US FDA approves French drugmaker Ipsen's rare bone disorder drug
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[August 17, 2023]
By Bhanvi Satija and Sriparna Roy
(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, Sohonos, was approved in adults and pediatric
patients with fibrodysplasia ossificans progressiva (FOP), a rare
genetic connective tissue disorder that causes 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.
Sohonos tablets will be sold at an estimated annual list price of
$624,000 based for 5mg (dose) per day, the company told Reuters, adding
that dose may vary based on an individual's weight and disease state.
The drug approval is based on data from a late-stage study, which showed
a 54% reduction in the volume of new abnormal bone formation in patients
compared to standard of care.
The approval puts Ipsen ahead of other drugmakers such as Regeneron
Pharmaceuticals, which is also developing an experimental drug,
garetosmab, for the disorder and expects to seek U.S. health regulator's
approval in 2024.
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Signage is seen outside of the Food and
Drug Administration (FDA) headquarters in White Oak, Maryland, U.S.,
August 29, 2020. REUTERS/Andrew Kelly/File Photo
![](http://archives.lincolndailynews.com/2023/Aug/17/images/ads/current/graue_quarter_FAIR_2023.png) Currently, FOP patients rely on high
doses on steroids at the start of a 'flare-up', which entail
unpredictable episodes of soft tissue swelling, pain, reduced
movement and stiffness.
FOP occurs in about 1 in 1,600,000 newborns, according to U.S.
government data, and about 800 people worldwide are known to have
the disease.
Ipsen is also developing another FOP drug licensed from Blueprint
Medicines Corp called BLU-782. It has been boosting its rare disease
portfolio by helping develop a string of drugs licensed from other
smaller developers.Sohonos will have a boxed warning for
embryo-fetal toxicity and premature epiphyseal closure, which can
lead to stunted growth and deformities in bone.
(Reporting by Bhanvi Satija, Sriparna Roy and Pratik Jain in
Bengaluru; Editing by Shinjini Ganguli)
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