Aug 19 – The U.S. FDA on Wednesday approved Regeneron’s experimental drug for a rare genetic disorder after it significantly reduced abnormal bone formation in certain soft tissues.
The New York-based Regeneron’s shares rose 4%.
The drug, garetosmab, branded as Pasatru, was approved for treating adults with fibrodysplasia ossificans progressiva — a condition where muscle, tendon and ligament tissue gradually turn into bone, leading to a “second skeleton” that causes progressive loss of mobility and reduced life expectancy.
It affects roughly 1 in 2 million people worldwide, with around 800 to 900 active diagnosed cases globally, data from the National Institutes of Health showed.
In a 56-week trial involving 63 participants, garetosmab reduced the development of new bone abnormalities by 94% in patients treated with a 3 mg per kg dose and by 90% in the case of a 10 mg per kg dose, when compared with placebo.
(Reporting by Bageshri Banerjee and Christy Santhosh in Bengaluru; Editing by Jonathan Ananda and Sahal Muhammed)
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