Danon disease

Orphan designation: Adeno-associated virus serotype 9 vector containing the human LAMP2 isoform B transgene Treatment of Danon disease, 16/08/2023 Positive

Retrieved on: 
Sunday, February 4, 2024
COMP, IRIS, European Commission, Committee, LAMP2, Patient, Danon disease, EMA, Pharmaceutical industry

Key facts

Key Points: 
  • Key facts
    - Active substance
    - Adeno-associated virus serotype 9 vector containing the human LAMP2 isoform B transgene
    - Intended use
    - Treatment of Danon disease
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/23/2826
    - Date of designation
    - Sponsor
    Rocket Pharmaceuticals B.V.
    Kingsfordweg 151
    1043 GR Amsterdam
    Noord-Holland
    Netherlands
    E-mail: [email protected]
    Patients' organisations
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Rocket Pharmaceuticals Reports Third Quarter 2023 Financial Results and Highlights Recent Progress

Retrieved on: 
Monday, November 6, 2023
FDA, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, Consensus, Patient, Research, Busulfan, American College, CGMP, Genetic testing, Clinical trial, AAV, PDUFA, Incidence, IMPD, LV, Danon disease, Malignancy, Medication, Mortality, CMC, Conference, CTA, Acquisition, PKP2, Disease, JACC, Journal, Consensus decision-making, Priority review, Male, PKD, Natural history, MHRA, Disorder, EMA, Arrhythmia, Journal of the American College of Cardiology, LAMP2, Safety, Investment, Diagnosis, BLA, Pediatric Blood & Cancer, Pharmaceutical industry, Medical device, Security (finance), Fine chemical, G&A

Supported peer-reviewed expert consensus paper to increase awareness of diagnosis and clinical management of patients with Danon Disease.

Key Points: 
  • Supported peer-reviewed expert consensus paper to increase awareness of diagnosis and clinical management of patients with Danon Disease.
  • Highlights of the publication include a review of diagnosing Danon Disease emphasizing the importance of genetic testing upon clinical suspicion, natural history, management recommendations and recent advances in potential gene therapy treatment.
  • Rocket is working towards initiation of Phase 2 pivotal trial activities in Europe and the UK.
  • As of September 30, 2023, Rocket had cash, cash equivalents and investments of $437.2 million.

Rocket Pharmaceuticals Announces Presentations Highlighting Lentiviral and AAV Gene Therapies at the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT)

Retrieved on: 
Thursday, October 19, 2023
Research, Genetics, Biotechnology, Other Health, Health, Pharmaceutical, General Health, Other Science, Science, Patient, PKP2, Medication, Danon disease, MBA, Arrhythmogenic cardiomyopathy, Pharming, PKD, Cell, Fanconi anemia, Disorder, Gene, ESGCT, European Society of Gene and Cell Therapy, Pyruvate kinase deficiency, Pharmaceutical industry

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced presentations at the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) in Brussels, Belgium, taking place October 24-27.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced presentations at the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) in Brussels, Belgium, taking place October 24-27.
  • Updated data will be presented from the Phase 1 clinical trial of RP-L301 for Pyruvate Kinase Deficiency (PKD).
  • Previously disclosed data will be presented from the Phase 2 pivotal trial of RP-L102 for Fanconi Anemia, Phase 1 trial of RP-A501 for Danon Disease and preclinical studies supporting the Phase 1 trial of RP-A601 for PKP2 arrhythmogenic cardiomyopathy (PKP2-ACM).
  • Kinnari Patel, Pharm.D., MBA, President and Chief Operating Officer, Rocket Pharma, will also give an Invited Talk about the importance of collaboration with and commitment to patients in gene therapy development.

Rocket Pharmaceuticals Reaches FDA Alignment on Pivotal Phase 2 Trial Design for RP-A501 in Danon Disease

Retrieved on: 
Tuesday, September 12, 2023
Research, FDA, Genetics, Clinical Trials, Cardiology, Biotechnology, Health, Pharmaceutical, Science, NYHA, Male, CMS, LV, Troponin, IMPD, CMC, Center for Biologics Evaluation and Research, CTA, KCCQ, Disorder, Natriuresis, Medication, New York Heart Association Functional Classification, Food and Drug Administration Amendments Act of 2007, Clinical trial, CGMP, Patient, Heart transplantation, Immunohistochemistry, Mortality, Danon disease, Death, Food, Safety, Disease, Pharmaceutical industry, Medical device, EU, LAMP2

The disease affects an estimated 15,000 to 30,000 patients in the U.S. and Europe.

