This section highlights FDA-related milestones and regulatory updates for drugs developed by Rocket Pharmaceuticals (RCKT).
Over the past two years, Rocket Pharmaceuticals has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
KRESLADI, RP-A501, RP-A601, RP-A701, and RP-L102. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
KRESLADI - FDA Regulatory Timeline and Events
KRESLADI is a drug developed by Rocket Pharmaceuticals for the following indication: For severe Leukocyte Adhesion Deficiency-I.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- KRESLADI
- Announced Date:
- June 28, 2024
- Indication:
- For severe Leukocyte Adhesion Deficiency-I
Announcement
Rocket Pharmaceuticals, Inc. announced a regulatory update for KRESLADI™ (marnetegragene autotemcel; marne-cel), a lentiviral (LV) vector-based gene therapy to treat severe leukocyte adhesion deficiency-I (LAD-I).
AI Summary
Rocket Pharmaceuticals, Inc. announced a regulatory update regarding its gene therapy KRESLADI™ (marnetegragene autotemcel; marne-cel), designed to treat severe leukocyte adhesion deficiency-I (LAD-I). The U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for the therapy’s Biologics License Application, requesting limited additional Chemistry Manufacturing and Controls (CMC) information.
The company recently met with senior FDA leaders from the Center for Biologics Evaluation and Research (CBER) to clearly outline the extra CMC details needed. This meeting emphasized a close collaboration between Rocket and the FDA, with both parties working together to address the high level of unmet need for LAD-I patients. Rocket’s leadership expressed optimism that this cooperative approach will help secure a swift review and approval of KRESLADI™, potentially allowing faster access to this promising treatment for children suffering from this serious condition.
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RP-A501 - FDA Regulatory Timeline and Events
RP-A501 is a drug developed by Rocket Pharmaceuticals for the following indication: Danon Disease.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- RP-A501
- Announced Date:
- May 27, 2025
- Indication:
- Danon Disease
Announcement
Rocket Pharmaceuticals, Inc announced an update related to RP-A501, its investigational gene therapy for Danon disease.
AI Summary
Rocket Pharmaceuticals, Inc. recently provided an update on RP-A501, its experimental gene therapy for Danon disease. During a Phase 2 pivotal trial, a patient experienced an unexpected serious adverse event linked to capillary leak syndrome. The event was associated with a new immune suppression agent added to the pre-treatment plan to reduce complement activation in patients. In response, Rocket voluntarily halted further dosing, and the FDA placed a clinical hold on the trial on May 23, 2025, to allow for a detailed review of the case. The company has begun a comprehensive root cause analysis and is actively working with the FDA, an independent Data Safety Monitoring Committee, clinical investigators, and scientific experts to ensure patient safety. Rocket expressed their deep regret over the loss of the patient and reaffirmed their commitment to advancing gene therapy options for those affected by Danon disease.
Read Announcement- Drug:
- RP-A501
- Announced Date:
- February 27, 2025
- Indication:
- Danon Disease
Announcement
Rocket Pharmaceuticals, Inc reported financial and recent operational results
AI Summary
Rocket Pharmaceuticals, Inc. reported its financial and operational results for the fourth quarter and full year ended December 31, 2024. The company highlighted progress in its gene therapy portfolio, particularly for Danon disease. Dosing in the Phase 2 pivotal study of RP-A501 is currently underway, with a program update anticipated in the first half of 2025. Additional long-term data from the Phase 1 study—published in The New England Journal of Medicine and showcased at the American Heart Association meeting—demonstrated sustained safety and meaningful efficacy for patients. The company is also advancing other clinical programs, including a Phase 1 trial for RP-A601 addressing PKP2-ACM. Financially, Rocket reported cash, cash equivalents, and investments of approximately $372.3 million, providing an operational runway into the third quarter of 2026. They also raised net proceeds of $182.5 million through a recent public offering.
Read Announcement- Drug:
- RP-A501
- Announced Date:
- November 19, 2024
- Indication:
- Danon Disease
Announcement
Rocket Pharmaceuticals, Inc presented long-term safety and efficacy results from the Phase 1 study of RP-A501 in male patients with Danon disease.
