Becoming the first type II RAF inhibitor for relapsed or refractory BRAF-altered pediatric low-grade glioma, Day One Biopharmaceuticals Inc.’s Ojemda (tovorafenib, DAY-101) gained U.S. FDA accelerated approval on April 23, a week earlier than its expected PDUFA date, bringing the Brisbane, Calif.-based company a rare pediatric disease priority review voucher.
About a year after the U.S. FDA cleared Krystal Biotech Inc.’s topical gene therapy, Vyjuvek (beremagene geperpavec), as the first drug for recessive or dominant dystrophic epidermolysis bullosa, competitor Abeona Therapeutics Inc. disclosed a complete response letter (CRL) from the FDA related to its cell-based graft therapy, prademagene zamikeracel.
After several delays, the interleukin-15 superagonist Anktiva (nogapendekin alfa inbakicept) from Immunitybio Inc. has been approved by the U.S. FDA for treating bladder cancer, specifically Bacillus Calmette-Guérin-unresponsive non-muscle-invasive bladder cancer with carcinoma in situ, with or without papillary tumors.
Lumicell Inc. sees a brighter future now that it has secured U.S. FDA approval for both its new drug application for Lumisight (pegulicianine) optical imaging agent and premarket approval for the Lumicell direct visualization system.
Genfleet Therapeutics Inc. has entered the KRAS G12C inhibitor race in the U.S. as it gears up to begin phase III trials of GFH-925 (IBI-351) in patients with refractory metastatic colorectal cancer (CRC) following FDA clearance. GFH-925 was the first KRAS G12C inhibitor to receive breakthrough therapy designation from China's National Medical Products Administration for previously treated advanced CRC.
In March, the U.S. FDA approved 30 new drugs, marking the highest monthly count in BioWorld’s records. The previous highest month of June 2020, with 29 FDA approvals, is followed by November 2017’s 27 approvals. In 2023, the FDA greenlit an average of about 16 drugs per month, 12.5 in 2022, and 17 in both 2021 and 2020. The surge in March marks a 173% increase from the 11 drugs approved in February.
Having addressed the manufacturing issues that resulted in a few complete response letters, Alvotech Holdings SA and Teva Pharmaceutical Industries Ltd.’s biosimilars partnership is now on a roll, with the U.S. FDA approving the team’s second biosimilar, Selarsdi, less than two months after approving the first one, Simlandi, as an adalimumab interchangeable.
The U.S. FDA granted fast track designation to Telix Pharmaceutical Ltd.’s TLX101-CDx for glioma imaging as the firm prepares to file its NDA in the first half of 2024, a Telix spokesperson told BioWorld.
To take clinical trial innovation to the next level, the U.S. FDA’s Center for Drug Evaluation and Research (CDER) is opening the CDER Center for Clinical Trial Innovation. The center, known as C3TI, “will be a central hub within CDER to support the implementation of innovative approaches to clinical trial design and conduct,” said Kevin Bugin, CDER’s lead for C3TI and deputy director of operations in the Office of New Drugs.
Preclinical data of rabbits having convulsions has prompted the U.S. FDA to place a clinical hold on Neumora Therapeutics Inc.’s phase I study of NMRA-266 in healthy adults. Neumora said about 30 participants had been dosed so far in the single ascending and multiple ascending dose study, with no evidence of convulsions seen.