FDA News

FDA Grants Orphan Drug Designation for Arrowhead’s Investigational Treatment of Homozygous Familial Hypercholesterolemia

PASADENA, Calif.–(BUSINESS WIRE)–Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ARO-ANG3 for the treatment of homozygous familial hypercholesterolemia (HoFH). ARO-ANG3 is a subcutaneously administered RNA interference (RNAi)-based investigational medicine targeting angiopoietin like protein 3 (ANGPTL3) being developed …

Read More »

FDA Grants Rare Pediatric Disease Designation for LogicBio’s LB-001 for the Treatment of Methylmalonic Acidemia

CAMBRIDGE, Mass., July 16, 2019 (GLOBE NEWSWIRE) — LogicBio Therapeutics, Inc. (NASDAQ:LOGC), a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients, today announced the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to LB-001, a recombinant adeno-associated viral vector …

Read More »

Seattle Genetics and Astellas Announce Submission of BLA to FDA for Enfortumab Vedotin for Advanced or Metastatic Urothelial Cancer

BOTHELL, Wash. & TOKYO–(BUSINESS WIRE)–Seattle Genetics, Inc. (Nasdaq:SGEN) and Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., “Astellas”) today announced submission of a Biologics License Application for accelerated approval to the U.S. Food and Drug Administration for the investigational agent enfortumab vedotin for the treatment of patients with locally advanced …

Read More »

FDA Grants Priority Review to Novartis’ BLA for its Investigational Sickle Cell Medicine Crizanlizumab

Basel, July 16, 2019 – Novartis today announced the US Food and Drug Administration (FDA) accepted the company’s Biologics License Application (BLA) and has granted Priority Review for its investigational sickle cell medicine crizanlizumab (SEG101). If FDA-approved, crizanlizumab is expected to represent the first monoclonal antibody targeting the P-selectin mediated multi-cellular …

Read More »

Dicerna Receives Breakthrough Therapy Designation for DCR-PHXC for Treatment of Primary Hyperoxaluria Type 1

CAMBRIDGE, Mass.–(BUSINESS WIRE)–DicernaTM Pharmaceuticals, Inc. (Nasdaq: DRNA) (the “Company” or “Dicerna”), a leading developer of ribonucleic acid interference (RNAi) therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted a Breakthrough Therapy Designation (BTD) to DCR-PHXC for the treatment of patients with primary hyperoxaluria type 1 (PH1). DCR-PHXC is …

Read More »

Janssen Submits Application to the FDA Seeking Approval of New Darzalex Subcutaneous Formulation for Treatment of Multiple Myeloma

RARITAN, N.J., July 12, 2019  — The Janssen Pharmaceutical Companies of Johnson & Johnson announced today the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking approval of a new subcutaneous (SC) formulation of DARZALEX® (daratumumab), an intravenous (IV) treatment approved for certain patients …

Read More »

FDA Accepts Merck’s sBLAs for Keytruda Six-Week Dosing Schedule for Melanoma and Multiple Other Indications

KENILWORTH, N.J.–(BUSINESS WIRE)–Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review six supplemental Biologics License Applications (sBLAs) to update the dosing frequency for KEYTRUDA, Merck’s anti-PD-1 therapy, to include an every-six-weeks (Q6W) dosing …

Read More »

ADMA Biologics Receives FDA Approval for License Transfers for Bivigam and Nabi-HB

RAMSEY, N.J. and BOCA RATON, Fla., July 08, 2019 (GLOBE NEWSWIRE) — ADMA Biologics, Inc. (NASDAQ: ADMA) (“ADMA” or the “Company”), a vertically integrated commercial biopharmaceutical and specialty immunoglobulin company that manufactures, markets and develops specialty plasma-derived biologics for the treatment of immune deficiencies and the prevention of certain infectious …

Read More »

FDA Grants Fast Track Designation to Prevail’s PR001 for the Treatment of Parkinson’s Disease with a GBA1 Mutation

NEW YORK, July 08, 2019 (GLOBE NEWSWIRE) — Prevail Therapeutics Inc. (Nasdaq: PRVL) (Prevail), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the Company’s lead gene therapy program, …

Read More »

FDA Approves Grifols’ Xembify, 20% Subcutaneous Immunoglobulin for Primary Immunodeficiencies

Barcelona, July 4, 2019.– Grifols (MCE:GRF, MCE:GRF.P NASDAQ:GRFS), a leading global producer of plasma-derived medicines, announced today that Xembify®, its new 20% subcutaneous immunoglobulin, has been approved by the U.S. Food and Drug Administration (FDA). Xembify® is used to treat primary immunodeficiencies. The FDA approval marks the culmination of an important …

Read More »