AI Summary
Rocket Pharmaceuticals, Inc. recently shared promising long-term results from its Phase 1 study of RP-A501 for male patients with Danon disease. The study found that the gene therapy was generally well tolerated and showed clear signs of success. Notably, all evaluable patients demonstrated sustained cardiac LAMP2 protein expression by 12 months, with effects continuing for up to 60 months. In addition, patients experienced a meaningful reduction in left ventricular mass index (≥10% reduction maintained through 54 months) and improvements in heart function, moving from NYHA Class II to Class I. Other benefits included substantial decreases in key cardiac biomarkers—troponin and BNP—by 84% and 57% respectively, and a significant rise in quality of life scores. These results, published in The New England Journal of Medicine, mark an important milestone in developing a one-time gene therapy aimed at addressing the root cause of Danon disease.
Read Announcement- Drug:
- RP-A501
- Announced Date:
- November 18, 2024
- Indication:
- Danon Disease
Announcement
Rocket Pharmaceuticals, Inc presented long-term safety and efficacy results from the Phase 1 RP-A501 study which showed that RP-A501 was generally well tolerated and all evaluable Danon disease patients demonstrated LAMP2 protein expression at 12 months (sustained up to 60 months) and reduction of left ventricular (LV) mass index by ≥10% at 12 months (sustained up to 54 months) after treatment.
AI Summary
Rocket Pharmaceuticals recently presented long-term safety and efficacy results from its Phase 1 RP-A501 study for Danon disease. In the study, RP-A501 was generally well tolerated by patients. Notably, all evaluable Danon disease patients showed cardiac LAMP2 protein expression at 12 months—a benefit that persisted for up to 60 months after treatment.
The study also demonstrated a reduction of left ventricular (LV) mass index by at least 10% at 12 months, with this improvement sustained for up to 54 months. These promising findings suggest that RP-A501 may offer a meaningful treatment option for patients with Danon disease, providing evidence of long-term protein restoration and cardiac improvement essential for managing this rare genetic disorder.
Read Announcement- Drug:
- RP-A501
- Announced Date:
- September 17, 2024
- Indication:
- Danon Disease
Announcement
Rocket Pharmaceuticals, Inc announced that all patients have been enrolled in the global, pivotal Phase 2 clinical trial evaluating RP-A501 to treat male patients with Danon disease.
AI Summary
Rocket Pharmaceuticals has completed enrollment of all patients in its global, pivotal Phase 2 clinical trial for RP-A501, an investigational gene therapy aimed at treating male patients with Danon disease. The trial, which follows a safety run-in with two pediatric patients, enrolled an additional 10 patients across the United States and European Union within just three months. This rapid patient recruitment highlights international collaboration and optimism within the clinical community. The study is designed to assess the efficacy and safety of RP-A501 by measuring improvements in LAMP2 protein expression and reductions in left ventricular mass, among other key endpoints. Rocket Pharmaceuticals plans to submit regulatory filings simultaneously in the U.S. and other regions, advancing the goal of providing a promising one-time treatment to help improve cardiac function and alleviate the severe impacts of Danon disease.
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RP-A601 - FDA Regulatory Timeline and Events
RP-A601 is a drug developed by Rocket Pharmaceuticals for the following indication: RP-A601 decreased arrhythmias and increased survival in the PKP2 knockout mouse model.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- RP-A601
- Announced Date:
- May 15, 2025
- Indication:
- RP-A601 decreased arrhythmias and increased survival in the PKP2 knockout mouse model
Announcement
Rocket Pharmaceuticals, Inc announced preliminary data from the Phase 1 clinical trial of RP-A601 for the treatment of plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM).
AI Summary
Rocket Pharmaceuticals, Inc. announced encouraging preliminary results from its Phase 1 clinical trial of RP-A601 for treating plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM). In this study, three patients received a single intravenous dose of 8.0E13 GC/kg, and RP-A601 was generally well-tolerated with no dose-limiting toxicities observed during up to 12 months of follow-up. The gene therapy increased the expression of PKP2 protein and improved the desmosomal localization of key proteins such as Plakophilin-2, Desmocollin-2, and Cadherin-2. Additionally, early signs pointed to improvements or stabilization in arrhythmia burden, heart function, and overall quality of life among the patients. These findings support the potential of RP-A601 as a promising one-time, gene therapy treatment for a rare and serious genetic heart condition.
