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.
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.
Fast Track designation by the FDA for the treatment of the hATTR -related polyneuropathy in adults
Submitted to the EMA
April 14, 2022:
2022: Europe and United States
Patents estimated to expire beginning in 2037
How will vutrisiran impact the market for ATTR polyneuropathy?
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.
“If I am giving Vutrisiran every three months, that would be perfect. I’m sure it would beat all the other therapies.”
– Source: Cortellis Competitive Intelligence, Drug Timeline & Success Rate prediction current as of December 15, 2021