Towards Healthcare

Alnylam Secures Dual FDA Approvals as siRNA Therapies Expand Beyond Liver

Alnylam wins FDA approvals for Qfitlia and Amvuttra, marking a major step in siRNA therapy. The biotech industry is now exploring new organ targets beyond the liver to enhance treatment options.

Author: Towards Healthcare Published Date: 10 June 2025
Share : linkedin twitter facebook

Alnylam’s Goal is to Expand Medication

Alnylam Secures Dual FDA Approvals as siRNA Therapies Expand Beyond Liver

Image Credits: Biospace

Announcement

Alnylam received two FDA approvals for Sanofi’s Qfitlia in hemophilia A and B, and Amvuttra in transthyretin-mediated amyloidosis. The approval is a part of Alnylam, along with other companies. Companies such as AbbVie, Eli Lilly, GSK, Ingelheim, and Boehringer are set to follow suit to develop therapies related to small interfering RNA (siRNA). It’s not only companies that expanding but also siRNA therapies approval for liver, initiating a two-way target. Alnylam and other pharmaceutical companies are now planning to target other organs to bolster medication and improve health safety.

Past success

The two years witnessed a billion-dollar positive difference in a bid and contributed to the fastest-growing field of the healthcare sector. For example, in January 2024, Boehringer Ingelheim introduced an agreement worth $2 billion with Chinese biotech Suzhou ribo life science and its interconnected Swedish unit Ribocure Pharmaceuticals to discover siRNA treatment for MASH or nonalcoholic. Last month, GSK initiated a $1.2 billion deal for Boston Pharmaceuticals' investigational FGF21.

Views and Statements

Analyst at William Blair, Myles Minter, follows Alnylam Pharmaceuticals' movements. He said the therapies have focused on the process of the liver, as well as Amvuttra. The Amvuttra focuses on cardiac outcomes to eliminate transthyretin in hepatic cells. The experts who were in conversation with Biospace noticed and acknowledged the change. The experts are also witnessing innovation and the efforts to deliver new tissues.

Additionally, Eli Lily’s group vice president and molecule discovery director of the Lilly Institute for Genetic Medicine, Andrew Adams, stated the current GaINAc has been a ‘unicorn’ for its specific and safe delivery. The company has a lot more work to do to develop an siRNA beyond the hepatocyte. Once the solution is developed, it will be similar to Alnylam medication and other GaINAc formulations initially developed.

Latest Insights