T O APPRECIATE the dilemmas the Food and Drug Administration (FDA) faces when it reviews new drugs targeting rare diseases, ...

The One Big Beautiful Bill Act takes a big step in the rare disease space by expanding the contours of the orphan drug ...

The approval is supported by strong preclinical data demonstrating MP1032's capacity to sustain muscle function.

Kedrion is proud to announce that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to ...

The FDA awarded orphan drug designation to VT3989 for mesothelioma, supporting the experimental cancer treatment’s ...

The legal battle over the scope of orphan drug exclusivity continues, as the U.S. Food and Drug Administration (“FDA”) has recently lost a second ...

SAR446523, an investigational antibody targeting GPRC5D, received FDA orphan drug status and is in phase 1 trials for ...

Precision BioSciences Inc., a clinical-stage company developing gene editing therapies for diseases with high unmet need, has ...

The FDA's Orphan Drug Designation program provides orphan status to drugs or biologics intended for the treatment of diseases that affect fewer than 200,000 people in the United States.

Drug companies have demonstrated an "increasingly common pattern of gaming the system" by slicing and dicing indications so that drugs qualify for lucrative orphan status benefits and "getting ...

Kedrion Biopharma receives US FDA orphan drug designation for plasma-derived treatment for congenital aceruloplasminemia: Forte Lee, New Jersey Saturday, August 9, 2025, 17:00 Hrs ...