DMD research

Pfizer gene therapy for DMD doesn’t meet primary or secondary endpoints in Phase 3 clinical trial

Pharmaceutical company Pfizer has announced that the results of its CIFFREO trial of mini-dystrophin gene therapy fordadistrogene movaparvovec to treat Duchenne muscular dystrophy (DMD) show that it didn’t meet its primary or secondary endpoints.

What the trial was measuring

CIFFREO was a randomized, double-blind, placebo-controlled study Phase 3 trial in ambulatory (walking) boys aged four to seven years of age with DMD.

The primary endpoint in the study was improved motor function and assessed by a change in the North Star Ambulatory Assessment (NSAA) at one year after treatment. The key secondary endpoints included measuring the ten-meter run/walk velocity and time to rise from floor velocity.

Further update from Pfizer

Pfizer has said that it will share more detailed results from the study at upcoming medical and patient advocacy meetings in the next few weeks, and we will share those results with you as soon as we receive them.

Pfizer has also said that it will continue to closely monitor all participants enrolled in the study and is evaluating appropriate next steps for the programme.

Gene therapy in DMD

DMD gene therapy aims to deliver a working version of the dystrophin gene, so that the body can produce functioning dystrophin. The potential benefits of this include protecting the muscles and preventing the progression of the DMD.

Special webinar to discuss gene therapy to treat DMD

While gene therapy has brought a lot of hope for DMD, these results are very disappointing.

There is still a lot more that we need to understand about gene therapy as a treatment for DMD and how it can be optimised as a treatment.

We think this is a good time to bring the community together to discuss what has been learnt so far about gene therapy for DMD and what research is taking place to develop it. We are going to organize a webinar for the community, and you can email questions you’d like raised in it to [email protected].

Published on 13 June 2024

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