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ALNYLAM PHARMACEUTICALS, INC. 8-K Report, Corporate Update (Aug 10, 2018)

Filed August 10, 2018For Securities:ALNY

Summary

Alnylam Pharmaceuticals, Inc. announced on August 10, 2018, a significant milestone with the U.S. Food and Drug Administration's (FDA) approval of its new drug application for ONPATTRO™ (patisiran) lipid complex injection. This approval marks the first-ever FDA-approved treatment for the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults, a rare and debilitating genetic disease. This approval represents a major commercial opportunity for Alnylam, validating its RNA interference (RNAi) therapeutic platform and opening the door for potential future pipeline advancements. Investors should closely monitor ONPATTRO's market adoption, physician and patient uptake, and the company's commercialization strategy as it transitions to a commercial-stage biopharmaceutical company.

Key Highlights

  • 1FDA approval of ONPATTRO™ (patisiran) for the treatment of hATTR amyloidosis polyneuropathy in adults.
  • 2ONPATTRO™ is the first FDA-approved therapy for hATTR amyloidosis.
  • 3This approval validates Alnylam's RNA interference (RNAi) therapeutic platform.
  • 4The drug is administered as a lipid complex injection.
  • 5The approval signifies Alnylam's transition to a commercial-stage company.
  • 6The press release detailing the approval is attached as Exhibit 99.1.

Frequently Asked Questions

ONPATTRO™ (patisiran) is approved for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.

This approval is significant because ONPATTRO™ is the first-ever therapy approved by the FDA to treat hATTR amyloidosis, addressing a critical unmet medical need for patients with this rare and progressive disease.

This approval represents a major commercial inflection point for Alnylam, validating its novel RNA interference (RNAi) platform and marking its transition from a development-stage company to a commercial-stage biopharmaceutical company with a flagship product.

Hereditary transthyretin-mediated (hATTR) amyloidosis is a rare, progressive, and debilitating genetic disease caused by mutations in the transthyretin (TTR) gene. These mutations lead to the misfolding and deposition of TTR protein as amyloid fibrils in various tissues and organs, most notably the peripheral nerves and heart, causing debilitating symptoms and organ damage.