CURE

FDA Grants Orphan Drug Status to Cintredekin Besudotox for Glioblastoma

The FDA granted orphan drug designation to cintredekin besudotox for glioblastoma, supporting further research for this ...
Targeted Oncology

FDA Grants Orphan Drug Status for MNV-201 in Low-Risk MDS

Orphan drug designation provides benefits such as tax credits, PDUFA fee waivers, and 7 years of market exclusivity upon ...
CURE

FDA Grants Orphan Drug Designation to AMXT 1501 With DFMO in Neuroblastoma

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AMXT 1501 in combination with difluoromethylornithine (DFMO) for the treatment of patients with neuroblastoma, it has ...
Wichita Eagle

FDA Approves Orphan-Drug Designation for Jaguar Health’s Crofelemer for Treatment of Diarrhea in Cholera

Crofelemer previously granted orphan-drug designation by the FDA and the European Medicines Agency (EMA) for both short bowel syndrome and microvillus inclusion disease SAN FRANCISCO, CA / ACCESSWIRE ...
BioSpace

FDA’s Orphan Drug Director Out Amid IRA Exemption Rollout

An unnamed source clarified that Sandra Retzky has not been fired from the FDA, but it remains unclear where she was ...
KTLA

AAVantgarde Announces FDA Orphan Drug Designation and UK CTA approval for AAVB-039 for the Treatment of Stargardt Disease

AAVB-039 addresses the root cause of the disease, benefitting patients with any ABCA4 mutation AAVB-039 is currently being assessed in the CELESTE interventional clinical trial and we continue ...
Hosted on MSN

Mesoblast (MESO) Gets 7 Years of Orphan-drug Exclusive Approval from US FDA for Ryoncil

Mesoblast Limited (NASDAQ:MESO) is one of the 7 Best ASX Stocks to Buy Now. The company announced that it received 7 years of orphan-drug exclusive approval from the US FDA for Ryoncil® (remestemcel-L ...