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

Little-known provisions in the Big Beautiful Bill lobbied by industry will cost Medicare billions in missed savings.

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

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

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

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

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

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 ...

That provision is the Orphan Cures Act, which exempts certain drugs that treat rare diseases from the scheme of price controls Democrats established in Medicare as part of the 2022 Inflation ...

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 ...

The case of Charleigh Pollock in B.C. shows that politics should have no place in difficult and complex medical decisions ...