FDA will convene adcomm for Bristol-Myers' Reblozyl in MDS; FDA grants 'orphan' tags to a trio of drugs
→ Celgene and Acceleron‘s Reblozyl (luspatercept) earned an OK from the FDA last month to treat anemia in adults who require regular transfusions, but still had a pending review with regulators about using the drug for myelodysplastic syndromes (MDS). Now, Bristol-Myers — which finally completed its $74 billion takeover of Celgene — and Acceleron said the FDA’s Oncologic Drugs Advisory Committee will meet later this month to discuss their supplemental biologics license application to to cover the treatment of adult patients with very low- to intermediate-risk MDS-associated anemia who have ring sideroblasts and require red blood cell (RBC) transfusions. The PDUFA date is set for April 4, 2020.
→ David Meeker’s KSQ Therapeutics — which earned a hefty $80 million from a financing round last year — has enlisted cell engineering service provider MaxCyte to help develop adoptive cell therapies for PD-1 refractory solid tumors. Under the terms of agreement, KSQ will be using MaxCyte’s tech to advance their engineered tumor-infiltrating lymphocyte (eTIL) programs.
→ The FDA has granted Swiss-based APR Applied Pharma Research (APR) orphan drug designation for its investigational drug, APR-TD011 — a hypotonic acid-oxidizing solution containing hypochlorous acid in a sprayable form — for the treatment of epidermolysis bullosa (EB).
→ Tetra Bio-Pharma, a cannabinoid-derived drug discovery and development company, has received orphan drug designation from the FDA for delta-9-tetrahydrocannabinol (THC) in the treatment of hepatocellular carcinoma.
→ Adastra Pharmaceuticals‘ zotiraciclib has been designated by the FDA as an orphan drug for glioma. The drug is currently undergoing two separate Phase Ib clinical trials in glioblastoma and the company expects results for both in 2020.