Danon Disease Gene Therapy & TGCT Treatment Win EMA PRIME Designations
Promising early clinical trial results from Rocket and Abbisko respectively helped the companies secure their places on the European Medicines Agency’s priority medicines scheme, which aims to help speed up the development and regulatory review of treatments for unmet medical needs.
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A total of four investigational products targeting unmet medical needs have made it onto the European Medicines Agency’s priority medicines scheme so far this year.
While the European Medicines Agency and industry speculate as to why 2022 saw far fewer applications for a place on the priority medicines scheme for drugs for unmet medical needs, they are enthusiastic about improvements to the initiative that are set to be trialed soon.
While other gene therapy companies faced major clinical and financing problems in 2022, Rocket Pharma sidestepped some of these pitfalls and is gearing up for two US filings this year.