Taldefgrobep Alfa (Biohaven) RESILIENT Trial Results and Updates
Taldefgrobep Alfa (Biohaven) RESILIENT Trial Results and Updates
Page last updated: 14th March 2023
Last updated: 14th March 2023
14th March 2023: Biohaven Pharmaceuticals announced that they received Fast Track designation from the U.S. Food and Drug Administration (FDA) for Taldefgrobep alfa.
Fast Track status is granted to drugs that show considerable potential for being able to treat life-threatening diseases, such as SMA. The designation means that the development and review processes of the promising drug can be streamlined in order to speed up translation into the clinic.
25th January 2023: The clinical-stage pharmaceutical company, Biohaven Pharmaceuticals, is recruiting participants in the USA for this Phase 3 trial. It will be randomized, double-blind and placebo-controlled. RESILIENT has been designed to test the efficacy and safety of weekly injections of taldefgrobep alfa given beneath the skin (“subcutaneous”) for 48 weeks.
The trial will be performed with ambulatory and non-ambulatory participants with SMA, aged 4 to 21 years. All enrolees will have all been treated with an SMA disease-modifying therapy (nusinersen, risdiplam and / or onasemnogene abeparvovec) and are anticipated to remain on that same treatment regimen and dose throughout the trial of taldefgrobep alfa.
It is expected that the trial will be completed in early 2025.
Proposed Trial Sites in the UK:
| Resilient Trial of Taldefrobep Alfa | ||
|---|---|---|
| All applicants must be:
-receiving risdiplam or nusinersen or have received Zolgensma more than 2 years ago -ambulant or non-ambulant -age 4-21 years |
||
| Trial Centre | Current status / Contact | Cut-off date |
| Glasgow | Approved by Pharma company. |
November 2023 |
| Oxford | ||
| Manchester | ||