A new self-injectable therapy for polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) will be available in January 2024 now that the U.S. FDA has approved Ionis Pharmaceuticals Inc.’s Wainua (eplontersen), a ligand-conjugated antisense oligonucleotide.

With the clock ticking down on 2023, the U.S. FDA seems to be scurrying to push final guidances out the door before the new year. This week, the agency has finalized several guidances, ranging from the development of monoclonal antibodies to treat or prevent COVID-19 to the use of real-world data in drug development.

Receiving a second complete response letter (CRL) from the U.S. FDA for gefapixant to treat refractory and unexplained chronic cough – an indication for which there are no approved treatments in the U.S., Merck & Co. Inc. said it is reviewing the agency’s feedback to determine the next steps, if any, for the oral selective P2X3 receptor antagonist.

Nearly four years after the COVID-19 pandemic closed government offices and sent federal employees home to work remotely, the U.S. FDA is returning to some semblance of normalcy, with its drug and biologics centers expanding in-person face-to-face industry meetings to include all PDUFA and BsUFA meeting types, beginning Jan. 22.

Given the challenges of generating chemistry, manufacturing and control information on the compressed timelines used in the EMA’s Priority Medicines scheme and the U.S. FDA’s breakthrough therapy designation program, the two regulators published a joint question-and-answer document discussing quality and good manufacturing practice aspects of applications for both programs, which are aimed at speeding development of innovative products to address unmet medical needs.

The data package for Filsuvez (birch triterpenes), a topical gel aimed at treating partial thickness wounds, satisfied U.S. reviewers the second time around. Chiesi Group said the FDA approved the treatment Dec. 19 for use in patients 6 months and older with rare blistering skin diseases junctional epidermolysis bullosa and dystrophic epidermolysis bullosa.

South Korea’s GC Biopharma Corp. has gained U.S. FDA approval for its immune-deficiency plasma drug, Alyglo (immune globulin intravenous, human-stwk; IVIG-SN 10%), five months after refiling its BLA and before its PDUFA date of Jan. 13, 2024.

Shares of Checkpoint Therapeutics Inc. dropped 44.8% Dec. 18 on news that the U.S. FDA issued a complete response letter (CRL), delaying potential approval of PD-L1 antibody cosibelimab in metastatic or locally advanced cutaneous squamous cell carcinoma. The FDA had accepted in March the BLA, seeking use for treating patients who are not candidates for curative surgery or radiation, setting a Jan. 3, 2024, PDUFA date.

Following the recommendation of its Oncology Drugs Advisory Committee, the U.S. FDA approved U.S. Worldmeds LLC’s eflornithine 192-mg tablets for use as a maintenance therapy in patients with high-risk neuroblastoma. Branded Iwilfin, the specific, irreversible inhibitor of ornithine decarboxylase previously known as DFMO, is expected to be available in the coming weeks.