SAN DIEGO, Calif. – Of the 46 cancer drugs that the U.S. Food and Drug Administration (FDA) granted accelerated approval between 2013-2017, 63% were converted to regular approval even though only 43% demonstrated a clinical benefit in confirmatory trials after more than five years of follow-up, according to a study...
treatment News
CHICAGO, Illinois – The American Society for Transplantation and Cellular Therapy® (ASTCT®) supports the decision by the U.S. Food and Drug Administration’s (FDA) to grant multiple myeloma patients earlier access to CAR T-cell therapy Abecma as a second-line treatment and the CAR T therapy Carvykti as a third-line treatment. The...
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...
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...
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...
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...
YONGIN, South Korea — GC Biopharma Corp. announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to its intracerebroventricular (ICV) Enzyme Replacement Therapy (ERT) candidate, GC1130A, designed for Sanfilippo Syndrome type A (mucopolysaccharidosis type IIIA), developed in collaboration with Novel Pharma. GC1130A has previously achieved notable...