Sanofi has halted work on a phase 2 candidate for a rare growth disorder as part of a pipeline clear-out that saw the French pharma discontinue two other rare disease assets and one of its mRNA flu vaccines.
The FDA’s Oncologic Drugs Advisory Committee (ODAC) has unanimously called for a revamp of perioperative clinical trial designs in resectable non-small cell lung cancer (NSCLC) to include assessment of the contribution of each treatment phase.
MELBOURNE, Australia, July 22, 2024 (GLOBE NEWSWIRE) -- Certa Therapeutics (Certa), a biotechnology company developing innovative precision therapies for patients with inflammatory and fibrotic...
LUND, Sweden, July 18, 2024 (GLOBE NEWSWIRE) -- Abliva AB (Nasdaq Stockholm: ABLI), a clinical-stage company developing drugs for the treatment of rare and severe primary mitochondrial disease, today announced a positive outcome of the interim analysis for FALCON, the potentially registrational study evaluating KL1333 in patients with primary mitochondrial disease. The study evaluates fatigue and myopathy as alternative independent primary endpoints, only one of which is required for a successful study readout. The independent Data Monitoring Committee (DMC) recommended continuing the study without modification. The favorable recommendation by the DMC to continue the FALCON study with a total of 180 patients validates the overall study design and confirms the strong safety profile of KL1333.
Renowned Rare-Disease Drug Developer Emil Kakkis, M.D., Ph.D., to Join Actio Biosciences Board of Directors
SHANGHAI, July 16, 2024 /PRNewswire/ -- Mabwell (688062.SH), an innovation-driven biopharmaceutical company with entire industry chain, announced that its self-developed novel B7-H3-targeting ADC (R&D code: 7MW3711) has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA), for the treatment of small cell lung cancer.
NEW YORK, July 16, 2024 (GLOBE NEWSWIRE) -- SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) (“SELLAS” or the “Company”), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to SLS009, a highly selective CDK9 inhibitor, for the treatment of pediatric acute myeloid leukemia (AML). The FDA previously granted Orphan Drug and Fast Track Designations to SLS009 for the treatment of AML.
LOS ANGELES, July 16, 2024 (GLOBE NEWSWIRE) -- Trethera Corporation, a clinical stage biopharmaceutical company committed to developing novel drugs targeting nucleotide metabolism for the treatment...
Achieved mean reduction in left ventricular mass index (LVMI) of 11.4% at 12 months and 18.3% at 18 months in participants with elevated LVMI at baseline >10% reduction in LVMI at 12 months...
BETHESDA, Md., July 15, 2024 /PRNewswire/ -- A new survey of 800 rare disease (RD) patients and caregivers from the U.S., United Kingdom (UK), Spain, and Germany, was conducted by Avant Health and Rare Patient Voice between February and April 2024. 66% of RD patients report taking up to five years to get the RD diagnosed; this ranged between 74% (U.S) and 56% (UK). These results were part of a survey asking RD patient/caregiver opinions about their diagnostic journey, access to healthcare, and perspectives on living with a RD.