Bloomberg News

Shire Tests Spinal Pump in ‘Next Frontier’ for Rare Disease Drug

October 19, 2011

Oct. 20 (Bloomberg) -- Shire Plc, a maker of medicines for rare genetic diseases, said it expects results next year from an early-stage trial that involves implanting a pump in patients’ spines to deliver drugs to their brains.

About 15 patients are participating in the trial of a version of Shire’s Elaprase in people with Hunter syndrome that has spread to their brain, Chief Executive Officer Angus Russell said in an interview in London yesterday. Almost two years after the study started, no serious side effects have been reported, Russell said.

The study is the first to deploy the technology to carry treatment to the brain, and involves implanting a pump at the base of the spine that the patient can then use to release drugs into the central nervous system. It’s part of Dublin-based Shire’s plan to expand the use of approved treatments such as Elaprase for people who have few treatment options when their disease causes complications in the brain, an advancement that can be fatal.

“What we’re looking for is to push the boundaries really of the next frontier of treating rare genetic diseases,” Russell said.

While larger drugmakers such as Pfizer Inc. and GlaxoSmithKline Plc are facing price cuts and the loss of patent protection on some of their biggest-selling drugs, analysts are expecting Shire to book full-year sales growth of 21 percent on expanding demand for its treatments, many of which are the only available for patients with rare diseases.

Sanfilippo A

Shire is also testing the pump technology in patients with Sanfilippo A syndrome, a rare and severe metabolism disorder, Russell said. That trial is using a drug known as HGT 1410.

“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, said in a telephone interview today. “If they can do the same with other rare disease areas that are affecting the brain, they could have a nice platform technology here.”

The stock has almost doubled in the past two years, giving the company a market value of 11.3 billion pounds ($17.9 billion) and making it the best performer in that period on the Bloomberg Europe Pharmaceutical Index of the region’s 17 biggest drugmakers.

Shire gained Elaprase with its $1.6 billion purchase of Transkaryotic Therapies Inc. in 2005. It now has four approved products from that deal, representing almost a third of revenue.

‘Much Bigger Piece’

If the pump technology works, “in a decade’s time, armed with that capability, then this could be a much bigger piece of our business,” Russell said.

Shire is interested in further acquisitions in the area of regenerative medicine, Russell said, following the company’s $750 million purchase of Advanced BioHealing Inc. in May. Advanced BioHealing’s lead product is Dermagraft, a skin substitute used to treat foot ulcers.

“We see that as a very new, fast-growing area,” he said. “We’re certainly looking for other assets that we could bring in to put in on top of the Advanced BioHealing acquisition. And then generally we’re always looking for other assets that would be associated with areas of business we’re already in,” such as attention deficit hyperactivity disorder, digestive tract illnesses and rare diseases.

--Editors: Kristen Hallam, Sara Marley

To contact the reporter on this story: Simeon Bennett in Geneva at

To contact the editor responsible for this story: Phil Serafino at

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