(Reuters) -The U.S. Food and Drug Administration has declined to approve the expanded use of Alnylam Pharmaceuticals’ drug to treat a rare and fatal heart disease, citing insufficient evidence of clinical meaningfulness, the company said on Monday.
The FDA, in its so-called complete response letter, said the clinical meaningfulness of the drug, patisiran, had not been established, adding that it did not identify any issues with respect to clinical safety, study conduct, drug quality or manufacturing.
Alnylam said it will no longer pursue an expanded indication for patisiran in the United States.
The company was seeking regulatory nod for the drug to treat adult patients with cardiomyopathy, a type of heart-muscle disease caused by wild-type or hereditary transthyretin-mediated amyloidosis.
Also known as ATTR amyloidosis, the disease leads to the accumulation of faulty transthyretin proteins in organs and tissues. This can cause a loss of sensation and heart, eye, kidney and thyroid diseases.
(Reporting by Mariam Sunny in Bengaluru; Editing by Pooja Desai)
