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.

Medtech Insight talked to two leaders at the Alzheimer’s Drug Discovery Foundation about current therapies and the role of biomarkers and digital tools to build on recent advancements in the space and accelerate progress to identify the disease early and slow cognitive decline.