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LONDON (Alliance News) - GlaxoSmithKline PLC on Friday said it received a positive opinion from a European advisory panel, recommending marketing authorisation for Strimvelis to treat patients with ADA-SCID, a very rare disease which prevents children from developing an immune system that can fight off everyday infections.
The medicine is a stem cell gene therapy created for an individual patient from their own cells, Glaxo said in a statement, which is intended to correct the root cause of the disease. ADA-SCID is estimated to occur in about 15 patients per year in Europe, Glaxo said.
The opinion was received from the Committee for Medicinal Products for Human Use of the European Medicines Agency, in conjunction with the Committee for Advanced Therapies.
Glaxo said the medicine will be commercialised under the brand name Strimvelis, should approval be given by the European Commission.
"We welcome this opinion from the CHMP which is an important step towards making Strimvelis available to the children living with this incredibly rare and fatal condition. Going forward, we hope to apply this gene therapy platform technology across other diseases, enabling many more patients to benefit from this innovative treatment approach," Patrick Vallance, president, R&D, said in a statement.
Martin Andrews, head of the rare disease unit at Glaxo, said: "This positive opinion is a major milestone in GSK's commitment to the development of innovative, transformative medicines. If approved, Strimvelis will become the first corrective ex-vivo gene therapy for children to achieve regulatory approval anywhere in the world. With our shared mission and complementary expertise we believe this collaboration will continue to deliver much needed new medicines for patients with rare genetic diseases."
Until the EU Commission decision, Strimvelis will remain an investigational gene therapy which is not approved for use anywhere in the world.
Shares in Glaxo were down 0.4% at 1,406.50 pence on Friday afternoon.
By Samuel Agini; samagini@alliancenews.com; @samuelagini
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