Sanofi and partner Swedish Orphan Biovitrum shared positive topline results from their ongoing Phase III trial on a candidate drug for patients diagnosed with severe hemophilia A.
BioMarin updated several of the company’s gene therapy programs on February 17 following a September 2021 update, when the drug manufacturer reported the U.S. Food and Drug Administration had placed a clinical hold on the BMN 307 Phearless Phase I/II trial.
BioMarin Pharmaceutical shared positive results from the company’s ongoing Phase III GENEr8-1 trial on the viability of valoctocogene roxaparvovec as a treatment for patients diagnosed with severe hemophilia A.
Genentech’s Hemlibra (emicizumab-kxwh) showed no new safety signals and continued to deliver consistent bleeding control in the final analysis of a long-term, late-stage hemophilia A study.
After touting positive Phase III results at the American Society of Hematology’s annual meeting, the U.S. FDA placed a clinical hold on uniQure”s hemophilia B treatment AMT-061 due to a patient developing liver cancer.
Sanofi Hits the Brakes on Its Hemophilia Program Published: Nov. 6, 2020 By Mark Terry BioSpace Three hemophilia organizations, the World Federation of Hemophilia, the European Haemophilia Consortium and […]
FDA Action Alert: Bristol Myers Squibb/Juno, Seattle Genetics, BioMarin and Tricida
Adeno-Associated Virus (AAV), B Cell Lymphomas, Biologics License Application (BLA), Brain Metastases, CAR-T Therapy, Chronic Kidney Disease, Diffuse large B-cell lymphoma (DLBCL), FDA, FDA/Regulatory, Hemophilia A, HER2-positive breast cancer, Immunotherapy, New Drug Applicationa, Project Orbis, Quarterly results, T-CellsThe U.S. Food and Drug Administration’s approval-review schedule for August includes the Biologics License Application for Bristol Myers Squibb and Juno Therapeutics’ lisocabtagene maraleucel (liso-cel) for relapsed or refractory large B-cell lymphoma after at least two previous therapies.
Ahead of a planned Phase III study, Pfizer and Sangamo Therapeutics released follow-up data from its Phase I/II Alta study in hemophilia A that showed an investigational gene therapy treatment provided blood-clotting activity levels for more than one year.
Denmark’s Novo Nordisk paused three clinical trials of concizumab over safety issues. The studies are evaluating the drug for hemophilia A and B.
Top 10 Pipelines To Watch
Acquisitions, Analysts, Annual Reports, Blockbuster Deals, Blockbusters, Business, Clinical Trials, Deals, Dengue, Department of Health and Human Services (HHS), Dermatology, Diffuse Large B-Cell Lymphoma (DLBCL), EMA, European Commission, European Medicines Agency (EMA), FDA, FDA/Regulatory, February 2020, Future Blockbusters, Hemophilia A, Hospital-Acquired Bacterial Pneumonia, Issue Archives, Licensing, Natural Killer Cells (NK Cells), New Drug Applications, New Molecular Entities, NMEs, Oncology, Potential Blockbusters, Priority Review, Priority Review Status, Product Pipelines, R&D, Rare Diseases, Senate Panel, Special Reports, Top 10 Pipelines, Top 10 Pipelines To Watch, VaccinesAs pharma companies bolster their pipelines via multibillion-dollar acquisitions, drug manufacturers continue to heavily invest in the oncology space.