HYOGO, Japan — JCR Pharmaceuticals Co., Ltd. Chairman and President Shin Ashida announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation (ODD) to JR-441, an investigational drug for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA, or Sanfilippo syndrome type A). JR-441 is a blood-brain barrier...
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SAN RAFAEL, Calif. – BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the U.S Food and Drug Administration (FDA) placed a clinical hold on the BMN 307 Phearless Phase 1/2 study. The Phearless study is evaluating BMN 307, an investigational AAV5-phenylalanine hydroxylase (PAH) gene therapy, in adults with phenylketonuria (PKU). ...
NORTH CHICAGO, Ill. — AbbVie (NYSE: ABBV) today announced that the U.S. Food and Drug Administration (FDA) has granted Priority Review of the supplemental Biologics License Application (sBLA) for epcoritamab-bysp, a subcutaneously administered T-cell engaging bispecific antibody for the treatment of adult relapsed or refractory (R/R) follicular lymphoma (FL) after...
SOMERSET, N.J. – Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global clinical-stage biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and other indications, has announced that the U.S. Food and Drug Administration (FDA) has accepted for priority review the Biologics License Application (BLA)...
RARITAN, N.J. – Johnson & Johnson announced today that the U.S. Food and Drug Administration (FDA) approved a supplemental New Drug Application (sNDA) for BALVERSA® (erdafitinib) for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma (mUC) with susceptible fibroblast growth factor receptor 3 (FGFR3) genetic alterations...
NEW YORK, NY – Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted its allogeneic cell therapy Revascor® (rexlemestrocel-L) an Orphan-Drug Designation (ODD) following submission of results from the randomized controlled trial in children...
Copenhagen, Denmark – Zealand Pharma A/S, a biotechnology company focused on the discovery and development of innovative peptide-based medicines, today announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for Part 1 of the New Drug Application (NDA) for dasiglucagon for the prevention...
It’s shortly after 5 a.m. when the phone rings, and on the line is a clearly anxious and worried parent. “Sierra is having a lot of problems tonight,” Shaylene Akery tells a CNN producer. “We have to take her to the hospital, but we still want to talk to you...
United States Sens. Tim Kaine (D-VA), Mark R. Warner (D-VA), Jerry Moran (R-KS) and Bill Cassidy (R-LA) introduced Thursday the Gabriella Miller Kids First Research Act 2.0, which would provide additional funding for childhood cancer research. The bill is named in honor of Gabriella Miller, a Leesburg resident who died...
BIRMINGHAM, Ala. – The University of Alabama at Birmingham startup TIXiMED, Inc., has obtained clearance from the U.S. Food and Drug Administration to proceed to clinical trials under an Investigational New Drug for TIX100, its novel oral Type 1 diabetes drug. This represents a major milestone in the development of...