Alnylam’s Amvuttra Approved for ATTR Cardiomyopathy, Challenging Pfizer and BridgeBio
The FDA approved Alnylam's Amvuttra (vutrisiran) for the treatment of cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults136.
Amvuttra is now the first and only therapeutic approved by the FDA for both ATTR-CM and polyneuropathy of hereditary ATTR amyloidosis (hATTR-PN)16.
It is the first RNAi therapeutic approved to reduce cardiovascular death, hospitalizations and urgent heart failure visits in ATTR-CM patients6.
In clinical trials, Amvuttra reduced the risk of all-cause mortality and recurrent cardiovascular events by 28% compared to placebo46.
Amvuttra works by knocking down both wild-type and mutant transthyretin RNA, lowering TTR protein levels14.
This approval sets up a three-way competition with Pfizer's tafamidis and BridgeBio's Attruby in the ATTR-CM market18.
Alnylam hopes Amvuttra's novel mechanism of action and convenient dosing (4 subcutaneous doses per year) will differentiate it from competitors145.
The ATTR-CM market is projected to reach over $10 billion by 203048.
Sources:
1. https://www.biospace.com/drug-development/alynlams-amvuttra-approved-as-first-rnai-silencer-for-rare-type-of-cardiomyopathy
3. https://arci.org/amvuttra-attr-cm-approval/
4. https://www.insideprecisionmedicine.com/topics/precision-medicine/alnylams-amvuttra-snags-approval-for-rare-heart-disease/
5. https://www.biopharmadive.com/news/alnylam-vutrisiran-fda-approval-ttr-amyloidosis-cardiomyopathy/742975/
6. https://investors.alnylam.com/press-release?id=28831
8. https://www.pharmaceutical-technology.com/news/alnylam-challenges-pfizer-and-bridgebio-with-fda-nod-for-attr-cm-drug/