FDA approves palovarotene capsules to treat fibrodysplasia ossificans progressiva
Palovarotene is now the first and only treatment for the ultra-rare bone disease fibrodysplasia ossificans progressive (FOP), which impacts approximately 400 people in the United States and 900 globally.
FDA approves palovarotene capsules to treat fibrodysplasia ossificans progressiva | Image Credit: © Calin - © Calin - stock.adobe.com.
The FDA has approved palovarotene (Sohonos; Ipsen) capsules for adult and pediatric patients aged 8 years and up for females and 10 years and up for males with the ultra-rare bone disease fibrodysplasia ossificans progressive (FOP), making it the first and only treatment for the disease. The capsules are indicated for a reduction in volume of new heterotopic ossification (HO) in these indications. Eligible patients can be prescribed palovarotene immediately in the United States, according to a press release from Ipsen Pharma.
Impacting approximately 400 people in the United States and 900 globally, FOP leads to progressive mobility loss, limits quality of life, and shortens median life expectancy to 56 years. Rapid bone growth is the result of the disease continually progressing with flare-up episodes. Function and mobility are severely restricted because of HO.
Individuals living with FOP “inevitably lose the ability to eat and drink on their own, cannot provide selfcare or use the restroom themselves,” Ipsen stated in the press release. Most people with FOP require a wheelchair and full-time assistance by the age of 30. Death can occur as a result of bone formation around the ribcage, leading to breathing issues and cardiorespiratory failure. Fractures or head injuries resulting from falls can occur because of joint ankylosis that prevents the ability to brace from a fall.
Efficacy and safety data demonstrated in the phase 3 MOVE trial (NCT03312634) was the basis for the FDA approval, which was granted Priority Review of the New Drug Application (NDA). The treatment received Orphan Drug and Breakthrough Therapy designations from the FDA and was also issued a Rare Pediatric Disease Priority Review Voucher (used for subsequent drug applications that do not qualify for priority review).
Published in the Journal of Bone and Mineral Research, 18-month data featured 107 individuals with FOP. They received oral palovarotene, compared with untreated individuals from Ipsen’s global FOP Natural History Study (NCT02322255). Weighted with a linear mixed effect model, the trial demonstrated a 54% reduction of annualized HO volume for palovarotene-treated patients compared with no treatment (beyond standard of care).
A well-characterized safety profile was also demonstrated, and adverse events were consistent with the systemic retinoid class. Lip dryness, dry skin, drug eruption, rash, alopecia, and pruritus and musculoskeletal events such as arthralgia and premature growth plate closure in growing children were the most common treatment emergent adverse reactions reported (≥ 10%).
According to Ipsen, palovarotene is contraindicated in patients amid pregnancy, or those with a history of allergy or hypersensitivity to retinoids, or any component of the treatment. Click here for more information on use in specific populations.
Reference:
US FDA approves Ipsen’s Sohonos (palovarotene) capsules, the first and only treatment for people with fibrodysplasia ossificans progressiva. Ipsen. Press release. August 16, 2023. Accessed August 17, 2023. https://www.ipsen.com/us/press-releases/us-fda-approves-ipsens-sohonostm-palovarotene-capsules-the-first-and-only-treatment-for-people-with-fibrodysplasia-ossificans-progressiva/
