GlaxoSmithKline (GSK)'s gene therapy has received a recommendation for approval from an EU regulatory panel for its proposed treatment of Bubble Boy Disease. If the treatment actually got approved, it would be the world's first corrective therapy for the disease.
The panel, consisting of The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency and Committee for Advanced Therapies (CAT), released their positive evaluation result on Friday. That was a major milestone for the company's progress in their innovative efforts for the disease.
ADA-SCID (Adenosine Deaminase Deficiency-Severe Combined Immunodeficiency), or Bubble Boy Disease, is a severe and rare immunodeficiency in which a single genetic defect prevent sufferers from developing a strong immune system. That condition led to physical vulnerability, leaving sufferers very sensitive to infections. Currently, what is considered to be best available treatment is a bone-marrow transplant, as reported by Microcap Magazine. However, the difficulties lie in finding the well-matched donor to ensure success.
The new treatment proposed by GSK was a corrective ex-vivo gene therapy, which involves inserting a new, healthy gene, directly into the patient's stem cells. The therapy, called Strimvelis, is developed from a patient's young bone marrow cells, which will be injected with a normal enzyme gene, as elaborated by TechTimes. The cells with the healthy enzyme gene will then be injected back to the patient, initiating a development of immune and blood cells.
The positive opinion from the European regulatory panel is a major step ahead for Strimvelis. The therapy has been used by a group of scientists and doctors in Milan over the past 14 years. According to The Wall Street Journal, the developer team has treated 22 children with the therapy. All of them are still alive, most without needing any further treatment. On the other hand, sufferers with no treatment could barely survive for two years. The recommendation could pave a way for final approval in a few months.
As for now, Strimvelis can only be performed in Milan, particularly in the hospital where it was developed by a team of scientists and doctors. Upon acquiring the right to market the therapy in 2010, GSK aims to make the therapy available in several other places, and is working on methods to enable the procedure to be done remotely by sending the sufferer's stem cells to a facility for a gene therapy.
A gene therapy owned by GSK is one step closer to be approved in Europe after receiving a recommendation from a regulatory panel on Friday. The therapy would help treat patients with Bubble Boy Disease by injecting a normal enzyme gene to the patient's stem cell.
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