While Spruce Biosciences Inc.’s tildacerfont missed its phase II primary endpoint in classic congenital adrenal hyperplasia (CAH) last March, the U.S. FDA approved Crenessity (crinecerfont) from Neurocrine Biosciences Inc. for treating pediatric and adult CAH patients. The nod could lead the drug to become a blockbuster, analysts said.

Heartbeam Inc. received U.S. FDA 510(K) clearance for its portable, non-invasive electrocardiogram system that enables on-the-go recording of heart signals. The cable-free, credit card-sized device uses five electrodes to capture signals from three directions and can be used whenever a patient feels symptoms, overcoming the challenge of identifying intermittent arrhythmias, which are often not experienced during scheduled appointments, without requiring patients to wear a device continuously.

The U.S. FDA’s emphasis on alternatives to ethylene oxide is gaining momentum with the help of internationally recognized standards — such as ISO 11737 — in a move that will enable a less cumbersome approach to non-EtO device sterilization.

Caliway Biopharmaceuticals’ lipolysis candidate, CBL-514, met all the primary and secondary efficacy endpoints in a phase IIb study for subcutaneous fat reduction, paving the way for the company to begin a global pivotal phase III study in 2025.

After Keros Therapeutics Inc.’s voluntary halt of dosing in two arms of its phase II study in pulmonary arterial hypertension, the company’s stock crumpled after a year of muscular performance and its recent and massive deal with Takeda Pharmaceutical Co. Ltd.

Splicebio S.L. has gained IND clearance from the FDA for its lead program SB-007, a protein splicing gene therapy to address the root genetic cause of Stargardt disease.

Kura Oncology, Inc. and Kyowa Kirin Co. Ltd.’s selective oral menin inhibitor ziftomenib showed encouraging data across multiple studies, the most encouraging of which were in combination with other standard of care therapies in patients with NPM1-mutant and KMT2A-rearranged  acute myeloid leukemia.

The FDA’s new program for communicating the hazards of devices is in play and resulted in the posting of a notice of a problem seen with the Ivenix line of large volume infusion pumps by Fresenius Kabi USA, which will require some repair work by the manufacturer.

Astria Therapeutics Inc. has gained IND clearance from the FDA for STAR-0310, a monoclonal antibody OX40 antagonist being developed for atopic dermatitis.