BOSTON, Mass. — The Barth Syndrome Foundation (BSF), the only patient advocacy organization dedicated to Barth syndrome and saving lives around the world through education, advances in treatment and finding a cure, today announced that it is petitioning the U.S. Food and Drug Administration (FDA) to review the New Drug...
treatment News
PLEASANTON, Calif. & NANJING, China – IASO Biotherapeutics (IASO Bio) and Innovent Biologics, Inc. (Innovent, HKEX: 01801), today jointly announced that the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) has granted Breakthrough Therapy Designation (BTD) for CT103A, an investigational chimeric antigen receptor (CAR)-T cell therapy...
WILMINGTON, Del. — Positive high-level results from an interim analysis of the ECHO Phase III trial showed AstraZeneca’s CALQUENCE® (acalabrutinib) in combination with standard-of-care chemoimmunotherapy, bendamustine and rituximab, demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS) versus standard of care in previously untreated adult patients with...
Cambridge, UK – A combination of two drugs could improve outcomes and reduce the need for toxic chemotherapy for B-cell acute lymphoblastic leukemia (B-ALL), the commonest cancer in childhood and one that can be particularly difficult to treat in older patients, according to Cambridge scientists. Although the research has so...
Madrid, Spain – A few years ago, a patient was cured of multiple myeloma after being treated for hepatitis C, astounding researchers from the group led by Joaquín Martínez, from the H12O-CNIO Haematological Tumours Clinical Research Unit, a collaboration between the Hospital 12 de Octubre (H12O) and the National Cancer...
The cohort of 7000 rare diseases is complex, heterogeneous and dynamic. Newer conditions are being identified and reported regularly in the medical literature on a day to day basis. For a long time, the medical fraternity, researchers, and policymakers were unaware of the wide spectrum of rare diseases and the...
MONTREAL, Québec – Neurenati Therapeutics Inc., a biotechnology company dedicated to developing transformative therapies for rare pediatric diseases, is pleased to announce that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to NEU-001, a novel combination therapy leveraging a neurotrophic growth factor for the treatment of Hirschsprung...
Results of part 1 of the ESSENCE phase 3 trial, published in The New England Journal of Medicine, demonstrated that a greater proportion of patients with MASH achieved resolution of steatohepatitis with no worsening of liver fibrosis compared to placebo. Patients with MASH also saw statistically significant and superior improvement...
BEERSE, BELGIUM — Janssen-Cilag International NV, a Johnson & Johnson company, today announced that the European Commission (EC) has approved a Type II extension of indication for RYBREVANT®▼ (amivantamab) in combination with chemotherapy (carboplatin and pemetrexed), for the treatment of adults with advanced non-small cell lung cancer (NSCLC) with epidermal...
PARAMUS, N.J. — NS Pharma, Inc., a subsidiary of Nippon Shinyaku Co., Ltd, announced today that the European Commission (EC) has granted orphan drug designation to NS-229, which is being developed for the treatment of the rare disease eosinophilic granulomatosis with polyangiitis (EGPA). The orphan drug designation by the EC...