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...
treatment News
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...
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...
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...
EDINBURGH, Scotland — RoslinCT, a global contract development and manufacturing organization (CDMO) specializing in advanced cell therapies, and BOOST Pharma, a clinical-stage biotechnology company developing first-in-class cell therapies for rare paediatric skeletal diseases, today announced a strategic manufacturing partnership to support the development of BOOST Pharma’s cell therapy, BT-101, for...
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...
Manchester, UK – Babies and toddlers with a rare and fatal genetic condition can now receive life-saving treatment on the NHS for the first time. This follows clinical research studies first carried out at the National Institute for Health and Care Research (NIHR) Manchester Clinical Research Facility (CRF) at Royal...
CAMBRIDGE, Mass. & LEICESTER, England — RS Oncology (RSO), a clinical stage biotechnology company developing innovative therapies to eradicate mesothelioma and other diseases, today announced the successful dosing of the first patient in the Phase 2 dose expansion portion of its United Kingdom multicenter study (MITOPE; NCT05278975). Malignant pleural effusion...
Plainsboro, New Jersey, Jacobus Pharmaceutical Company Inc. is voluntarily recalling 3 lots of Ruzurgi® (amifampridine) 10 mg tablets to the consumer level. The products have been found to be contaminated with yeast, mold, and aerobic bacteria based on laboratory test results. Oral products heavily contaminated with yeast, mold, and aerobic...
KRAKOW, Poland — Ryvu Therapeutics (WSE: RVU), a clinical-stage drug discovery and development company focused on novel therapies targeting emerging oncology indications, announces its involvement in the MEDWAY project with a new non-commercial academic Phase I study to evaluate the CDK8/19 inhibitor RVU120 in combination with everolimus in children with recurrent or progressive...