Summary
Alnylam Pharmaceuticals, Inc. (ALNY) announced on October 9, 2023, that it received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its supplemental New Drug Application (sNDA) for patisiran. The sNDA sought approval for patisiran, an RNA interference (RNAi) therapeutic, for the treatment of the cardiomyopathy associated with transthyretin-mediated (ATTR) amyloidosis. This development is a setback for Alnylam's efforts to expand the label of patisiran into this significant indication.
Key Highlights
- 1Alnylam Pharmaceuticals received a Complete Response Letter (CRL) from the FDA for its supplemental New Drug Application (sNDA) of patisiran.
- 2The sNDA was for the treatment of cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis.
- 3Patisiran is an investigational RNAi therapeutic.
- 4The CRL indicates that the FDA has not approved the supplemental application in its current form and has identified deficiencies that need to be addressed.
- 5This news directly impacts the potential market expansion for patisiran into a new, significant patient population.
Frequently Asked Questions
A Complete Response Letter (CRL) is issued by the U.S. Food and Drug Administration (FDA) when it has completed its review of a New Drug Application (NDA) or supplemental NDA and has determined that the application cannot be approved in its present form. The CRL outlines the deficiencies identified by the FDA and provides guidance on what the sponsor must do to address these issues before the application can be reconsidered for approval.
The CRL means that patisiran is not currently approved by the FDA for the treatment of ATTR amyloidosis cardiomyopathy. Alnylam will need to address the FDA's concerns, which could involve further clinical trials, additional data analysis, or other actions. This will likely delay the commercialization of patisiran for this specific indication and could potentially impact its market potential if significant hurdles remain.
ATTR amyloidosis cardiomyopathy is a serious and progressive condition characterized by the buildup of amyloid protein (specifically transthyretin) in the heart muscle, leading to stiffening, thickened walls, and impaired function of the heart. This can result in heart failure and other cardiovascular complications.
Patisiran (marketed as ONPZYO) is an RNA interference (RNAi) therapeutic developed by Alnylam Pharmaceuticals. It works by silencing the TTR gene in the liver, thereby reducing the production of the abnormal TTR protein that forms amyloid deposits in various tissues, including the heart and nerves.