Buzz for gene therapies is loud, but drugmakers struggle to get treatments off the ground

Roctavian held promise of treating hemophilia A, but has only been used for one patient since its approval in June 2023, Bloomberg reported Feb. 20. 

Made by BioMarin Pharmaceutical, the therapy marked the company's first moves into gene therapies that seek to correct genetic conditions and treat inherited disorders. But, Roctavian so far hasn't taken off commercially, and did not live up to its expected promise of bringing in $100 million for the pharmaceutical company in 2023. 

BioMarin isn't alone, though. Several drugmakers have struggled to see gene therapies take off in recent years, according to Bloomberg

A major barrier for many companies in the space is sheer cost to develop these advanced medical therapies. Though the Biden administration and CMS announced Jan. 30 plans to bring down prices for gene edited therapies, progress will take time. 

The number of patients being treated with the existing gene therapies that are approved and available on the market is expected to decline year over year by nearly 33%, according to Bloomberg. Spending on these solutions is also expected to decrease by $4.3 million in 2024. 

In the meantime, BioMarin is also looking at enhancing the effectiveness of Roctavian's long-term benefits so patients and providers can avoid needing any further treatments in the future. 

The company is still standing behind its gene therapy solution, noting to Bloomberg that it is still "encouraged by the high level of interest in Roctavian from patients, physicians, patient advocacy groups, hemophilia treatment centers and payers."

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