Ipsen’s Sohonos gets FDA approval for fibrodysplasia ossificans progressive

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来源: PMLiVE

Ipsen’s Sohonos (palovarotene), a drug treatment for fibrodysplasia ossificans progressive (FOP), has been approved by the US Food and Drug Administration (FDA).

FOP is a rare muskuloskeletal condition that progressively transforms muscles and tendons into bone, affecting the movement of the body. As a rare disease, FOP is estimated to Impact around 900 people globally.

Sohonos has been approved as a treatment for adults and paediatric patients with FOP – for females 8 years and older and for males 10 years and older – to mediate the interactions between the receptors, growth factors and proteins within the retinoid signaling pathway to reduce new abnormal bone formation.

The drug has 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.

“The first treatment for FOP has been proven to reduce the volume of new abnormal bone growth, which may result in better health outcomes for people living with FOP,” said Michelle Davis, executive director of International FOP Association.

The FDA approval is based on the pivotal efficacy and safety data from the phase 3 MOVE trial, the first and largest multicentre, open-label trial in adult and paediatric patients.

The 18-month data published in the Journal of Bone and Mineral Research included 107 patients (12% of the estimated number of individuals worldwide living with FOP) who received Sohonos compared with untreated individuals from Ipsen’s global FOP Natural History Study.

The study results demonstrated Sohonos effectively reduced annualised heterotopic ossification volume compared with no treatment beyond standard of care (a 54% reduction with weighted linear mixed effect model).

The study also demonstrated that Sohonos has a well-characterised safety profile, with adverse events consistent with the systemic retinoid class.

“The FDA approval of Sohonos is a breakthrough for the US FOP community. For the first time, doctors have an approved medicine available to them,” said Howard Mayer, head of research and development, Ipsen.

Sohonos is currently authorised for use for eligible patients in the US and Canada, and has a conditional approval in the United Arab Emirates.

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