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...
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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...
Los Angeles, CA – The gene SYNGAP1, the variants of which are top risk factors for Autism Apectrum Disorder (ASD), has previously unappreciated effects on the developing brain, according to a new study published in Nature Neuroscience. The study shows how disease-causing variants of SYNGAP1, thought primarily to affect synapses between mature neurons, could disrupt...
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...
Boston — 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, recognizes the first annual Barth Syndrome Awareness Day. Barth syndrome is an ultra-rare, life-threatening, genetic disease primarily affecting males....
Boston — 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, recognizes the first annual Barth Syndrome Awareness Day. Barth syndrome is an ultra-rare, life-threatening, genetic disease primarily affecting males....
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...
SAN FRANCISCO and SUZHOU – Innovent Biologics, Inc. (Innovent) (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of cancer, metabolic, autoimmune and other major diseases, and IASO Biotherapeutics (IASO Bio), today jointly announce that the Center for Drug Evaluation (CDE) of China’s National Medical Products...
Baltimore, Maryland – The Bespoke Gene Therapy Consortium, a public-private collaboration involving the National Institutes of Health, FDA, industry and patient groups intended to help accelerate the delivery of AAV-based gene therapies for rare diseases, is making slow but steady progress, a representative said at the 2024 American Society of...
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...