This post was originally published on this site
(Reuters) -The U.S. Food and Drug Administration (FDA) on Wednesday approved French drugmaker Ipsen’s drug for a rare bone disorder, making it the first treatment available to patients with the condition that causes abnormal bone growth.
The company said its drug, to be sold under brand name Sohonos, was approved in adults and pediatric patients with fibrodysplasia ossificans progressiva (FOP), which results in a progressive loss of mobility and reduced life expectancy.
The regulator approved use of the drug in girls aged 8 years and above, and 10 years and above in boys.
FOP is a rare genetic connective tissue disorder that occurs in about 1 in 1,600,000 newborns, according to U.S. government data, and about 800 people worldwide are known to have FOP.
UBS analyst Michael Leuchten had estimated Ipsen’s drug price at $400,000 per year ahead of the FDA’s decision.
The decision follows the recommendation of an independent panel of advisors to the FDA that had voted in favor of the drug as an effective treatment for FOP in June.
The approval puts Ipsen ahead of other drugmakers such as Regeneron (NASDAQ:REGN) Pharmaceuticals, which is also developing an experimental drug, garetosmab, for the disorder and expects to seek U.S. health regulator’s approval in 2024.
FOP patients rely on high doses on steroids at the start of a ‘flare-up’, in which patients experience unpredictable episodes of soft tissue swelling, pain, reduced movement and stiffness.
Ipsen has been boosting its rare disease portfolio by helping develop a string of drugs that it licensed from other smaller developers.
The company gained access to the approved drug, chemically known as palovarotene, through its 2019 acquisition of U.S. based Clementia Pharmaceuticals in a deal worth $1.31 billion. It is also developing another FOP drug licensed from Blueprint Medicines (NASDAQ:BPMC) Corp called BLU-782.
