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Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-L301 Gene Therapy for Pyruvate Kinase Deficiency (PKD). RMAT designation was granted based on robust safety and efficacy data from the ongoing Phase 1 RP-L301 clinical trial and its potential to cure a life-threatening disease for which no curative therapies currently exist.

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CALQUENCE (acalabrutinib) is a next-generation, selective inhibitor of BTK that binds covalently to BTK, thereby inhibiting its activity. The drug initially received accelerated approval based on overall response rate in the US in October 2017 for the treatment of adult patients with MCL who have received at least one prior therapy, and later in August 2022 received full approval for the same. Although, now AstraZeneca has been investigating CALQUENCE for earlier use in treatment. However, it is important to note that CALQUENCE is not yet approved for MCL treatment in Japan or the EU.

Unlock In-Depth Expert Insights and Analysis with DelveInsight's Exclusive EHA 2024 Reporting @ https://www.delveinsight.com/eha-conference/eha-2024/calquence-phase-iii-echo-trial?utm_source=eha&utm_medium=promotion&utm_campaign=kpr