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Glaxo's `bubble boy' gene therapy wins EU drug agency nod

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(Bloomberg) — GlaxoSmithKline PLC (NYSE:GSK) won the backing of the European Union’s drug regulator for its first gene therapy, which treats a rare disease that makes newborn babies unable to fight off everyday infections.

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The therapy, which will be marketed as Strimvelis, was recommended for approval by the European Medicines Agency’s Committee for Medicinal Products for Human Use, the London-based drugmaker said Friday in a statement. The European Commission usually follows the committee’s advice.

If approved in Europe, it would mark the first time patients anywhere in the world would have access to the therapy. Strimvelis, derived from patients’ own cells, is for an illness known as “bubble-boy disease” after a 1976 film starring John Travolta about a patient living in a sterile environment.

The Glaxo treatment aims to restore the ability to make healthy lymphocytes — a type of white blood cell essential to the body’s immune response. It’s the most advanced among a group of Glaxo products that seek to correct faulty genes that cause rare diseases and cancer. The company said it plans to file for approval with the U.S. Food and Drug Administration next year at the earliest.

Pricing models

About 15 infants are born with the disorder, which is officially known as severe combined immunodeficiency (ADA-SCID) syndrome and is caused by adenosine deaminase deficiency, in Europe each year, and global estimates range from 120 to 300 patients a year, according to Martin Andrews, head of Glaxo’s rare-diseases unit. Although the prevalence of ADA-SCID is low, Glaxo’s product could benefit the eight out of 10 patients who can’t find a matching donor for a bone-marrow transplant, which has the potential to cure patients, Andrews said.

Glaxo will explore new models for pricing the drug, Andrews said. One approach could be allowing amortized payments over the period the treatment is used. Another option is a risk-sharing program where payers get a refund if the therapy stops working or the patient dies, Andrews said.

The Glaxo therapy, which is administered in a one-time procedure, could offer patients a more affordable alternative to enzyme replacement, which requires weekly injections and can cost as much as 3 million pounds ($4.25 million) for one patient over 10 years, Andrews said.

Sales estimates

Strimvelis could see sales of 67.5 million pounds in 2020, according to analyst estimates compiled by Bloomberg.

The manufacturing platform and technology for Strimvelis can also be used for other gene therapies in Glaxo’s pipeline, including a therapy for cancer it’s studying in collaboration with Adaptimmune Therapeutics PLC.

“I can tell you it’s been very expensive to invest in this over the last six years,” Andrews said. “But actually we expect to spread the benefits across other medicines in GSK’s portfolio. By having a portfolio approach and by applying it to increasingly larger diseases, even though they may still be rare, it does become economically viable.”

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