NICE provides “Final draft guidance” for Pompbiliti + Opfolda

NICE provides “Final draft guidance” for Pompbiliti + Opfolda

Posted On: 11/07/2023

An appraisal of the Amicus ERT, Pombiliti plus the chaperone pill, Opfolda took place on 3rd May 2023 where submissions from stakeholders, including patient experts and Pompe Support Network, were considered. The committee recommended the therapy be approved and final draft guidance was published. If there are no objections to the committee decision, then it is expected that Pombiliti + Opfolda will be given market approval by NICE in the next week or so.

The committee conclusion echoed the statements made by patients and patient groups:

“while there are 2 treatment options available for people with LOPD, the response to treatment is variable. Also, the response to ALGLU (Myozyme) typically wanes over time. So, alternative treatment options are welcome for people with Pompe disease, especially when there is clinical decline on current treatment.”

The Final draft guidance can be downloaded from the NICE website and describes information provided and the committee’s reasoning: