1 April 2016 - The EMA has recommended granting a marketing authorisation in the European Union for a new gene therapy for the treatment of patients with adenosine-deaminase-deficient severe combined immunodeficiency (ADA-SCID), who have no matching donor for a stem cell transplant. Children born with this disorder have virtually no immunity to fight off everyday bacterial, fungal or viral infections.

ADA-SCID is an ultra-rare immune disorder, caused by a faulty gene inherited from both parents that stops the production of adenosine deaminase. Without this enzyme, the body is unable to break down a toxic substance called deoxyadenosine. The toxin builds up and destroys infection-fighting lymphocytes. Children born with ADA-SCID are severely impaired in their ability to fight infections. The disorder can also lead to various non-immunological health problems, including a failure to grow and develop normally, hearing loss and liver and kidney problems.

The assessment of Strimvelis was carried out by the Committee on Advanced Therapies (CAT), EMA’s specialised scientific committee for advanced therapy medicinal products, such as gene or cell therapies. At its March 2016 meeting, the CAT recommended the adoption of a marketing authorisation for Strimvelis. The CAT’s recommendation was considered by the Committee for Medicinal Products for Human Use which agreed with the CAT and issued a positive opinion.

For more details, go to: http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/04/news_detail_002504.jsp&mid=WC0b01ac058004d5c1