Catalyst
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StrimvelisTM receives European marketing authorisation to treat very rare disease, ADA-SCID
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Additional Information
GlaxoSmithKline (GSK), Fondazione Telethon (Telethon) and Ospedale San Raffaele (OSR) today announced that the European Commission has approved Strimvelis, the first ex-vivo stem cell gene therapy to treat patients with a very rare disease called ADA-SCID (Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency). A child born with ADA-SCID does not have a healthy, fully-functioning immune system and as a consequence, is unable to fight off everyday infections. Strimvelis (autologous CD34+ cells transduced to express ADA) is the first corrective gene therapy for children to be awarded regulatory approval anywhere in the world. It is indicated for the treatment of patients with ADA-SCID for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.
The marketing authorisation decision was based on data collected from 18 children treated with Strimvelis. A 100% survival rate at three years post-treatment with Strimvelis was observed for all children in the pivotal study (n=12) and every child receiving the treatment who contributed to the marketing authorisation data package is alive today (n=18), with a median follow-up duration of approximately seven years. Full results of the analysis have recently been published in BLOOD1.
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Catalyst Date
Occurred on:
May 27, 2016
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Related Keywords
Cd34+ Cells Transduced, Evere Combined Immunodeficiency Due To Adenos
