Strimvelis® Gene Therapy

Cross-border access to personalized treatment: the example of Strimvelis

The ex-vivo gene therapy Strimvelis® was approved in Europe in 2016 to treat children affected by severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) who lack a matching bone marrow donor. ADA-SCID is an inherited disorder caused by mutations in the ADA gene and results in a seriously compromised immune system.

An ultra rare disease: New patients per year affected by ADA-deficiency are estimated between 7 and 13 in Europe. Before the approval of Strimvelis® ADA-SCID was often fatal early in life for patients who lack a matching bone marrow donor.

Description of the „Just like home” programme at Fondazione Telethon

Contact: Stefano Benvenuti

“Strimvelis® represents a change of paradigm because, with a fresh product administered only at a single treatment centre, the patient has to travel, not the drug”

Michela Gabaldo, Head of Alliance Management & Regulatory Affairs at Fondazione Telethon

How the therapy works

The therapy involves collecting patients’ bone marrow derived CD34+ cells and modifying them to produce functional copies of the adenosine deaminase enzyme. Simplifying the procedure, this means to extract patient’s cells, manipulate the cells in a specialized and authorized laboratory to insert correct copies of the ADA gene and then reinfuse these modified cells into the patient. Because Strimvelis is a fresh product with a very limited shelf-life, its administration can occur only at the San Raffaele Hospital which is co-located with the only approved manufacturing facility in Europe. As summarized and simplified in the figure below, the overall treatment path requires around 4 months.

«Double» personalized medicine: Strimvelis is a compound targeting a specific genetic modification. The Patient’s own cells are both the starting material for the manufacturing of the therapeutic products and, after manipulation, the “drug” itself which is administered back to the patient.

Strimvelis scheme ®Fondazione Telethon

How much does it cost?

The overall cost of the treatment includes the cost of Strimvelis (officially € 594.000,00 – VAT excluded), the cost of hospitalization and short-term follow-up conducted at the centre in Milan, and the cost of travel and accommodation for the family for the period abroad. Most of the EU non-Italian patients therefore access the treatment using the EU Social Security Regulations 883/2004 and 987/2009  where the payment is directly covered by their National Health System/Social Insurance. In very few cases though the National Health System covers the travel and accommodation costs for the family that, considering a permanent stay at the clinical centre in Milan of approximately 3-4 months, can become a relevant barrier for equitable access to the treatment.

We need EU measures to coordinate and fund access to cross-border ATMP treatment.

Just like home

To overcome this limitation, Fondazione Telethon, the Italian charity which co-developed Strimvelis® together with Ospedale San Raffaele then licenced to GSK (then transferred to Orchard therapeutics), launched the “Just like Home” program. The programme supports the families coming to Milan by organizing travel, lodging and logistics, helping in getting visa and resident permits, giving psychological support and linguistic and cultural assistance before, during and after their stay in Milan. The “Just like Home” team includes a care coordinator, nurses, psychologists, administrative assistants, linguistic and cultural mediators and care givers.

Shana, patient with Strimvelis® treatment
In the photo above, Shana, one of the patients treated with Strimvelis® and hosted by the “Just Like Home program”, together with her parents in Milan, in the park around the Hospital. ®Fondazione Telethon

Explanatory notes

Molecular Tumour Board

For some cancer patients, e.g. those with very rare tumours, no standard therapy is available. These cases are discussed by experts from a wide range of diciplines in molecular tumour boards. Based on the molecular characteristics of the specific case, they try to find recommedations for off-label therapy or for an inclusion into an ongoing clinical trial.


Examples shown have been reviewed and selected by members of ICPerMed. However, ICPerMed does not take over any responsibility for the work performed or the data shown.

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