Half of the FDA’s upcoming 2024 user fee goals target rare diseases, but highly prevalent conditions like uncomplicated urinary tract infections and atopic dermatitis are also up for agency action in the second half of the year.

The new panel’s inaugural 2 August meeting will take up a challenging drug application, currently in its second review cycle, for Niemann-Pick disease type C, an ultra-rare condition without approved therapeutics.

With the urea cycle disorder treatment Olpruva launched and the NPC therapy arimoclomol closing in on US approval, Zevra is one to watch in the rare disease space.

Newly announced US FDA advisory committee meetings on Lilly’s Alzheimer’s antibody, Lykos’ pioneering psychedelic therapy for PTSD relieve drought in non-oncology AdComms as the 2024-‘25 COVID vaccine strain selection meeting is postponed.