Rocket Pharma reaches FDA alignment on Phase 2 pivotal trial for RP-A501 in Danon Disease

ROCKET PHARMACEUTICALS, INC.

FDA agreed to single-arm, open-label trial with 12 patients, natural history comparator, and co-primary endpoint of LAMP2 expression and LV mass at 12 months for accelerated approval.
In-house cGMP manufacturing completed with sufficient drug product to supply the pivotal study; potency assays qualified per FDA guidance.
ICD-10 code secured from CMS for LAMP2 deficiency; CTA/IMPD filing for EU initiation expected by end of Q3 2023.
RP-A501 is a one-time infusion targeting a uniformly fatal cardiomyopathy with no approved therapies; affects 15,000–30,000 in US and Europe.

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