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FDA Approves Oral Tx for RARE Fibrodysplasia Ossificans Progressiva Sohonos

Catching up on recent FDA approvals.

The FDA recently approved a new oral therapy, Sohonos (palovarotene) from Ipsen Biopharmaceuticals, for reduction in the volume of new heterotopic ossification (extra-skeletal bone formation) in adults and children aged 8 years and older for females, and 10 years and older for males with fibrodysplasia ossificans progressiva.  Sohonos is the first drug approved for patients with this condition.

Fibrodysplasia ossificans progressiva is a rare, autosomal dominant disease where connective tissue such as muscle, tendons and ligaments gradually turn into bone tissue, causing limited movement, deformities and severe disability. Sohonos is a retinoic acid receptor gamma agonist that curbs progression of the genetic disease by reducing the volume of new, abnormal bone growth.

US approval followed four years in application queue, two clinical trial holds, a complete response letter, a European Commission rejection, a scrapped trial in another indication and multiple safety flags, resubmissions, and other delays.

Ipsen confirmed that Sohonos will launch at a cost of $624,000 annually for a 5-mg daily dose for an ‘average’ patient….. but dosing will also depend on disease severity and patient weight.

Ipsen did not disclose distribution plans for the launch. Given its ‘rare’ designation, cost, and management challenges, it is expected that distribution will be through limited distribution.

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