Sept. 11 (Bloomberg) -- Shire Plc, the U.K. maker of the hyperactivity drug Adderall, may reap a $50 million windfall in 2010 after its treatment for the rare genetic disorder Gaucher disease was rushed to the market a year earlier than planned because of a manufacturing disruption at Genzyme Corp.

Genzyme’s setback also hands Shire the chance to garner $139 million in revenue in 2011, according to Citigroup Inc., which says Shire’s velaglucerase alfa may grab 15 percent of the $1.1 billion market for Gaucher disease treatments by 2012.

“The question for us isn’t how much market share we’re going to get in the short term, the question is how much can we get to the market,” Shire Chief Executive Officer Angus Russell said in an interview this week. “I’m pretty sure demand will exceed that, hence the need for more drugs.”

The introduction of the Gaucher drug is accelerating Russell’s plan to cut Shire’s dependence on attention deficit hyperactivity disorder drugs. Hyperactivity drugs Adderall and Vyvanse made up half of 2008 sales.

Revenue sank 19 percent in the second quarter on generic competition to Adderall. Shares of Basingstoke, England-based Shire have risen 13 percent in the past year, compared with a 9.4 percent drop in the Bloomberg Europe Pharmaceutical Index.

The majority of Gaucher patients took Genzyme’s Cerezyme until a virus at a Genzyme factory prompted regulators to halt output in June. The virus, Vesivirus 2117, originated in a nutrient used in the manufacturing process. With the resulting shortage, the U.S. Food and Drug Administration allowed Shire to rush velaglucerase alfa to the neediest patients even though the drug isn’t approved. The first patient began treatment this week.

Enzyme Replacement

Gaucher disease is an inherited disorder caused by low levels of an enzyme responsible for breaking down a fatty substance that can build up in the spleen, liver and brain, according to the U.S. National Institute of Neurological Disorders and Stroke. Depending on the type of the disease, it can lead to bruising, anemia, seizures, brain damage and death.

It’s one of the world’s rarest diseases, affecting an estimated 5,000 to 8,000 people globally, said John Barranger, a Gaucher specialist at the University of Pittsburgh. Cerezyme and velaglucerase alfa are both enzyme replacement therapies.

Shire said Sept. 8 it could supply from 300 to 600 patients, depending on dosing variations. The company is accelerating plans for a new manufacturing plant in Lexington, outside of Boston, to help boost capacity for velaglucerase alfa, Russell said. He confirmed Citigroup’s expectation that Shire will expand capacity to 900 patients next year and more than 1,000 patients after that.

Added Sales

“We do have a very big manufacturing facility we hope will come on stream the back end of next year, perhaps,” Russell said. “If that’s successful and we manage to achieve our objectives, we hope to achieve broadly those sets of numbers.”

The earlier launch could add 1 to 2 percent to total Shire sales in the “the short term,” he said. Shire had revenue of $629.7 million in the second quarter.

Genzyme started rationing Cerezyme in June after the closing of the company’s Allston Landing plant in Boston for decontamination, alarming patients who relied on it as the only enzyme replacement treatment available.

“Patients are concerned that they will begin to deteriorate, especially because much of this deterioration can be irreversible,” said Barranger, who helped develop the first enzyme replacement therapy for Gaucher patients in the 1970s.

Safety Plan

Shire submitted a safety plan to the FDA to allow velaglucerase to be available while Genzyme makes a new supply, which the Cambridge, Massachusetts-based company has said should reach patients in November. A Genzyme spokesman didn’t return a call for comment.

“We’re working as fast as we can to accommodate the need where we can,” Sylvie Gregoire, head of Shire’s human genetic therapies, or HGT, unit, said in an interview. Shire said Sept. 1 it applied to the FDA for approval of the drug and would seek European approval by year’s end.

Hyperactivity “remains very important for Shire but I think it’s kind of rational and sensible that the rest of the business gets more focus,” Jack Scannell, a Sanford C. Bernstein & Co. analyst in London, said in an interview. “The change in the perception has been something that Shire has been trying to do for ages and now Genzyme has done it for them.”

Scannell has a “market perform” rating on the shares.

Strategic Goal

“It’s always been a strategic goal to get vela into the market; the only benefit now is that’s coming a year ahead of time,” said Russell, the Shire CEO. “That’s obviously good as that down the road can lead to faster profits, faster cash flow, and more reinvestment into the HGT business in the longer term.”

Cerezyme, approved in 2001, costs about $200,000 annually per patient. Citigroup’s Peter Verdult, who has had a “buy” rating on Shire since April 2007, estimates Shire will discount its drug by about 25 percent to Cerezyme’s price, allowing Shire to grab more market share.

“The drug is proven to be equivalent to Genzyme’s product so I don’t see any major need for significant discounting,” said Russell, who declined to specify the price. “There will still be other strategic issues down the road so those final decisions haven’t been made.”

Shire on Aug. 3 said initial data from final-phase trials showed velaglucerase alfa met its main goals of boosting the oxygen-transporting protein hemoglobin in the blood, while managing swelling in the spleen and liver. Full data will be presented this year, and the company is in talks with regulators for approval expected in the U.S. early next year and in Europe by the end of 2010.

“There’s really nothing in the way of having good discussions,” said Gregoire, adding that Shire will hire a “small” number of sales people to promote the drug. “There’s a need and the data is positive.”

To contact the reporter on this story: Trista Kelley in London at tkelley2@bloomberg.net

Last Updated: September 10, 2009 19:02 EDT