FDA approves Atebrioz tablets for fibrodysplasia ossificans progressiva
Key takeaways:
- The tablets have been approved for use in patients aged 12 years or older.
- This is the third approved treatment for the rare genetic disease.
Editor’s note: This is a developing story. Please check back soon for updates.
The FDA approved zilurgisertib tablets for the reduction of heterotopic ossification among patients aged 12 years or older with fibrodysplasia ossificans progressiva, according to a press release from the organization.
This is the third approved treatment for fibrodysplasia ossificans progressiva (FOP), alongside Garetosmab-grts (Pasatru, Regeneron) and palovarotene (Sohonos, Ipsen), as Healio previously reported.
The indication is supported by positive efficacy data from a randomized, double-blind, placebocontrolled trial, which randomly assigned 63 patients with FOP to treatment with 100 mg zilurgisertib (Atebrioz, Mirum Pharmaceuticals) or placebo. According to the release, patients were treated once daily for 24 weeks followed by a single-arm, open-label extension period during which patients received zilurgisertib 100 mg daily for an additional 292 weeks.
The study utili...