June 16, 2024

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FDA approves Alnylam’s RNAi therapy Amvuttra

FDA approves Alnylam’s RNAi therapy Amvuttra


FDA approves Alnylam’s RNAi therapy Amvuttra.

On June 13, Alnylam announced that the FDA approved the company’s RNAi therapy Amvuttra (vutrisiran) for the treatment of adults with hereditary transthyretin-mediated (hATTR) amyloidosis polyneuropathy.

This is the first FDA-approved RNAi therapy to reverse neuropathic damage with just subcutaneous injections every 3 months.


FDA approves Alnylam's RNAi therapy Amvuttra


Amvuttra is also the fourth RNAi therapy approved by Alnylam. Its first RNAi therapy, patisiran, was approved by the FDA in 2018 for the treatment of hATTR amyloidosis polyneuropathy, but requires an intravenous infusion every 3 weeks.


hATTR is a rare autosomal dominant genetic disease, the cause of which is amyloidosis caused by abnormal TTR protein deposition in multiple tissues and organs due to mutations in the transthyretin gene.

Progressive neuropathy and cardiomyopathy are the main features of the disease.

There is an unmet need for the treatment of hATTR disease with approximately 50,000 patients worldwide. Median survival after diagnosis was 4.7 years, and survival was even lower in patients with features of cardiomyopathy (3.4 years).


Amvuttra is a double-stranded small interfering RNA (siRNA) that targets mutant and wild-type transthyretin (TTR) messenger RNA (mRNA).

Amvuttra uses Alnylam’s Enhanced Stability Chemistry (ESC)-GalNAc conjugate delivery platform with increased potency and high metabolic stability.


FDA approval is based on positive 9-month results from a global, randomized, open-label, multicenter Phase III HELIOS-A study evaluating Amvuttra in different groups of patients with hATTR amyloidosis polyneuropathy and security.


164 patients were randomized 3:1 to receive vutrisiran (25mg, N=122) or patisiran (0.3mg/kg, N=42) for 18 months.

The efficacy of Amvuttra was assessed by comparing the Amvuttra arm in the HELIOS-A study with the placebo arm (N=77) in the Phase III APOLLO study.


Amvuttra met the primary endpoint of the study, with a mean reduction (improvement) of 2.2 points from baseline in the Amvuttra group (N=114) compared to baseline in the Amvuttra group (N=114) compared to baseline in the Amvuttra group (N=67 ) increased (worsened) by a mean of 14.8 points, and the mean difference between Amvuttra relative to placebo was 17.0 points (p<0.0001); Amvuttra significantly improved signs and symptoms of polyneuropathy at month 9 in more than 50% of patients cessation or reversal of disease manifestations.


Amvuttra also met all secondary endpoints of the study at 9 months, with efficacy results at month 18 consistent with the data at month 9.


Amvuttra was safe and well tolerated, with no drug-related discontinuations or deaths. The most frequently reported adverse events (AEs) in patients receiving Amvuttra included arthralgia (11%), dyspnea (7%) and decreased vitamin A (7%). Injection site reactions (ISRs) were reported in 5 patients (4%), all of which were mild and transient.


During the randomized treatment extension (RTE) phase of the ongoing HELIOS-A trial, Alnylam is evaluating the therapeutic efficacy of the twice-yearly 50 mg Amvuttra dosing regimen.

In addition, the Phase III HELIOS-B study is evaluating Amvuttra in patients with ATTR amyloid cardiomyopathy, including hATTR and wild-type ATTR (wtATTR) amyloidosis.









subcutaneous injection every 3 months! FDA approves Alnylam’s 4th RNAi therapy

FDA approves Alnylam’s RNAi therapy Amvuttra

(source:internet, reference only)

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