Case Study: Strimvelis

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

Gene Therapy in Peripheral Blood Lymphocytes and Bone Marrow for ADA− Immunodeficient Patients | Science

Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning | Science

• This study is the first demonstration of clinically successful gene therapy with conditioning.

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency

Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products

• This paper summarises the development history for Strimvelis.
• Figure 1 shows a Strimvelis development timeline.

Patient‐driven search for rare disease therapies: the Fondazione Telethon success story and the strategy leading to Strimvelis

• This paper illustrates Fondazione Telethon’s strategies and operational model of industrial partnership contributing to the Strimvelis success story.

Gene therapy using haematopoietic stem and progenitor cells

• A review on the recent advances in HSPC gene therapy for a range of diseases, including ADA-SCID.

Update on Clinical Ex Vivo Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases

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. 

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

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

Protocol assistance from Committee for Medicinal Products for Human Use (CHMP)

• See EMA's Scientific advice and protocol assistance
• See also EuroGCT's resource: Support for innovative medicines’ development at the EMA level

Orphan designation: Strimvelis (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)

• A public summary of positive opinion for orphan designation
  • Section 'Update history' contains the history of sponsorship.
• See EuroGCT's resource on: ATMPs and Orphan medicinal products

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

• Learn more about: Paediatric medicines and Paediatric investigation plans
• See EuroGCT's resource on 'ATMPs and paediatric medicinal products'. (sub-entry currently under development.) 

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:

• CAT's opinion on Strimvelis can be found in this CAT meeting minutes
• CHMP's Summary of opinion

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. 

Procedural steps taken and scientific information after the authorisation

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.

Indication on the European Union register of medicinal products for human use

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

Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency | Guidance | NICE

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.