FDA Declines Expanded Use of Alnylam Pharmaceuticals’ Drug for Rare Heart Disease

In a recent announcement, the U.S. Food and Drug Administration (FDA) has chosen not to approve the expanded use of Alnylam Pharmaceuticals’ drug, patisiran, for the treatment of a rare and fatal heart disease. The FDA cited insufficient evidence of clinical meaningfulness as the reason for their decision.

Alnylam Pharmaceuticals had sought regulatory approval for the drug to treat adult patients with cardiomyopathy, a type of heart-muscle disease caused by wild-type or hereditary transthyretin-mediated amyloidosis. This condition leads to the accumulation of faulty transthyretin proteins in organs and tissues, resulting in a range of health issues such as heart, eye, kidney, and thyroid diseases.

While the FDA did not identify any concerns regarding clinical safety, study conduct, drug quality, or manufacturing, they stated that the clinical meaningfulness of patisiran had not been established. As a result, Alnylam Pharmaceuticals has decided to no longer pursue an expanded indication for the drug in the United States.

Despite this setback, Alnylam Pharmaceuticals remains committed to advancing the treatment options for patients with rare diseases. The company will continue to explore other avenues and potential indications for patisiran.

It is important to note that the FDA’s decision does not impact the current approved use of patisiran for the treatment of hereditary transthyretin-mediated amyloidosis in patients with polyneuropathy.

Alnylam Pharmaceuticals and the FDA will continue to work together to address the concerns raised and explore further clinical evidence to potentially support the expanded use of patisiran in the future.

While this news may be disappointing for patients and healthcare professionals, it underscores the rigorous evaluation process that the FDA follows to ensure the safety and effectiveness of new treatments. The FDA’s commitment to evidence-based decision-making is crucial in maintaining public trust and safeguarding patient health.

As research and development in the field of rare diseases continue to progress, it is hoped that innovative treatments like patisiran will eventually receive the necessary approvals to provide relief and improved outcomes for patients in need.