Key Points: 
  • The disease affects an estimated 15,000 to 30,000 patients in the U.S. and Europe.
  • A global natural history study will serve as an external comparator and run concurrently to the Phase 2 pivotal trial.
  • In-house manufacturing has been completed with sufficient high-quality drug product produced to fully supply the Phase 2 pivotal study.
  • Additionally, Rocket has secured an ICD-10 code from CMS for LAMP2 deficiency in Danon Disease

Rocket Pharmaceuticals Reports Second Quarter 2023 Financial Results and Highlights Recent Progress

Retrieved on: 
Thursday, August 10, 2023
Health, Genetics, Research, Pharmaceutical, Science, Biotechnology, University of California, San Diego Performance-Based Skills Assessment, Communication, Advanced Therapy Medicinal Product, LV, University, PKP2, Marketing, European Medicines Agency, Survival, Clinical trial, Bone marrow, UCSD, Arrhythmia, AAV, Disorder, Medication, Fast track (FDA), EMA, ATMP, MMC, BAG3, Investigational New Drug, Invitae, Patient, IRB, Investment, CGMP, LAMP2, Genetic testing, Cardiomyopathy, FDA, Death, BLA, IND, Gene, Safety, Pharmaceutical industry, Vaccine, Fine chemical, Cryptocurrency, G&A, Danon disease

“The second quarter of 2023 marked a period of strong forward momentum across our cardiovascular AAV and hematology LV portfolios highlighted by the BLA submission for our LAD-I program, Rocket’s first product filing.

Key Points: 
  • “The second quarter of 2023 marked a period of strong forward momentum across our cardiovascular AAV and hematology LV portfolios highlighted by the BLA submission for our LAD-I program, Rocket’s first product filing.
  • ATMP designation is intended for medicines that offer groundbreaking new opportunities and allows for a single evaluation and authorization procedure.
  • Initiating two-patient pediatric safety run-in for RP-A501 pivotal study; approaching final alignment with FDA on primary composite endpoint to support accelerated approval.
  • As of June 30, 2023, Rocket had cash, cash equivalents and investments of $307.0 million.

IPS HEART Receives U.S. FDA Rare Pediatric Drug Designation for ISX9-CPC Stem Cell Therapy for Treatment of Cardiomyopathy Associated with Danon disease

Retrieved on: 
Thursday, July 6, 2023
Research, FDA, Diabetes, Stem Cells, Cardiology, Biotechnology, Health, Pharmaceutical, Science, Danon disease, Duchenne muscular dystrophy, Heart failure, Vertex, Cardiac fibrosis, Food, Cardiac muscle, Facial muscles, Patient, Cardiomyopathy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IPS, Disease, Pharmaceutical industry, HEART, Duchenne

IPS HEART, a privately held cell therapy company advancing its stem cell platform to develop new skeletal muscle and cardiac muscle generation treatments for Duchenne muscular dystrophy (a rare disease) and heart failure, today announced that the U.S. Food and Drug Administration (FDA) awarded a third Rare Pediatric Drug Designation (RPDD) for its product candidate, ISX9-CPC, for treatment of cardiomyopathy associated with Danon disease.

Key Points: 
  • IPS HEART, a privately held cell therapy company advancing its stem cell platform to develop new skeletal muscle and cardiac muscle generation treatments for Duchenne muscular dystrophy (a rare disease) and heart failure, today announced that the U.S. Food and Drug Administration (FDA) awarded a third Rare Pediatric Drug Designation (RPDD) for its product candidate, ISX9-CPC, for treatment of cardiomyopathy associated with Danon disease.
  • ISX9-CPC stem cell therapeutic candidate is designed to create new functional human heart muscle along with reducing cardiac fibrosis.
  • The FDA previously awarded RPDDs for ISX9-CPC and GIVI-MPC for the treatment of Duchenne muscular dystrophy.
  • Separately, the FDA granted Orphan Drug Designation for its GIVI-MPC for the treatment of Duchenne muscular dystrophy.

Rocket Pharmaceuticals Receives European Medicines Agency (EMA) Priority Medicines (PRIME) Designation for RP-A501 Gene Therapy for Danon Disease

Retrieved on: 
Wednesday, May 31, 2023
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Regenerative Medicine Advanced Therapy, Marketing, Pharming, European Medicines Agency, Quality of life, Risk assessment, Fast Track, MBA, Cardiology, AAV, RP, Disorder, PRIME, Medication, EMA, Troponin I, New York Heart Association Functional Classification, Patient, BNP, Danon disease, Pharmaceutical industry

PRIME designation offers the benefits of early and enhanced support from the EMA for the development of medicines that target unmet medical needs, as well as the opportunity for an accelerated review of the marketing application.

Key Points: 
  • PRIME designation offers the benefits of early and enhanced support from the EMA for the development of medicines that target unmet medical needs, as well as the opportunity for an accelerated review of the marketing application.
  • Rocket was also recently granted Regenerative Medicine Advanced Therapy (RMAT) designation for its RP-A501 program, which also holds Fast Track , Orphan Drug (U.S.) and Rare Pediatric designations.
  • “PRIME designation from the EMA further highlights the positive benefit/risk profile of RP-A501 in addressing the critical unmet need of patients facing Danon Disease,” said Kinnari Patel, Pharm.D., MBA, President and Chief Operating Officer, Rocket Pharma.
  • The results demonstrated improvements and/or normalization across multiple quantifiable parameters that cardiologists use in clinical practice to enable risk assessment and treatment decisions.

