FDA approves first stem cell therapy to treat severe aplastic anemia

The U.S. Food and Drug Administration announced the approval of the first hematopoietic stem cell transplant therapy to treat ...

FDA approves first gene therapy for rare immune disorder

The U.S. Food and Drug Administration said on Tuesday it had approved the first gene therapy for a rare and life-threatening ...

Rezolute's rare disease drug fails to meet main goal in late-stage trial

Rezolute said on Thursday its experimental drug for a rare genetic condition that causes dangerously low blood sugar failed ...

How a rare drug made from scientists’ blood saves babies from botulism

An infant botulism outbreak has sickened at least 39 babies across the U.S., underscoring the crucial role of a medication ...

FDA approves BioCryst's oral pellet version of rare disease drug for children

The U.S. Food and Drug Administration has approved an oral pellet formulation of BioCryst Pharmaceuticals' drug to prevent ...
Opinion
MinnPostOpinion

Congress can save lives with Rare Pediatric Disease PRVs

For Americans living with rare diseases, the Rare Pediatric Disease Priority Review Voucher Program is a vital tool. Yet its reauthorization is at risk.
Fierce Pharma

Italian nonprofit lands FDA approval for 1st gene therapy to treat rare genetic disease

The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the ...
BioSpace

Saol To Pitch Ultrarare Disease Drug as “Poster Child” for RDEP Program

Saol Therapeutics received a complete response letter for its pyruvate dehydrogenase complex deficiency treatment a week ...
BioSpace

FDA Greenlights First Gene Therapy for Rare Pediatric Disease

Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a ...
STAT

In a first, a nonprofit wins FDA approval for rare disease gene therapy

The FDA has granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug ...

Roxadustat Granted Orphan Drug Designation for the Treatment of Myelodysplastic Syndromes by the U.S. Food and Drug Administration

Company is on track to file the Phase 3 protocol in the fourth quarter of 2025 SAN FRANCISCO, Dec. 15, 2025 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN) today announced that the Office of Orphan ...

Fortress Biotech and Cyprium Therapeutics Announce FDA Acceptance of CUTX-101 NDA Resubmission

New PDUFA Target Action Date of January 14, 2026 set by FDAMIAMI, Dec. 15, 2025 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc.