Posted by Michael Wonder on 17 Jan 2023
Vutrisiran in hereditary transthyretin amyloidosis: hint of minor added benefit
17 January 2023 - Vutrisiran is approved for the treatment of patients with hereditary transthyretin amyloidosis with stage 1 or 2 polyneuropathy.
Like all active ingredients for rare diseases, sales below the statutory minimum sales threshold of 50 or recently €30 million per year, vutrisiran would not have had to go through a regular early benefit assessment. However, the manufacturer decided to initiate a benefit assessment procedure now. This option was introduced in 2019 with the Law for More Safety in Drug Supply.
