Regeneron Pharmaceuticals has reported success in a pivotal study for its drug garetosmab, aimed at treating fibrodysplasia ossificans progressiva (FOP), an ultra-rare disease characterized by abnormal bone growth. This condition, which affects approximately 1,000 people worldwide, is triggered by a mutation in the ALK-2 gene, resulting in the formation of bone in areas where it should not occur, often following minor injuries. The abnormal bone growth can lead to joint immobility and respiratory issues, with a median life expectancy in the 50s. Regeneron plans to submit garetosmab for approval to U.S. regulators by the end of this year and to health agencies in other countries in the following year, marking a significant step forward in addressing this challenging condition.