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.