Applied Therapeutics Inc.

A third-party vendor undermined Applied Therapeutics ’ bid to bring the first drug for the rare genetic metabolic disease galactosemia to market by deleting electronic clinical outcome assessment dat

With only six novel candidates on its November 2024 user fee calendar, the US Food and Drug Administration is running out of chances to reach its 2023 total of 72 new molecular entities and novel biol

Rare diseases are almost certain to remain one of the biggest areas for novel drug development in 2024, but the orphan drug pipeline will be making space for some novel therapies for common conditions

Two newly announced June dates go some way to alleviating the recent drought in advisory committee meetings outside oncology. Both gatherings of committees under the Office of Neuroscience promise fir