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 ...

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 development.

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 ...

Sanofi's SAR446523 gains FDA orphan drug status, offering hope for relapsed multiple myeloma patients with its targeted ...

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

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

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 ...

Daewoong Pharmaceutical (Co-CEOs Seong-Soo Park and Chang-Jae Lee) and Daewoong Therapeutics (CEO Bok-Ki Kang) ...

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 ...