The Phase II LUMINA 1 trial assessed the safety and efficacy of garetosmab, a monoclonal antibody that blocks activin A signaling pathways, in 44 adults with fibrodysplasia ossificans progressiva (FOP).
- The number and volume of new heterotopic ossifications, as well as that of soft-tissue inflammatory flare-ups, were significantly reduced on garetosmab compared with placebo.
- During the 76-week open-label period, there were no new heterotopic ossifications.
- Thirty-four participants elected to continue treatment at the end of the open-label extension.
- All participants on garetosmab experienced at least one treatment-emergent side effect, including epistaxis, eyelash and/or eyebrow loss and skin abscesses.
- Five out of 44 participants died during the open-label phase, but this could not be linked to garetosmab.
In order to better define the benefit-risk profile of garestomab in FOP, the OPTIMA phase III trial is currently underway, including in France (Montpellier, Paris).