Rocket Pharmaceuticals Presents Positive Data from LV Hematology and AAV Cardiovascular Gene Therapy Programs at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)

Retrieved on: 
Friday, May 19, 2023
Health, Pharmaceutical, Biologics license application, PVC, LV, Hemolysis, Gene, ACM, Arrest, Survival, Bone marrow, Monogenic, Quality of life, PKD, Liver injury, Research, Arrhythmia, AAV, Fanconi anemia, Disorder, Pyruvate kinase deficiency, Natriuresis, Medication, First, MMC, HIV disease progression rates, Patient, Arrhythmogenic cardiomyopathy, Recombinant, Busulfan, Hematology, Disease, Cell, Kaplan–Meier estimator, Toxicology, Danon disease, Pivotal, Mortality, FA, FDA, BLA, IND, Society, Safety, Pharmaceutical industry, Medical device, Nursing, Birth control, Vaccine, PKP2

“I am pleased that we continue to deliver outstanding clinical results that highlight the momentum across both our LV hematology and AAV cardiovascular platforms,” said Jonathan Schwartz, M.D., Chief Gene Therapy Officer, Rocket Pharma.

Key Points: 
  • “I am pleased that we continue to deliver outstanding clinical results that highlight the momentum across both our LV hematology and AAV cardiovascular platforms,” said Jonathan Schwartz, M.D., Chief Gene Therapy Officer, Rocket Pharma.
  • Both adult patients reported improved quality of life with documented improvements via formal quality of life assessments.
  • The safety profile appears highly favorable, with no RP-L301-related serious adverse events in either of the adult patients.
  • Rocket is initiating Phase 1 study start-up activities and rapidly advancing the first investigational gene therapy for PKP2-ACM into the clinic.

Rocket Pharmaceuticals Reports First Quarter 2023 Financial Results and Highlights Recent Progress

Retrieved on: 
Thursday, May 4, 2023
Health, Genetics, Research, Pharmaceutical, Science, Biotechnology, LV, Frontiers, PKD, Rare Disease Day, Stanford University School of Medicine, Genetic distance, Patient, Investment, Division, Cell, Commercial, Danon disease, BLA, Disease, Inflammation, Gene, University, Rare disease, Medication, CGMP, Recombinant, Medicine, Diagnosis, Immunology, Rare, UCLA, Whole genome sequencing, Conference, Caregiver, The Human Side, University of California, Empire, AAV, Fanconi anemia, Haematopoietic system, First, ATM, Rocket, Molecule, Education, Doctor of Philosophy, Los Angeles Convention Center, AstraZeneca, Disorder, Sale, Regenerative medicine, FA, Pharmacology, Society, Pharmaceutical industry, Vaccine, Dentistry, Cryptocurrency, G&A, EU, Pediatrics, Microbiology

We look forward to leveraging this strong cash position, which will take us through planned launches in 2024," said Dr. Shah.

Key Points: 
  • We look forward to leveraging this strong cash position, which will take us through planned launches in 2024," said Dr. Shah.
  • We look forward to continuing this progress into the next quarter and year.”
    Danon, FA, LAD-I, PKD trials and plans for PKP2-ACM and BAG3-DCM preclinical programs remain on track.
  • As a founding consortium member, Rocket will play a leading role advocating for early diagnosis of patients with genetic rare diseases.
  • As of March 31, 2023, Rocket had cash, cash equivalents and investments of $360.0 million.

Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2022 Financial and Operational Results

Retrieved on: 
Monday, February 27, 2023
Research, Other Health, General Health, Pharmaceutical, Oncology, Biometrics, Genetics, Clinical Trials, Science, Cardiology, Biotechnology, Health, Other Science, CGMP, Fanconi anemia, Bone marrow, Cell, Acquisition, Lighting, Empire, Hemolysis, CD18, Regenerative Medicine Advanced Therapy, AAV, PKD, Novartis, BM, Danon disease, Senior, Biologics license application, Chief of Naval Staff (Pakistan), Patient, LV, Heart Failure Society of America, Rare disease, Pyruvate kinase deficiency, Transplant, Kaplan–Meier estimator, Schering-Plough, Center for International Blood and Marrow Transplant Research, Eli Lilly, Investment, Heart failure, Congress, Gene, Disease, MMC, Quality of life, Survival, LAMP2, PKP2, Disorder, DCM, BLA, FDA, ASH, Conference, FA, Medication, Society, Pharmaceutical industry, Medical device, Cryptocurrency, EU, G&A

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2022.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2022.
  • The Company previously presented positive data from the study at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022.
  • Based on the data from the Phase 2 pivotal Fanconi Anemia trial, Rocket anticipates submitting a BLA in the fourth quarter of 2023.
  • As of December 31, 2022, Rocket had cash, cash equivalents and investments of $399.7 million.