Related coverage linked through entity extraction aliases.
Jul 29, 2026
FDA granted rolling review for Palvella's QTORIN rapamycin NDA. Also covered: global expedited approval pathways, accelerated approval scrutiny, and multi-regional CDx strategies.
Mar 26, 2026
Quoin Pharmaceuticals has secured Orphan Drug Designations for QRX003 in the U.S. and Europe for Netherton Syndrome, while advancing regulatory pathways in Japan and Saudi Arabia. The company reported clinical progress including expansion of pediatric studies and potential for a Priority Review Voucher worth up to $200 million.
Mar 25, 2026
Interim results from the ABC phase 1/2b trial show a calcineurin inhibitor-free GVHD prevention regimen combining posttransplant cyclophosphamide, bortezomib, and abatacept achieved low GVHD rates and favorable survival outcomes. The approach enables earlier introduction of posttransplant maintenance therapies while reducing toxicity associated with conventional immunosuppressants.
Feb 28, 2026
Researchers develop multiple strategies to enhance CAR T-cell therapy, including dual PGE2 receptor knockout for solid tumors and small-molecule switches using venetoclax and caffeine to control cellular activity and improve safety.
Feb 25, 2026
Palvella Therapeutics announced positive topline results from its Phase 3 SELVA study of QTORIN 3.9% rapamycin for microcystic lymphatic malformations, meeting all primary and secondary endpoints with strong tolerability. The company plans an NDA submission in H2 2026.
