The FDA recently granted EXG-34217 Rare Pediatric Disease Designation (RPDD) and Regenerative Medicine Advanced Therapy (RMAT) Designation.

Biodexa, which has already received U.S. FDA Orphan Drug designation for eRapa in FAP, plans to seek a similar designation in Europe. eRapa is a proprietary oral tablet formulation of rapamycin ...

Ac-SSO110 has now received FDA orphan drug designation and investigational new drug application clearance for the treatment ...

Ensoma Inc.’s lead program, EN-374, has been granted orphan drug and rare pediatric disease designations by the FDA for the ...

The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) and rare paediatric disease designation (RPDD) to Arbor Biotechnologies' gene editing therapeutic, ABO-101, to treat ...

"The Orphan Drug Designation not only acknowledges the critical need for new, effective treatments for HAE in Japan but underscores the potential of sebetralstat to provide meaningful relief for ...

Zydus has announced that the USFDA has granted Orphan Drug Designation (ODD) to Usnoflast, a novel oral NLRP3 inhibitor, for the treatment of amyotrophic lateral sclerosis (ALS). The designation ...