The U.S. Food and Drug Administration issued Incyte Corporation with a Complete Response Letter (CRL) over the company’s Biologics License Application (BLA) for retifanlimab for anal cancer.
Fast Track, Breakthrough and Priority Reviews: Sanofi and ViiVAcid alpha-glucosidase (GAA) enzyme, Biologics License Application (BLA), Breakthrough Therapy Designation, Bruton's Tyrosine Kinase (BTK) Inhibitors, Chronic Immune Thrombocytopenia (ITP), Clinical Trials, Enzyme Replacement Therapy (ERT), Fast Track Designation, FDA, HIV pre-exposure prophylaxis (PrEP), HIV Prevention, Muscle Disease, Orphan Drug Designation, PDUFA, Pompe disease, Priority Review Status, R&D, Sanofi
The U.S. Food and Drug Administration granted various designations for Sanofi’s avalglucosidase alfa for Pompe disease, Sanofi’s rilzabrutinib for immune thrombocytopenia, and ViiV Healthcare’s cabotegravir for HIV prevention.
The U.S. Food and Drug Administration approved Incyte’s Pemazyre (pemigatinib), a kinase inhibitor indicated for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 fusion or other rearrangement as detected by an FDA-approved test.
The U.S. Food and Drug Administration granted orphan drug designation to AFM13, Affimed N.V.’s lead CD30- and CD16A-binding innate cell engager, for the treatment of patients with T-cell lymphoma.
A day after the announcement that remdesivir received Orphan Drug designation for the treatment of COVID-19 from the U.S. Food and Drug Administration, Gilead Sciences submitted a request for the regulatory agency to rescind that offer after an outcry was raised over the potential for exclusivity for the experimental pandemic treatment.
Zogenix Reports Positive Results from Phase III Trial of Rare Childhood Epilepsy StudyChildren, Clinical Data, Clinical Trials, Dravet syndrome, European Commission, European Medicines Agency (EMA), FDA, FDA/Regulatory, Lennox-Gastaut Syndrome (LGS), New Drug Application (NDA), Orphan Drug Designation, Orphan Drug Designation, Priority Review, R&D
Emeryville, California-based Zogenix announced positive topline data from the company’s Phase III trial of Fintepla (fenfluramine) in Lennox-Gastaut Syndrome.
Horizon Therapeutics Wins FDA Approval for First Thyroid Eye Disease TreatmentApprovals, Autoimmune Disorders, FDA, FDA/Regulatory, Graves' Disease, Infusions, Monoclonal Antibodies, New Drug Application (NDA), Orphan Drug Designation, Priority Review, Proptosis (Eye Bulging), Rare Diseases, Thyroid Eye Disease (TED), Thyroid Hormones
Ireland’s Horizon Therapeutics became the first to win regulatory approval in the United States for the treatment of Thyroid Eye Disease (TED), a progressive autoimmune disorder that can threaten the vision of those afflicted.
The U.S. FDA granted Orphan Drug Designation for OBI Pharma Inc.’s first-in-class antibody drug conjugate (ADC) OBI-999 for the treatment of pancreatic cancer.
The U.S. FDA approved Celgene and Acceleron Pharma’s Reblozyl for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions.