The FDA has cleared an IND application for a phase I/II study of KSQ-004EX, KSQ Therapeutics Inc.’s second engineered tumor-infiltrating lymphocyte (eTIL) program. KSQ-004EX consists of TIL in which the genes encoding SOCS1 and Regnase-1 are inactivated using CRISPR/Cas9 gene editing.

Michelle Tarver, the acting director of the U.S. FDA’s device center appeared on a Sept. 24 webinar to discuss her priorities for the agency going forward, one of which is global health equity.

Chengdian (Suzhou) Biopharmaceutical Co. Ltd. has received IND clearance from the FDA, allowing it to initiate a first-in-human phase I trial of CD-001.

August saw the U.S. FDA approve 22 drugs, an increase from July's 17 but fewer than the 28 approved in June. This brings the 2024 monthly average to just over 19 approvals, outpacing last year's average of 16 per month, as well as the 12.5 per month seen in 2022 and 17 per month in both 2021 and 2020.

The U.S. FDA wrapped up the pilot version of the Accreditation Scheme for Conformity Assessment (ASCA) and reported the formation of a permanent ASCA program, which has been seven years in the making.

Innate Pharma SA has obtained IND clearance from the FDA for IPH-4502, its novel and differentiated topoisomerase I inhibitor antibody-drug conjugate (ADC) conjugated to exatecan targeting Nectin-4 in solid tumors. In nonclinical models, IPH-45 was well tolerated and showed antitumor efficacy in vitro and in vivo.

Following the recent recommendation of its advisory committee, the U.S. FDA gave the nod to Zevra Therapeutics Inc.’s arimoclomol, a day ahead of its Sept. 21 PDUFA date, as the first treatment for Niemann-Pick disease type C (NPC), a rare genetic disease linked to progressive neurological symptoms that is almost always fatal. Branded Miplyffa, it is indicated for use in combination with enzyme inhibitor miglustat in adults and children, ages 2 and older.