Shire pump bypasses hurdle to sneak enzyme drug into brain

Shire is zipping ahead with clinical research of pump implants in the spine, potentially overcoming a natural hurdle to delivering its enzyme-replacement drug Elaprase into the brains of patients with the rare genetic disease Hunter syndrome, Bloomberg reported. Angus Russell, CEO of Shire, told the news service that the company is testing the pump in 15 patients with the syndrome whose illness has impacted the brain, and no safety red flags have been raised.

For patients with the disease, which causes buildups of sugar molecules in the body, Elaprase has been a breakthrough drug. Yet the blood-brain barrier keeps the enzyme treatment, which is traditionally given intravenously, from entering the brain. Patients lose mental functions as the disease progresses, and the effects of the disorder when it spreads to the brain can be deadly. With an experimental pump implanted at the base of the spine, the drug can enter the central nervous system as the treatment couldn't with the intravenous method of delivery, Bloomberg reported.

This not only helps patients, but it could also aid the rare-disease business that accounts for nearly a third of Shire's revenue. Shire, which acquired Elaprase as part of its $1.6 billion buyout of rare-disease drug developer Transkaryotic Therapies in 2005, is also testing the pump implant to deliver a drug called HGT 1410 to treat a rare metabolic disorder called Sanfilippo syndrome. And Russell told Bloomberg that products delivered with the pump could be "significant" to the business a decade from now.

"It sounds very exciting, like they're really being smart about life cycle management of their existing franchises," Navid Malik, an analyst at Merchant Securities Ltd. in London, told Bloomberg. "If they can do the same with other rare disease areas that are affecting the brain, they could have a nice platform technology here."

- read the report

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