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

• 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, September 12, 2023 – 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 has always been a point of reference in the non-profit context for the development of therapies for rare and ultra-rare genetic diseases,” says Francesca Pasinelli, General Manager of Fondazione Telethon. “Now, we are making a paradigm shift to also be at the forefront in terms of providing patients with access to life-saving therapies that may otherwise be withdrawn from the market or halted during development because they are not viable based on current industrial business models. We are the first non-profit organization to take on the commercialization of a gene therapy. This step is necessary to remain true to our mission and continue ensuring access to this important therapy. We’d like to extend our gratitude to Orchard Therapeutics for its support throughout this transition process.”

Fondazione Telethon will continue to make Strimvelis available to eligible patients through the San Raffaele Hospital in Milan, Italy.

“ADA-SCID is a devastating disease with limited treatment options. It was very important for Orchard to find a solution that would enable continued access to Strimvelis for eligible patients and their families,” said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard Therapeutics. “We appreciate Fondazione Telethon’s commitment to the ADA-SCID community and were pleased to support a seamless transfer. We also look forward to our continued collaboration with SR-TIGET on our other approved and investigational therapies to make our shared vision of ending the devastation caused by severe genetic diseases through the curative potential of gene therapy a reality.”

About Fondazione Telethon ETS

Fondazione Telethon is one of the main Italian biomedical charities, founded in 1990 on the initiative of a group of patients suffering from muscular dystrophy. Its mission is to achieve the cure of rare genetic diseases through scientific research of excellence, selected according to the best practices shared internationally. Through a unique method in the Italian panorama, it follows the entire "research chain" dealing with fundraising, selection and funding of projects and the research activity itself carried out in the centers and laboratories of the Foundation. Telethon also develops collaborations with public health institutions and pharmaceutical industries to translate the results of research into therapies accessible to patients. Since its foundation, Telethon has invested 660,3 million euros in research, has funded 2,960 projects with 1,720 researchers involved and 630 diseases studied. To date, thanks to Fondazione Telethon, the first gene therapy with stem cells in the world has been made available, thanks to the collaboration with the pharmaceutical industry: since 2023 Fondazione Telethon has gained the marketing authorization. Strimvelis, this is the commercial name of the therapy, is intended for the treatment of ADA-SCID, a severe immunodeficiency that compromises the body's defenses from birth. Another gene therapy resulting from Telethon research made available is the one for a serious neurodegenerative disease, metachromatic leukodystrophy, with the commercial name of Libmeldy. Another therapeutic approach is in an advanced stage of clinical trials for Wiskott-Aldrich syndrome, another immunodeficiency. Other diseases on which the gene therapy developed by Telethon researchers has been evaluated in patients include beta thalassemia and two metabolic diseases of childhood, mucopolysaccharidosis type 6 and type 1. In addition, within the Telethon institutes a targeted therapeutic strategy is being studied or developed for other genetic diseases, such as haemophilia or various hereditary vision defects. In parallel, the study of basic mechanisms and potential therapeutic approaches for diseases still unanswered continues in all laboratories funded by Telethon.  

About Orchard Therapeutics

At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patient’s own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSK’s rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard is advancing a pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit www.orchard-tx.com, and follow us on Twitter and LinkedIn.

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 intramuscular 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.

3https://www.statista.com/statistics/253401/number-of-live-births-in-the-eu/.

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