Rocket Pharmaceuticals Receives FDA Fast Track and Orphan Drug Designations for RP-A601 Gene Therapy for PKP2 Arrhythmogenic Cardiomyopathy (ACM) - Seite 2

About FDA Fast Track Designation

Fast Track designation is designed to facilitate the development and expedite the review of drug candidates that are intended to treat serious conditions and for which nonclinical data has demonstrated the potential to address unmet medical need. The program enables increased communication with the FDA for the purpose of expediting the product's development, review, and potential approval. Therapies may qualify for accelerated approval and priority review if criteria are met, in addition to Rolling Review, which affords the opportunity to submit completed sections of the Biologic License Application (BLA) for review by the FDA, rather than waiting until every section is completed before the entire application can be reviewed.

About FDA Orphan Drug Designation

Orphan Drug designation is granted by the FDA for the treatment, prevention or diagnosis of diseases that are life-threatening or chronically debilitating with a prevalence of fewer than 200,000 patients annually. The designation provides certain benefits, including market exclusivity upon regulatory approval, exemption of FDA application fees, and tax credits for qualified clinical trials.

About PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM)

PKP2-ACM is an inherited heart disease caused by mutations in the PKP2 gene and characterized by life-threatening ventricular arrhythmias, cardiac structural abnormalities, and sudden cardiac death. PKP2-ACM affects approximately 50,000 adults and children in the U.S. and Europe. Patients living with PKP2-ACM have an urgent unmet medical need, as current medical, implantable cardioverter defibrillator (ICD), and ablation therapies do not consistently prevent disease progression or arrhythmia recurrence, are associated with significant morbidity including inappropriate shocks and device and procedure-related complications, and do not address the underlying pathophysiology or genetic mutation. RP-A601 is being investigated as a one-time, potentially curative gene therapy treatment that may improve survival and quality of life for patients affected by this devastating disease.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare childhood disorders. The Company’s platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rocket’s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. Rocket also has received IND clearance for the AAV-based gene therapy program for PKP2-arrhythmogenic cardiomyopathy (ACM) and is advancing a preclinical program for BAG3-associated dilated cardiomyopathy (DCM). For more information about Rocket, please visit www.rocketpharma.com.