Tag Archive for: Alnylam Pharmaceuticals

Following the regulator’s denial of patisiran’s label expansion, Alnylam has published late-stage data for the RNAi therapeutic in The New England Journal of Medicine demonstrating its efficacy in ATTR-cardiomyopathy.

The FDA on Monday denied approval of Alnylam Pharmaceuticals’ siRNA therapy Onpattro (patisiran) for the treatment of cardiomyopathy of transthyretin-mediated amyloidosis.

The Cardiovascular and Renal Drugs Advisory Committee voted 9-3 in favor of Alnylam’s patisiran on whether its benefits outweigh its risks for patients with cardiomyopathy induced by transthyretin amyloidosis.

This week, GSK is expecting a verdict for its myelofibrosis candidate momelotinib and Alnylam will make the case for patisiran in cardiomyopathy of ATTR amyloidosis.

Alnylam Pharmaceuticals is already making progress on its July 2023 deal with Roche to co-develop and commercialize zilebesiran, the former’s investigational RNAi therapeutic. The Cambridge biotech reported positive topline Phase II results Thursday for its partnered gene-silencing therapy to treat hypertension. 

The trial data reinforces the potential for zilebesiran to offer “new possibilities in a field of medicine that has seen limited innovation in nearly 20 years,” said Simon Fox, program lead for the drug’s development.

With a potential $2.9 billion on the line, Ionis Pharmaceuticals and AstraZeneca announced new results from the Phase III study of eplontersen in hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). The results will be presented Monday at the American Academy of Neurology (AAN) 2023 Annual Meeting in Boston.

Biopharma rallies behind FDA after Texas abortion pill ruling Published: Apr 11, 2023 By Tristan Manalac BioSpace In an open letter published Monday, more than 480 biopharma industry leaders, including […]

The agency’s top five represent an eclectic mix of cancer, cardiovascular and rare disease drugs.

Sarepta Therapeutics and Alnylam Pharmaceuticals have announced the pricing of $1 billion and $900 million in convertible senior notes respectively.