The FDA grants orphan drug designation to drugs aimed at treating rare diseases or conditions affecting fewer than 200,000 people in the US. This is a significant milestone for Sanofi (NASDAQ:SNY ...

Orphan Drug Designation is a critical regulatory pathway that supports the development of treatments for rare diseases affecting fewer than 200,000 individuals in the U.S. Rett Syndrome, a severe ...

The FDA’s Office of Orphan Drug Products grants ODD and RPDD to encourage the development of medicines for rare conditions affecting less than 200,000 people in the U.S. The ODD offers several key ...

The FDA Orphan Drug Designation is a special status granted to drugs and biologics targeting rare diseases, defined as those affecting fewer than 200,000 people in the United States. This designation ...

The FDA Office of Orphan Products Development grants orphan drug designation to support the development of medicines for rare disorders that affect fewer than 200,000 people in the U.S. Under the ...

The FDA granted an orphan drug designation to novel hedgehog pathway inhibitor taladegib, also known as ENV-101, for treating patients with idiopathic pulmonary fibrosis, according to a press ...

The FDA’s orphan drug designation is granted to drugs and biologics intended for the treatment, diagnosis, or prevention of rare diseases affecting fewer than 200,000 people in the United States. The ...

"Trofinetide, which received FDA approval in 2023 for Rett Syndrome, also holds Orphan Drug Status. Given the encouraging data from our study, we see a strong case for MB-204 to follow a similar ...