The IFOPA is pleased to share the letter (linked below) to the FOP community from Regeneron Pharmaceuticals providing an update on the LUMINA-1 trial which was placed on clinical hold by the FDA in October 2020.
Regeneron is announcing that the Phase II LUMINA-1 safety and efficacy trial will be closing and that a new Phase III trial of the drug garetosmab is being planned for launch in 2022. The details of that trial, including the inclusion/exclusion criteria and clinical trial sites, are still under development. A Phase III trial is the next step in the drug development and regulatory review process.
As you may remember, a decision was made to place the trial on clinical hold based on reports of fatal serious adverse events (SAEs) in the trial during the open-label portion in which all participants took the drug. As shared in their letter on June 4, stopping the study to evaluate safety and efficacy was the top priority for Regeneron. During this process, Regeneron met with the US Food & Drug Administration (FDA) about the safety and efficacy of garetosmab. It is standard practice to pause clinical trials when SAEs occur to fully evaluate their connection or lack of connection to the study drug.
Safety is the highest priority of the FDA and other regulatory agencies around the globe, which is why drugs are first tested in patient cells in laboratory dishes, more than one species of animal model, healthy human Phase I trials without patients, and then brought to Phase II of safety and efficacy trials in patients with the disease.
The IFOPA views this as encouraging news because it shows that the FDA is willing to continue exploring the potential of garetosmab as a treatment for FOP.
Regeneron is committed to sharing scientific data and is currently preparing a publication of LUMINA-1 results to submit to a peer-reviewed scientific journal.
If you were a participant in the LUMINA-1 trial and have additional questions, please contact your Study Coordinator.
Read the community letter from Regeneron leadership