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Alnylam Pharmaceuticals today announced the European Commission's (EC) approval of its RNAi therapy Amvuttra (vutrisiran), which can treat stage 1 and 2 polyneuropathy in adults with hereditary transthyroxine-mediated (hATTR) amyloidosis at a frequency of every 3 months
.

This approval is based on a recommendation
from the Human Medicines Commission (CHMP) in July this year.

This is also Alnylam's second approved RNAi therapy for amyloidosis in
hATTR.

hATTR amyloidosis is an inherited, progressive debilitating and lethal disease caused
by mutations in the TTR gene.

TTR proteins are mainly produced in the liver and are normally carriers
of vitamin A.

TTR gene variants lead to abnormal amyloid accumulation and damage to body organs and tissues such as peripheral nerves and heart, leading to refractory peripheral sensory-motor neuropathy, autonomic neuropathy and/or cardiomyopathy, and other manifestations of
the disease.

amyloidosis of hATTR has significant unmet medical needs, with a median survival of only 4.
7 years
after diagnosis.

Amvuttra is an investigational, subcutaneously administered RNAi therapeutic drug developed to treat ATTR amyloidosis, including hATTR and wild-type ATTR (wtATTR) amyloidosis
.

Blocks the production of wild-type and variant transthyroxine protein (TTR) by targeting and silencing specific mRNAs
.

Through Alnylam's Enhanced Stable Chemical (ESC)-GalNAc Coupled Delivery Platform design, Amvutra increases potency and metabolic stability, allowing subcutaneous injections at a quarterly frequency and has the potential
to be reduced to semi-annual injections.

Amvuttra is qualified as an orphan drug in the European Union and the United States for the treatment of transthyroxine-mediated amyloidosis, and in June 2022, it was approved by the US FDA for the treatment of multiple neuropathy in adults with hATTR amyloidosis
.

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