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 ...

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 ...

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

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

FDA granted Orphan Drug Designation to Sanofi’s SAR446523, a monoclonal antibody targeting GPRC5D, for relapsed/refractory ...

The One Big Beautiful Bill Act ("OBBB") expands exemptions for orphan drugs from mandatory Medicare price negotiations and modifies their ...

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 ...

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

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

In April 2025, the FDA granted orphan drug designation to spesolimab-sbzo for the treatment of pyoderma gangrenosum. This ...