Health Canada Approves Ipsen’s Sohonos (palovarotene capsules) as the First Approved Treatment for Fibrodysplasia Ossificans Progressiva

Regulatory News:

Ipsen (Euronext: IPN; ADR: IPSEY) today announced the Health Canada approval of Sohonos (palovarotene capsules), an oral selective retinoic-acid receptor gamma (RARγ) agonist indicated to reduce the formation of heterotopic ossification (HO; new bone formation) in adults and children aged 8 years and above for females and 10 years and above for males with fibrodysplasia ossificans progressiva (FOP).¹ Sohonos is approved for the treatment of patients with FOP for both chronic use, and for flare-ups, in these patient populations. This decision marks the first approval for Sohonos worldwide.

Dr. Howard Mayer, Executive Vice President and Head of Research and Development, Ipsen, said “FOP is a progressive and debilitating condition which has such a profound impact on patients, and their families. Until today, there was no approved medicine, and we are proud to bring this important new medicine to the FOP community.”

FOP is characterized by new bone formation outside of the normal skeletal system, like in soft connective tissues, a process known as heterotopic ossification,² which can be preceded by painful soft tissue swelling or “flare-ups.”² Flare-up episodes are common and are a substantial contributor to the formation of new bone, however bone can also form in the absence of a flare-up. Once formed, it is irreversible and leads to loss of mobility and shortened life expectancy.² It is an ultra-rare genetic disorder with an estimated prevalence of 1.36 per million individuals; however, the number of confirmed cases varies by country.^3,4.

As part of the ongoing commitment to the FOP and rare disease community, Ipsen plans to file in the US in H1 2022 and is in discussions with other regulatory authorities around the world.

About the MOVE clinical program
This approval is based on data from the ongoing MOVE trial, the first global multi-center Phase III trial in FOP. MOVE is an open-label, single-arm trial, evaluating the efficacy and safety of a chronic/flare-up dosing regimen of palovarotene in decreasing new annualized HO volume in patients with FOP

About Sohonos Sohonos is an oral, selective RARγ agonist developed as a treatment for people living with the debilitating ultra-rare genetic disorder, FOP. The treatment was acquired by Ipsen through the acquisition of Clementia Pharmaceuticals in April 2019. It is a member of the retinoid class of drugs that is associated with birth defects in humans. Sohonos must not be used by patients who are, or intend to become, pregnant due to the risk of teratogenicity. To minimize fetal exposure, Sohonos is to be administered only if all conditions for pregnancy prevention are met. Sohonos has been shown to cause premature physeal closure in growing children with FOP; periodic monitoring is recommended.