46% reduction in the risk of disease progression or death, and 27% reduction in the risk of death, in an aggressive cancer type with limited survival and few treatment options 1 First Phase III study in ES-SCLC first-line maintenance to demonstrate clinically meaningful improvements in both progression-free and overall survival 2,3 Data...
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CRANBURY, N.J. — Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Company’s pivotal Phase 2 trial of...
CRANBURY, N.J. — Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has accepted the resubmission of the Biologics License Application (BLA) for KRESLADI™ (marnetegragene...
HEBER CITY, Utah (ABC-4 Sports) – Just eight months ago, Jade Hafen wasn’t sure if he would be able to walk again. After competing in a calf roping event, Hafen collapsed, unable to move his legs. He was eventually diagnosed with Guillain-Barre syndrome, a dehabilitating nerve disorder in which the...
BASEL, Switzerland, LONDON, UK, NEW YORK, NY and DURHAM, N.C. — Roivant (Nasdaq: ROIV) and Priovant Therapeutics today announced positive results from the Phase 3 VALOR study evaluating brepocitinib in dermatomyositis (DM). On the primary endpoint, brepocitinib 30 mg achieved a week 52 mean TIS of 46.5 compared to 31.2...
Catonsville, Maryland – Prostate cancer is the most common cancer in men other than skin cancer, and more than 288,000 new cases are diagnosed every year, according to the American Cancer Society. The disease’s fatality rate has decreased by more than half since the 1990s, but there is still room...
Cambridge, UK – Large-scale genetic analysis has helped researchers uncover the interplay between cancer-driving genetic mutations and inherited genetic variants in a rare type of blood cancer. Researchers from the Wellcome Sanger Institute, the University of Cambridge, and collaborators, combined various comprehensive data sets to understand the impact of both...
NORTH CHICAGO, Ill. — BLR Bio, a biotechnology company in Rosalind Franklin University’s Helix 51 incubator, announced new data on its novel therapy for systemic sclerosis and lung fibrosis at the British Society for Rheumatology (BSR) Annual Conference this week in Manchester, UK. Interstitial Lung Disease (ILD) is a devastating disorder, encompassing a group of chronic lung...
Paris, France – With ECRD 2024 scheduled for 15 and 16 May in Brussels and online, EURORDIS’ session, Revolutionising Funding Strategies for Breakthrough Therapies in Rarer Diseases, will address this critical issue. This session will not only highlight best practices but also showcase examples of therapies developed through innovative funding...
SAN FRANCISCO, Calif. — FibroGen, Inc. (NASDAQ: FGEN) today announced that the Office of Orphan Products Development of the U.S. Food and Drug Administration (FDA) has granted roxadustat Orphan Drug Designation for the treatment of myelodysplastic syndromes (MDS). “The Orphan Drug Designation granted to roxadustat for MDS underscores the significant...