For a progressive disease that has a lot of unmet need, this drug brings efficacy, a generally favorable safety profile and improvements in delivery that will provide benefits for patient quality of life.

About vutrisiran

  1. Alnylam® Pharmaceuticals
  2. siRNA transthyretin (TTR) gene inhibitor delivered using a GalNAc-conjugate delivery platform
  3. Every-three-month SC administration for treatment of ATTR polyneuropathy
  4. Two subtypes of ATTR:
    • Hereditary ATTR (hATTR):
    1,233 people in the United States in 2021
    • Wild-type ATTR:
    1,300 people in the United States in 2021

Why is it a drug to watch?

This patient population, especially with wild-type ATTR, has few treatment options. Vutrisiran becoming reimbursable for wild-type ATTR polyneuropathy would be a huge win. It also has more convenient dosing than other ATTR-specific drugs.

  1. Phase 3 HELIOS trials are underway.
    • Results to date have shown improvements in polyneuropathy, quality of life and cardiac biomarkers.
  2. Vutrisiran demonstrated statistically significant efficacy on the same key endpoints for hATTR polyneuropathy as ONPATTRO® (patisiran).
  3. Compared with ONPATTRO, it has
    • a more convenient administration method (SC vs one-hour IV infusion) and
    • less frequent dosing (every three months vs weekly or every three weeks).
  4. It has demonstrated good tolerability and an encouraging safety profile.

Review and approval status

May 2018:
Orphan Drug designation by FDA and EMA for the treatment of ATTR

May 2020:
Fast Track designation by the FDA for the treatment of the hATTR -related polyneuropathy in adults

September 2021:
Submitted to the EMA

April 14, 2022:
PDUFA date

Expected launch:
2022: Europe and United States

Patents estimated to expire beginning in 2037

How will vutrisiran impact the market for ATTR polyneuropathy?

  1. Market is currently composed of therapies approved for other indications (e.g., multiple myeloma) that are used off-label to treat the effects of amyloid build-up in nerves and major organs (e.g., the heart and liver).
  2. Treatment also includes chemotherapy and, as a last resort, organ transplant.
  3. For hereditary ATTR polyneuropathy, there are two approved drugs:
    – ONPATTRO, administered intravenously every three weeks and
    – TEGSEDI® (inotersen), administered via SC injection once a week.
  4. For wild-type ATTR amyloidosis polyneuropathy, there are no approved treatments.

What gaps in treatment does vutrisiran fill?

ATTR is a rare, undiagnosed, rapidly progressive, debilitating and fatal disease with very few effective treatment options. Vutrisiran represents another drug in the therapeutic arsenal with a less-frequent dosing schedule and relatively convenient administration method (SC vs IV infusion). With its MOA, vutrisiran should help slow, if not stop, the accumulation of amyloid throughout the body. It has generally mild side effects, compared with TEGSEDI and standard chemotherapy.

What hurdles might it need to overcome to reach blockbuster status?

Vutrisiran might face some competition from ONPATTRO and TEGSEDI for patients with hATTR polyneuropathy, especially with the limited patient population and if it enters the market at a high price.

Expected sales in 2026
probability of success for
vutrisiran in the United States.
Source: Cortellis Competitive Intelligence, Drug Timeline & Success Rates Prediction current as of December 15, 2021

Drug Timeline & Success Rates

– Source: Cortellis Competitive Intelligence, Drug Timeline & Success Rate prediction current as of December 15, 2021

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