Sanofi’s rilzabrutinib designated breakthrough therapy in the US and orphan drug in Japan for the treatment of warm autoimmune hemolytic ...

Zydus Lifesciences on Friday said the U.S. health regulator has granted Orphan Drug Designation to Desidustat, a novel oral ...

Rilzabrutinib is an investigational, oral reversible Bruton tyrosine kinase inhibitor. The Food and Drug Administration (FDA) has granted Orphan Drug designation to rilzabrutinib for the treatment of ...

Zenocutuzumab--zbco, marketed as BIZENGRI (R), received Breakthrough Therapy Designation from the FDA in October 2025 and was granted accelerated approval in December 2024 for adults with advanced ...

Half of the planned 200 ALS patients have joined Medicinova’s expanded access program evaluating the experimental drug MN-166.

Investor enthusiasm and evolving FDA pathways are accelerating rare disease drug development, with ultrarare conditions like ...

The One Big Beautiful Bill Act takes a big step in the rare disease space by expanding the contours of the orphan drug exclusion, a once narrow exception that permitted manufacturers of rare disease ...

Confirmation Received from MHLW That QRX003 Qualifies for Both Orphan Drug Designation and Fast Track Regulatory Review ...

Aug. 1, 2005 — Europe has approved orphan drugs pegylated arginine deiminase for the treatment of hepatocellular carcinoma, soluble yeast beta-1,3/1,6-glucan for the prophylaxis of mucositis due to ...

Sanofi's rilzabrutinib designated breakthrough therapy in the US and orphan drug in Japan for the treatment of warm autoimmune hemolytic anemia Rilzabrutinib is the first and only investigational BTKi ...

Policy strategist Kristi Martin discusses how the Orphan Drug Act reshapes rare disease treatment and drug-pricing challenges ...

A milestone in the treatment of Duchenne muscular dystrophy (DMD) passed recently with remarkably little public notice. That ...