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.
(Reuters) -Alnylam Pharmaceuticals said on Monday it would not pursue expanded use of its drug to treat a potentially fatal heart disease in the U.S. after the Food and Drug Administration declined to approve the medication despite a favorable recommendation from its advisers. The regulator, in its so-called complete response letter, said the drug patisiran did not meaningfully improve the condition of patients with heart muscle issues or cardiomyopathy caused by ATTR amyloidosis, characterized by abnormal deposits of transthyretin proteins in organs and tissues. Patisiran, also branded as Onpattro, is already approved in the U.S. to treat nerve damage in adult patients with hereditary ATTR amyloidosis.
By Colin Kellaher The U.S. Food and Drug Administration has rejected Alnylam Pharmaceuticals request for expanded approval of Onpattro for transthyretin-mediated amyloidosis with.
Alnylam Pharmaceuticals’ stock price fell after FDA advisers weigh the risks and benefits of the company’s ATTR-CM treatment; Digital health startup Akili cuts staff by 40%, abandons its prescription model; Florida surgeon general advises residents under 65 not to get new COVID boosters.
(Reuters) - An outside panel of experts to the U.S. Food and Drug Administration on Wednesday backed the expanded use of Alnylam Pharmaceuticals' gene.