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Gene Therapies Will Be A Bigger Cost Issue In 2021, PwC Predicts

Executive Summary

The expected launch of BioMarin's Roctavian for hemophilia A is expected to heighten the profile of expensive gene therapies on payers' radar. Specialty drug costs are expected to continue to grow in 2021. 

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Not Such A Sure Thing: FDA Knocks Back BioMarin’s Roctavian

BioMarin’s confidence that its first-ever hemophilia gene therapy would be cleared by the US FDA was shaken by a complete response letter that the company claims changed the requirements for approval.

Not Such A Sure Thing: FDA Knocks Back BioMarin’s Roctavian

BioMarin’s confidence that its first-ever hemophilia gene therapy would be cleared by the US FDA was shaken by a complete response letter that the company claims changed the requirements for approval.

BioMarin All Set For Hemophilia Gene Therapy Approval, But Is It Overestimating Demand?

Analysts forecast a slow climb towards peak sales of $2bn – but competitors could erode that figure.

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