Nine years on from securing $3.84 million for a phase I clinical trial to test the formulation, with results showing it overcame side effects that had confounded its forerunner, the schizophrenia treatment Karxt met its PDUFA date Sept. 26 with no decision by midday. If approved, the fixed combination of xanomeline-trospium will be the first in a new drug class, and as a dual M1/M4 muscarinic agonist, the first new therapy to act via a novel mechanism for the serious psychiatric disorder in over 50 years.

The U.S. FDA’s inspection of two facilities run by San Francisco-based Irhythm Technologies Inc., illuminated some of the usual problems with corrective and preventive action. The agency indicated that problems with skin irritation associated with the company’s Zio device should have been handled as medical device reports, a view the company apparently did not share.

The FDA has awarded U.S. orphan drug designation to Eydisbio Inc.’s EYD-001 (formerly HS-276), a highly selective and potent, orally bioavailable TAK1 inhibitor for the treatment of systemic sclerosis. Eydisbio plans to initiate clinical trials in the near future.

The U.S. FDA announced a class I recall declared by Baxter Healthcare Corp., of Deerfield, Ill., for the automated compounding inlets used with the company’s Exactomix compounding systems due to the risk of particulate matter in the inlets.

With two drugs cleared by the U.S. FDA for Niemann-Pick disease type C (NPC) in less than seven days, Wall Street was pondering the differences between the compounds, given what’s known so far about each. Most recently, the FDA approved Intrabio Inc.’s Aqneursa (levacetylleucine) on its PDUFA date for the treatment of neurological manifestations of NPC in adults and pediatric patients weighing at least 15 kg, making Aqneursa the only approved stand-alone therapy indicated for NPC. On Sept. 20, Zevra Therapeutics Inc. won FDA clearance for Miplyffa (arimoclomol) as the first treatment for NPC.

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.