Alnylam Pharmaceuticals (ALNY) FDA Announcement summary

Introduction and purpose

• FDA approved Amvuttra (vutrisiran) for the treatment of cardiomyopathy of wild-type or hereditary ATTR amyloidosis in adults, expanding its previous indication for polyneuropathy and making it the first and only FDA-approved therapy for both conditions.

• Amvuttra is the first RNAi therapeutic to reduce cardiovascular death, hospitalizations, and urgent heart failure visits in adults with ATTR-CM.

• The approval is seen as a major milestone and inflection point, positioning Amvuttra as a potential new standard of care in ATTR cardiomyopathy.

Details of approval or decision

• Approval is based on the HELIOS-B Phase 3 clinical trial, which demonstrated significant reductions in all-cause mortality and recurrent cardiovascular events compared to placebo.

• The label expansion allows Amvuttra to reduce cardiovascular mortality, hospitalizations, and urgent heart failure visits in adults with ATTR-CM, with the label uniquely including urgent heart failure visits.

• Amvuttra is administered as a 25 mg subcutaneous injection every three months by healthcare professionals.

• No new safety issues were identified in the HELIOS-B study, and the label supports use as monotherapy or in combination with stabilizers.

Impact on industry and stakeholders

• Amvuttra's approval is expected to drive significant long-term revenue growth and establish a flagship franchise, with broad patient access and payer engagement strategies underway.

• ATTR-CM diagnoses have grown tenfold since 2019, with over 300,000 patients globally and significant unmet need; 80% remain undiagnosed.

• Extensive patient support programs, including Alnylam Assist, copay assistance, and financial aid, are in place to ensure access.

• Regulatory approvals in Japan, EU, UK, Brazil, and Canada are expected throughout 2025, with additional countries in 2026-2027.