FONDAZIONE TELETHON AND ORCHARD THERAPEUTICS COMPLETE TRANSFER OF MARKETING AUTHORIZATION OF STRIMVELIS FOR ADA-SCID IN EUROPE

(Adnkronos) – Following a positive opinion from the EMA, the transfer of marketing authorization of Strimvelis from Orchard Therapeutics has been approved by the European Commission Fondazione Telethon will now be responsible for providing the gene therapy to eligible patients in the European Union MILAN, BOSTON, and LONDON, Sept. 12, 2023 /PRNewswire/ — Fondazione Telethon, one of the main Italian biomedical charities, and Orchard Therapeutics, a global gene therapy leader, today announced the completion of the transfer of the marketing authorization for Strimvelis, a gene therapy approved by the European Medicines Agency in 2016 for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID). The marketing authorization transfer was approved on July 17th by the European Commission[1] following a positive opinion from the European Medicines Agency (EMA). The European manufacturing and distribution rights have been fully transferred to Fondazione Telethon from its former holder, Orchard Therapeutics, which previously announced it would discontinue investment in and seek strategic alternatives for its programs in rare primary immune deficiencies, including Strimvelis. Strimvelis originated from research carried out by the San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET) and a commitment by Fondazione Telethon to make these scientific achievements widely available to patients who may potentially benefit. It was approved for reimbursement in Italy by the Agenzia Italiana del Farmaco (AIFA) in 2016 and has been administered exclusively at the San Raffaele Hospital in Milan, Italy. A total of 45 patients from over 20 countries worldwide have been treated with Strimvelis in clinical trials and commercially, to date. Fondazione Telethon will continue to make Strimvelis available to eligible patients through the San Raffaele Hospital in Milan, Italy. About Strimvelis Strimvelis was authorised by the EMA on 26 May 2016 for the treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available. Strimvelis gene therapy for ADA-SCID is a one-time administration. It consists of a viral-derived vector containing a corrected version of the defective gene in the patient (ADA). When brought into contact with haematopoietic stem cells taken from the patient, this vector aims to restore the production of the missing protein. Reinfused into the bloodstream, the corrected cells are also able to differentiate into the previously missing elements (lymphocytes), thus capable of defending the body against infections. About ADA-SCID ADA-SCID (adenosine deaminase deficiency severe combined immunodeficiency) is a very rare, potentially life-threatening genetic disorder in which a defective gene blocks the production of an essential enzyme called adenosine deaminase (ADA), which is necessary for the differentiation and functioning of lymphocytes (a particular type of white blood cell). Children born with ADA-SCID do not develop a healthy immune system, so they cannot fight the most common infections and are forced to live in a sterile and isolated environment. Considering that the incidence of the disease in Europe is estimated to be between 1:375,000 to 1:660,000 live births[2] and that, according to Statista[3] the number of live births in 2021 in EU 27 countries was close to 4 million, the estimated number of new patients per year affected by ADA-deficiency are between 6 and 11. Today, there are different treatment options for ADA-SCID. The first is the transplantation of haematopoietic stem cells from a compatible family donor, which can cure the disease but is available in less than 20% of cases[4]. The second, represented by gene therapy, is based on a single administration of stem cells with the correct gene, which are taken from the patient's own bone marrow, thus eliminating possible transplant reactions against the host. In the absence of these options, transplantation from a registry-matched or partially matched donor can be opted for, with a potentially risk of incurring the graft versus host disease (GvHD), infections and other complications, even fatal in certain cases. Finally, enzyme replacement therapy (ERT) is available, i.e., the periodic intravenous administration of the missing, artificially produced enzyme; ERT is usually administered for short periods of time, waiting for a long-term treatment such as HSC transplantation or gene therapy[5]. 

 
[1] https://ec.europa.eu/health/documents/community-register/2023/20230717159799/dec_159799_en.pdf
 
[2] A. M. Flinn and A. R. Gennery, "Adenosine deaminase deficiency: A review," Orphanet Journal of Rare Diseases, vol. 13, no. 1. BioMed Central Ltd., p. 65, 24-Apr-2018, doi: 10.1186/s13023-018-0807-5. 
[3] https://www.statista.com/statistics/253401/number-of-live-births-in-the-eu/
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[4] J. Heimalla & M. Cowan, "Long term outcomes of severe combined immunodeficiency: therapy implications". Expert Rev Clin Immunol. 2017 November ; 13(11): 1029–1040. doi:10.1080/1744666X.2017.1381558 
[5] E. Grunenbaum et al, "Updated Management Guidelines for Adenosine Deaminase Deficiency". J Allergy Clin Immunol Pract Vol 11, NUMBER 6. June 2023 
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