RCKT Rocket Pharmaceuticals

Rocket Pharmaceuticals Secures up to $150M Credit Facility from Hercules to Fund RP-A501 Pivotal Study

Rocket entered a credit facility with Hercules Capital providing up to $150M, with $35M received at closing. The financing supports execution of the pivotal Phase 2 study of RP-A501 for Danon disease and advancement of its cardiovascular gene therapy pipeline. Additional tranches are tied to clinical milestones and committee approval.

RP-A501 · NCT06092034 registry update

primary_completion: 2028-04 → 2028-06; completion: 2032-04 → 2032-06; enrollment: 14 → 18; primary_outcomes: ['Evaluation of efficacy via primary endpoint comprised of LAMP2 myocardial tissue expression and left ventricular mass index'] → ['Evaluation of Efficacy: Improvement as assessed via primary endpoint comprised of LAMP2 myocardial tissue expression and left ventricular mass index (LVMI)']

Rocket Pharmaceuticals: FDA aligns on pivotal Phase 2 trial for RP-A501 in Danon disease

FDA confirmed the pivotal efficacy population of 12 male patients at the recalibrated dose of 3.8 × 10¹³ GC/kg, with the first three patients already treated counting toward this total. The agency also endorsed the 12-month co-primary endpoints of myocardial LAMP2 protein expression and a 10% reduction in left ventricular mass index, intended to support a potential accelerated approval pathway. Enrollment and dosing under the modified protocol may continue, with completion of dosing expected by mid-2027.

Rocket Pharmaceuticals: FDA aligns on 12-patient pivotal Phase 2 for RP-A501 in Danon disease

FDA confirmed the pivotal efficacy population of 12 patients, including the first three treated under the modified protocol, and endorsed the 12-month co-primary endpoints intended to support accelerated approval. The agency also cleared continued enrollment and dosing at the recalibrated 3.8 × 10¹³ GC/kg dose using commercial-grade product. Rocket now has a defined path to complete the pivotal study by mid-2027.

Rocket Pharmaceuticals sells KRESLADI PRV for $180 million

Rocket monetized the Rare Pediatric Disease Priority Review Voucher awarded after FDA accelerated approval of KRESLADI. The $180 million non-dilutive proceeds extend the company's cash runway into Q2 2028 and will fund its cardiovascular gene therapy pipeline.

Rocket Pharmaceuticals, Inc. · FDA licenses kresladi (marnetegragene autotemcel)

FDA's Purple Book lists BLA 125806 (kresladi, marnetegragene autotemcel; Rocket Pharmaceuticals Inc, CBER) as licensed on 2026-03-26.

Rocket Pharmaceuticals: KRESLADI PDUFA set for March 28, 2026

FDA accepted the resubmission of the BLA for KRESLADI (marnetegragene autotemcel) in severe LAD-I and assigned a PDUFA target action date of March 28, 2026. The company is eligible for a Rare Pediatric Disease Priority Review Voucher upon approval. This establishes a near-term regulatory milestone for the lentiviral hematology program.

Rocket Pharmaceuticals: KRESLADI™ PDUFA set for March 28, 2026

The FDA accepted the resubmission of the BLA for KRESLADI™ (marnetegragene autotemcel) and assigned a PDUFA target action date of March 28, 2026. Rocket is also eligible for a Rare Pediatric Disease Priority Review Voucher upon approval. The company states the asset is on track for this date.

Rocket Pharmaceuticals: FDA accepts KRESLADI BLA resubmission; PDUFA March 28, 2026

FDA has accepted the resubmitted BLA for KRESLADI (marnetegragene autotemcel) in severe LAD-I. The agency set a PDUFA target action date of March 28, 2026. Rocket is eligible for a Rare Pediatric Disease Priority Review Voucher upon approval.

Rocket Pharmaceuticals withdraws BLA for RP-L102 in Fanconi Anemia

Rocket voluntarily withdrew its U.S. BLA for mozafancogene autotemcel (RP-L102) on October 3, 2025. The withdrawal follows the July 2025 decision to cease new internal investment and the prior withdrawal of the European MAA. The action is strategic and does not reflect safety or efficacy concerns; the company retains the option to re-engage regulators via external partnership.