Lexeo Therapeutics has secured Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for LX2020, its investigational AAV-based gene therapy for PKP2 arrhythmogenic cardiomyopathy.
The decision follows interim clinical data from the ongoing HEROIC-PKP2 phase 1/2 trial.
The company said LX2020 now holds RMAT, Orphan Drug and Fast Track designations, strengthening its regulatory position as development continues.
Narinder Bhalla, Chief Medical Officer of Lexeo Therapeutics, said: “Receiving RMAT designation for LX2020 is an important regulatory milestone that underscores the strength of the emerging clinical evidence from our HEROIC-PKP2 trial and the potential of LX2020 to address the underlying genetic cause of PKP2-ACM.”
He added: “With no approved disease-modifying treatments currently available for patients living with this serious, progressive cardiovascular disease, this designation provides a valuable opportunity for early and ongoing engagement with the FDA as we work to advance LX2020 as a potential one-time gene therapy designed to address this significant unmet need.
“We look forward to sharing additional clinical and regulatory updates before the end of the year.”
RMAT designation is intended to support the development and review of regenerative medicine therapies for serious conditions where early clinical evidence suggests potential to meet unmet medical needs.
It also offers enhanced interaction with the FDA, including guidance on clinical development, manufacturing and regulatory pathways.
Alongside Orphan Drug and Fast Track status, RMAT designation may provide eligibility for accelerated approval, priority review and rolling review.