Read Announcement- Drug:
- RP-A601
- Announced Date:
- May 9, 2025
- Indication:
- RP-A601 decreased arrhythmias and increased survival in the PKP2 knockout mouse model
Announcement
Rocket Pharmaceuticals, Inc. announced that the company will be presenting data for the first time from the Phase 1 clinical trial of RP-A601 to treat patients with plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM) at the Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) being held from May 13-17 in New Orleans, LA.
AI Summary
Rocket Pharmaceuticals, Inc. announced that it will present its first Phase 1 clinical trial data for RP-A601 at the Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in New Orleans, LA, from May 13-17. The therapy aims to treat patients with plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM), a serious heart condition associated with dangerous arrhythmias and sudden cardiac death. The presentation, part of the Late-Breaking Abstracts session, will include preliminary data showcasing the safety and potential benefits of administering RP-A601—an investigational gene therapy delivered as a single intravenous infusion—to adult patients suffering from this inherited disorder. The data presentation highlights Rocket’s ongoing commitment to advancing therapies that address unmet medical needs in rare cardiovascular diseases.
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RP-A701 - FDA Regulatory Timeline and Events
RP-A701 is a drug developed by Rocket Pharmaceuticals for the following indication: For the Treatment of BAG3-associated Dilated Cardiomyopathy.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- RP-A701
- Announced Date:
- June 30, 2025
- Indication:
- For the Treatment of BAG3-associated Dilated Cardiomyopathy
Announcement
Rocket Pharmaceuticals, Inc. announced that it has received clearance from the U.S. Food and Drug Administration (FDA) for the Company's Investigational New Drug (IND) application for RP-A701, an AAVrh.74-based gene therapy candidate for the treatment of BAG3-associated Dilated Cardiomyopathy (BAG3-DCM), a severe form of heart failure characterized by progressive ventricular enlargement and impaired systolic function. "The FDA clearance of RP-A701, our third clinical-stage gen
AI Summary
Rocket Pharmaceuticals, Inc. has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application for RP-A701, an AAVrh.74-based gene therapy candidate. This new treatment is aimed at combating BAG3-associated dilated cardiomyopathy, a rare and severe form of heart failure that causes enlarged ventricles and weakened heart function.
The therapy works by addressing the mutation in the BAG3 gene, which contributes to early-onset heart failure, high morbidity, and premature death. With this FDA clearance, the company is preparing to launch a Phase 1 clinical trial in the U.S. This trial will test the safety of RP-A701, observe its biological activity, and look for early signs of efficacy as it moves toward treating the first patient.
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RP-L102 - FDA Regulatory Timeline and Events
RP-L102 is a drug developed by Rocket Pharmaceuticals for the following indication: Fanconi Anemia.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- RP-L102
- Announced Date:
- May 10, 2024
- Indication:
- Fanconi Anemia
Announcement
Rocket Pharmaceuticals, Inc. announced longer-term data updates from its lentiviral (LV) vector hematology portfolio presented at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT).
AI Summary
Rocket Pharmaceuticals, Inc. recently presented longer-term data updates from its lentiviral (LV) vector hematology portfolio at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). The new findings highlight the progress made in using LV vectors to treat blood disorders, underscoring improvements in both safety and effectiveness. The data provides valuable insights that may help guide future clinical studies and patient treatment, as researchers continue to explore gene therapy options for hematological conditions.
This update is an important milestone for Rocket Pharmaceuticals, as it demonstrates commitment to developing advanced gene therapy treatments using innovative LV vector technology. The longer-term results aim to build confidence in the treatment approach and offer hope to patients who may benefit from these therapies. The presentation at ASGCT marks another step forward in the ongoing efforts to make safe and effective treatments available to those in need.
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