Alnylam Pharmaceuticals Inc.’s decision to change its analysis plan for the phase III Helios-B trial of RNAi therapeutic Amvuttra (vutrisiran) to treat transthyretin amyloidosis with cardiomyopathy, pushing top-line results back by three months, pressured its share price (NASDAQ:ALNY) down by 10% on Feb. 15, while also boosting shares of competitor Bridgebio Pharma Inc. (NASDAQ:BBIO) by 14%.
Armed with strong phase III safety data in Japanese patients, Palo Alto, Calif.-based Bridgebio Pharma Inc. is planning to file for Japan approval of its investigational drug acoramidis for a rare heart disorder.
Armed with strong phase III safety data in Japanese patients, Palo Alto, Calif.-based Bridgebio Pharma Inc. is planning to file for Japan approval of its investigational drug acoramidis for a rare heart disorder.
A new self-injectable therapy for polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) will be available in January 2024 now that the U.S. FDA has approved Ionis Pharmaceuticals Inc.’s Wainua (eplontersen), a ligand-conjugated antisense oligonucleotide.
Following an unexpected FDA complete response letter (CRL), Alnylam Pharmaceuticals Inc. said it will no longer pursue an expanded indication for Onpattro (patisiran) in the U.S. The RNAi therapeutic was approved in 2018 to treat polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis and seemed well on its way to snagging a second U.S. indication after the FDA’s Cardiovascular and Renal Drugs Advisory Committee voted 9-3 in September that the drug’s benefits outweighed its risks as a treatment for cardiomyopathy of ATTR amyloidosis. The FDA disagreed with the committee, saying in the CRL that patisiran’s clinical meaningfulness had not been established in the proposed indication.
A U.S. FDA advisory committee’s backing keeps Alnylam Pharmaceuticals Inc.’s Onpattro (patisiran) on the road to a supplemental approval in treating a rare heart disease, but it couldn’t stop the company stock from sliding. Shares (NASDAQ:ALNY) closed Sept. 14 down 8.8% at $193.06, the day after the Cardiovascular and Renal Drugs Advisory Committee voted 9-3 that patisiran’s benefits outweigh the risks in treating cardiomyopathy of transthyretin-mediated amyloidosis.
With Alnylam Pharmaceuticals Inc.’s FDA clearance for Amvuttra (vutrisiran) in the rearview mirror, investors are looking ahead to potentially label-widening phase III data related to another, already approved drug: Onpattro (patisiran).
NHS England has announced a new £340 million (US$481 million) Innovative Medicines Fund, as the country grapples with the issue of bringing ultra-pricey specialist medicines to patients on the country’s taxpayer-funded health care system.
Alnylam Pharmaceuticals Inc. said the 164-patient Helios-A phase III study with next-generation RNAi drug vutrisiran hit its primary endpoint as well as both secondary goals in the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy. The primary endpoint was change from baseline in the modified Neuropathy Impairment Score at nine months as compared to historical placebo data from the Apollo phase III study of Alnylam’s Onpattro, cleared by the FDA for ATTR polyneuropathy in August 2018.
Intellia Therapeutics Inc. is looking to disrupt the transthyretin (TTR) amyloidosis (ATTR) market with NTLA-2001, its CRISPR-based treatment designed to be a potential cure for the disease. The drug, which is delivered via a lipid nanoparticle, edits the patient's DNA in vivo to create a stop codon and eliminate the expression of TTR, the protein that aggregates in ATTR patients' nervous systems and hearts, disrupting their functions.
