The EMA has recommended granting a marketing authorization
("MA") in the EU for orphan-designated Strimvelis, 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. ADA-SCID is an ultra-rare immune disorder caused by a
faulty gene inherited from both parents that stops the production
of adenosine deaminase. Strimvelis is manufactured from a
patient's own immature bone marrow cells into which a normal
adenosine deaminase enzyme gene has been inserted. Following an
assessment by an EMA specialized scientific committee, the
Committee for Medicinal Products for Human Use ("CHMP")
adopted the opinion at its March 2016 meeting. Adopting the opinion
is an intermediary step on Strimvelis's path to patient access.
The CHMP opinion will now be sent to the European Commission for
the adoption of a decision on an EU-wide MA.
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