Alnylam shares rose following the FDA's approval of AMVUTTRA.

Alnylam Pharmaceuticals (NASDAQ: ALNY) shares rose 5% on Friday morning as trading resumed following the announcement of FDA approval for AMVUTTRA, a treatment for ATTR Amyloidosis. The FDA's approval is considered a significant advancement in the treatment of transthyretin-mediated amyloidosis (ATTR), affecting the heart and nerves. This drug is now the first and only FDA-approved treatment for both cardiomyopathy and polyneuropathy symptoms in adults with ATTR Amyloidosis. This news follows positive results from the HELIOS-B Phase 3 clinical trial, which demonstrated that AMVUTTRA significantly reduces the risk of all-cause mortality and recurrent cardiovascular events.

Alnylam's CEO, Yvonne Greenstreet, expressed gratitude to everyone involved in reaching this milestone and emphasized the company's commitment to providing innovative solutions for ATTR amyloidosis patients. Administered through quarterly subcutaneous injections, the treatment targets the root cause of the disease by reducing transthyretin (TTR) production. This approval is particularly noteworthy for a condition that can lead to early death due to irreversible damage caused by TTR fibril accumulation. The HELIOS-B study’s findings were well-received, achieving statistical significance across all primary and secondary endpoints. AMVUTTRA reduced the risk of all-cause mortality and recurrent cardiovascular events by 28% during a double-blind treatment period lasting up to 36 months.