FDA approves oral palovarotene as first treatment for rare bone disease
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Key takeaways:
- Oral palovarotene received FDA approval for the treatment of fibrodysplasia ossificans progressiva.
- The medication reduced heterotopic ossification volume by 54% in a phase 3 trial.
The FDA approved palovarotene capsules as the first treatment for people with the rare bone disease fibrodysplasia ossificans progressiva, according to an industry press release.
Palovarotene (Sohonos, Ipsen) is indicated for the treatment of fibrodysplasia ossificans progressiva in adult and pediatric patients (girls aged 8 years and older and boys aged 10 years and older). Palovarotene is an oral capsule with particular selectivity for the gamma subtype of retinoic-acid receptors, which are an important regulator of skeletal development and ectopic bone in the retinoid signaling pathway, according to the release. The medication is designed to mediate the interactions within the retinoid signaling pathway to reduce new abnormal bone formation.
In findings from the phase 3 MOVE trial, researchers compared 18-month data from 107 people with fibrodysplasia ossificans progressiva receiving palovarotene with a group of untreated people from Ipsen’s fibrodysplasia ossificans progressiva natural history study. Adults receiving palovarotene had a 54% greater reduction in annualized heterotopic ossification volume compared with no treatment. Adverse events for palovarotene were consistent with other systemic retinoid class medications. The most common adverse events were mucocutaneous events, such as dry skin, lip dryness, alopecia, drug eruption, rash and pruritus, and musculoskeletal events, such as arthralgia and premature growth plate closure for growing children, according to the release.
“As a clinician caring for patients with fibrodysplasia ossificans progressiva, I personally see the daily challenges and stresses that our patients and their families must contend with,” Edward Hsiao, MD, PhD, professor of medicine in the division of endocrinology and metabolism at University of California, San Francisco, said in the release. “The published phase 3 MOVE study showed that Sohonos can decrease new heterotopic ossification, and that palovarotene can be tolerated by many patients with fibrodysplasia ossificans progressiva. Sohonos is not for everyone. As with all medicines there are risks in this case especially for young children who may develop early growth plate closure. In addition, Sohonos has the same side effects as other retinoids, including dryness of the skin and mucus membranes. However, since the accumulation of heterotopic ossification in fibrodysplasia ossificans progressiva is progressive, irreversible, and life altering, this medication is an important treatment option for our fibrodysplasia ossificans progressiva community.”
Palovarotene will be available immediately in the U.S., according to the release. People with fibrodysplasia ossificans progressiva and their caregivers can call 1-866-435-5677 to receive educational support and ask questions about coverage, access and reimbursement for the medication.
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