said it would spin off its wholly owned business focused on clinical drug trials. The planned spin-off will result in two independent, publicly traded companies – Labcorp and the clinical development business.