News & Meetings

Akcea Therapeutics UK Ltd., has announced that NICE has issued a positive Final Evaluation Document (FED) for Tegsedi™ (inotersen) for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). This decision will allow patients in England with this rare, inherited, severely debilitating and fatal disease to access …

Tegsedi™ to be Available on the NHS for hATTR Amyloidosis Read More »

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 …

Prothena Reports Results From the Phase 3 VITAL Amyloidosis Study of NEOD001 (Birtamimab) in AL Amyloidosis Read More »

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 …

U.S. FDA Approves VYNDAQEL® and VYNDAMAX™ for Use in Patients with Transthyretin Amyloid Cardiomyopathy, a Rare and Fatal Disease Read More »

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 …

Alnylam Pharmaceuticals Announces Initiation of APOLLO-B Phase 3 Study of Patisiran for the Treatment of Transthyretin Amyloidosis with Cardiomyopathy Read More »

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 …

Positive Phase 1 Results of AKCEA-TTR-LRx Presented at the Heart Failure Society of America Annual Meeting Read More »

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 …

Janssen Submits Application Seeking U.S. FDA Approval of DARZALEX FASPRO™ (Daratumumab and Hyaluronidase-fihj) for the Treatment of Patients With Light Chain (AL) Amyloidosis Read More »

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 …

Alnylam Reports Positive Topline Results From HELIOS-A Phase 3 Study of Vutrisiran in Patients With hATTR Amyloidosis With Polyneuropathy Read More »

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 …

FDA Approves Subcutaneous Daratumumab Plus VCd for Newly Diagnosed Light-Chain Amyloidosis Read More »