CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the supplemental New Drug Application (sNDA) for olezarsen for severe hypertriglyceridemia (sHTG). The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of June 30, 2026. “Current...
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WHITE PLAINS, N.Y. – Oligomerix, Inc., a privately held company pioneering the development of small molecule therapeutics targeting tau for rare neurodegenerative and Alzheimer’s diseases, today announced the appointment of William Erhardt, M.D., Chief Medical Officer of Oligomerix, to its Board of Directors. “As Chief Medical Officer, Bill has provided...
INDIANAPOLIS, Indiana — Eli Lilly and Company (NYSE: LLY) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to olomorasib as a monotherapy for the treatment of adult patients with advanced pancreatic cancer who have received at least one prior systemic therapy and have a KRAS G12C mutation, as determined by an FDA-approved...
SUZHOU, China, and ROCKVILLE, Md. — Ascentage Pharma, a global biopharmaceutical company engaged in developing novel therapies for cancer, chronic hepatitis B (CHB), and age-related diseases, announced today that olverembatinib (R&D Code: HQP1351) has been included in the latest guidelines from the National Comprehensive Cancer Network (NCCN) for the management...
On a Wednesday morning in March 2021, Louise Storey, a champion rower who represented New Zealand at the London Olympics and several World Championships, pulled into a car park next to a Cambridge petrol station for a phone call that would change her family’s life. Louise (née Trappitt) had dropped...
SEATTLE, Washington — Omeros Corporation (Nasdaq: OMER) today reported that clinical trial site activation for enrollment is underway for the company’s Phase 3 program evaluating zaltenibart in paroxysmal nocturnal hemoglobinuria (PNH). Zaltenibart (OMS906) is Omeros’ investigational inhibitor of MASP-3, the key and most proximal activator of the alternative pathway of...
Four decades after a rare neurological disorder first surfaced, an Oklahoma Medical Research Foundation scientist has determined its cause – thanks to tiny flies. Scientist Wan Hee Yoon used Drosophila, the common fruit fly, to pinpoint a genetic mutation that more recently caused severe developmental and metabolic issues in four...
RemeGen Co., Ltd., a commercial-stage biotechnology company, continues to strengthen its social responsibility and rare disease advocacy in unison with Rare Disease Day 2023's message of continuing to increase visibility and generate change for the millions of people globally living with a rare disease, their families and carers
WASHINGTON, D.C. —- Tuesday, U.S. Sen. Ben Ray Luján (D-N.M.), a member of the Senate Committee on Health, Education, Labor and Pensions, and U.S. Sen. Martin Heinrich (D-N.M.) reintroduced the Cerebral Cavernous Malformations Clinical Awareness, Research and Education Act (CCM-CARE) which would expand National Institutes of Health (NIH) research related...
ROCKVILLE, Md. — OncoC4, Inc. announced that the first patient with metastatic castration resistant prostate cancer (“mCRPC”) has been dosed in a Phase 1/2 trial evaluating the anti-CTLA-4 antibody candidate BNT316/ONC-392 (gotistobart) in combination with radioligand therapy, lutetium (177Lu) vipivotide tetraxetan (Pluvicto®). BNT316/ONC-392 is being jointly developed by BioNTech and...