Fondazione Telethon and Orchard Therapeutics 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 17 by the European Commission 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 Teleth on 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.