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Case Study: Strimvelis

A successful public-private partnership bringing ATMP to market

In 2016, Strimvelis (autologous CD34+ cells transduced to express ADA) was approved in Europe to treat ADA-SCID (Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency). ADA-SCID is a rare immunodeficiency and estimated to occur in about 15 patients per year in Europe.

Strimvelis is the first ex vivo gene therapy to be licensed, and its success is the result of a joint effort among different stakeholders. The gene therapy for treating ADA-SCID was originally developed in Milan by the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), a joint venture between Fondazione Telethon (Telethon) and Ospedale San Raffaele (OSR). The development was then taken forward by GSK through a strategic collaboration formed in 2010 between GSK, OSR and Telethon. This alliance has led to the approval of Strimvelis as well as a research pipeline of more ex vivo gene therapies for treating disorders including Metachromatic Leukodystrophy (MLD) and Wiskott-Aldrich Syndrome (WAS). See also: Case Study: Libmeldy (in development)

Here we collated existing information about each stage of development for Strimvelis as a guide for the research community to learn about its development process, and how the collaboration between academia and industry can overcome the many hurdles in developing new ATMPs. Read the following resources to learn more:

Key Resources

Preclinical Research

An in Vivo Model of Somatic Cell Gene Therapy for Human Severe Combined Immunodeficiency

  • preclinical proof-of-concept that gene therapy for treating ADA-SCID was feasible and had an acceptable safety profile

Other Articles

Strimvelis: learnings from a cell and gene therapy forerunner

  • An interview with Dr Sven Kilit, the VP and Development Head for the Cell and Gene Therapy division of GSK

NA-ATTC Pathways from Translational Research to Industrialisation

  • This document describes the different stages involved in ATMP product development from bench to bedside
  • Page 7 of the document contains a development timeline for Strimvelis® 


Orphanet: Severe combined immunodeficiency due to adenosine deaminase deficiency

  • Summaries and detailed information on this disease are available in several other languages.

Orphanet: Research and trials

  • search for 'Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency'' within 'Research projects', 'Clinical trials', 'Registries & biobanks', 'Platforms', and 'Download dataset' tabs for relevant information. 

Regulatory tools and measures applied pre-authorisation

ATMP Classification: Strimvelis is classified as Gene Therapy Medicinal Product (GTMP) 

  • This EMA report provides information on ATMP approvals, classifications, and more.

Orphan designationStrimvelis (autologous CD34+ cells transfected with retroviral vector containing adenosine deaminase gene) was designated as an orphan medicinal product for the treatment of severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID)

Paediatric Investigation Plan (PIP): Information on Strimvelis's PIP

EMA Authorisation Details

Summary of the European public assessment report (EPAR) for Strimvelis

  • This summary explains how EMA assessed the medicine to recommend its authorisation in the EU and its conditions of use

Received positive opinion from Committee for Advanced Therapies (CAT) and Committee for Medicinal Products for Human Use (CHMP) at EMA:

Strimvelis Product Information 

  • Strimvelis, INN-Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence
  • Strimvelis product information document contain information including: summary of product characteristics, manufacturing authorisation holder responsible for batch release, conditions of the marketing authorisation, labelling and package leaflet. 


Direct healthcare professional communication (DHPC)

  • it contains important information for healthcare professionals prescribing, dispensing or administering the medicine(s). It also includes a communication plan with details of intended recipients and the dissemination date.

Gene therapy in rare diseases: the benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCID

  • A unique, patient-focused design has been implemented in the Strimvelis registry design to achieve the retention rates and data completeness required for fulfilling pharmacovigilance requirements

HTA Evaluation

Strimvelis® for Treating Severe Combined Immunodeficiency Caused by Adenosine Deaminase Deficiency: An Evidence Review Group Perspective of a NICE Highly Specialised Technology Evaluation | SpringerLink

  • This paper describes the manufacturing company’s submission of clinical and economic evidence, the independent Evidence Review Group’s (ERG’s) review and the resulting NICE guidance.

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

  • Case study by the ICPerMed Project
  • Strimvelis as a best practice example, in terms of its successful translation and facilitated access programme.
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