Prothena Corporation PLC, a clinical-stage neuroscience company, today reported final results from the Phase 3 VITAL Amyloidosis study of NEOD001 (birtamimab) in newly diagnosed, treatment naive patients with AL amyloidosis and cardiac dysfunction (N=260), which was discontinued in 2018. The final hazard ratio (HR) for the composite primary endpoint (time to all-cause mortality or time …
Pfizer Inc. announced today that the U.S. Food and Drug Administration (FDA) has approved both VYNDAQEL® (tafamidis meglumine) and VYNDAMAX™ (tafamidis) for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. VYNDAQEL and VYNDAMAX are two oral formulations of the first-in-class transthyretin stabilizer …
Researchers at Boston Medical Center (BMC) and Boston University School of Medicine (BUSM), in collaboration with Columbia University Irving Medical Center (CUIMC), are leading a national, multi-site study aimed to achieve earlier diagnosis of transthyretin cardiac amyloidosis (ATTR-CM). The National Institutes of Health has awarded a five-year, $7.2M grant to fund the Screening for Cardiac …
National Study Seeks Earlier Diagnosis of ATTR Cardiac Amyloidosis in Minorities Read More »
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the Company has initiated APOLLO-B, a global Phase 3 placebo-controlled clinical trial of patisiran, an intravenously administered RNAi therapeutic, for the treatment of transthyretin amyloidosis (ATTR amyloidosis) with cardiomyopathy. The primary endpoint is the change from baseline in the 6-minute walk test …
Akcea Therapeutics, Inc. (NASDAQ: AKCA), an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), presented data today from the Phase 1 clinical trial of AKCEA-TTR-LRx in a poster presentation at the Heart Failure Society of America 23rd Annual Scientific Meeting in Philadelphia, Pennsylvania. AKCEA-TTR-LRx is an antisense drug developed using Ionis’ proprietary …
The Janssen Pharmaceutical Companies of Johnson & Johnson announced today the submission of a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) seeking approval of DARZALEX FASPRO™ (daratumumab and hyaluronidase-fihj), a subcutaneous formulation of daratumumab, for the treatment of patients with light chain (AL) amyloidosis, a rare and potentially fatal …
The treatment uses CRISPR/Cas9 editing technology, a technique derived from bacteria that allows researchers to edit the genome of a cell by specifying regions of DNA to be added, removed, or edited. NTLA-2001 is designed to eliminate the TTR gene from the DNA of liver cells, which would reduce TTR production significantly and prevent the …
UK Approves Phase 1 Trial of Potential Gene Editing Therapy, NTLA-2001 Read More »
Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today that the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, met its primary and both secondary endpoints at nine months in patients with hATTR amyloidosis with polyneuropathy. The primary endpoint was the change from …
The FDA has approved daratumumab and hyaluronidase-fihj (Darzalex Faspro), a subcutaneous formulation of daratumumab, for use in combination with bortezomib, cyclophosphamide, and dexamethasone (D-VCd; VCd) in the treatment of patients with newly diagnosed light-chain amyloidosis. Continued approval for this indication could be dependent upon verification and description of clinical benefit in a confirmatory trial, according …
People with hereditary transthyretin (ATTR) amyloidosis have a mutation in the transthyretin (TTR) gene, which means they produce an abnormal protein that gradually builds up in the heart and nerves. Symptoms include numbness in the hands and feet, loss of control of the bowel and bladder and immobility. The condition gets progressively worse and is …
Gene Editing Therapy Paves Way for Revolution in Treatment of Genetic Disorders Read More »