GlaxoSmithKline Plc (GSK) and Human Genome Sciences Inc. (HGSI) won U.S. approval to sell Benlysta, a potential $3.6 billion product that will be the first new drug in 52 years for the auto-immune disease lupus.
The Food and Drug Administration approved the medicine for treatment of systemic lupus erythematosus, the most common form of the disorder, the agency said yesterday in a statement. The therapy will be available within about two weeks, the companies said in a statement.
Benlysta is the first drug designed specifically for lupus, an incurable condition that affects 1.5 million Americans and 5 million people worldwide. Most treatments, including steroids to ease inflammation, were used for other disorders when they were cleared for lupus more than a half-century ago.
“It’s a landmark achievement for Human Genome Sciences and a testament to what well-managed, productive biotech companies can achieve,” Mark Schoenebaum, an analyst with ISI Group in New York, said in an e-mail.
Glaxo shares rose 9 pence, or 0.8 percent, to 1,193 pence at 8:14 a.m. in trading in London, where the company is based. Before today, the stock had fallen almost 3 percent this year, including reinvested dividends.
Shares of Rockville, Maryland-based Human Genome rose 1 cent to $25.68 at 4 p.m. New York time in Nasdaq Stock Market trading before the FDA’s announcement. Extended trading was halted before the statement’s release.
Sales of Benlysta, an intravenous product also known as belimumab, may reach $2.3 billion in the U.S. and $3.6 billion worldwide in 2015, Maged Shenouda, an analyst with Stifel Nicolaus in New York, said Feb. 25 in an interview.
Benlysta’s initial U.S. market will be about 200,000 people with moderate to severe systemic lupus, Barry Labinger, Human Genome’s chief commercial officer, said yesterday during a conference call. The drug will cost about $35,000 a year for an average patient, Labinger said during the call.
“This is a historic day for the millions of people with lupus and their families around the world who have waited more than 52 years for a treatment breakthrough,” said Sandra Raymond, president of the Washington advocacy group Lupus Foundation of America. Benlysta’s approval “is a significant first step toward reaching our goal of developing an arsenal of new, safe, effective, and tolerable treatments.”
Black patients in the U.S. didn’t appear to respond to Benlysta in clinical studies, though the trials lacked sufficient numbers to establish any conclusions, the FDA said in its statement. The companies agreed to conduct an additional study of these lupus patients to further evaluate the safety and effectiveness of the drug, the agency said.
The disease is linked to an overproduction of proteins known as autoantibodies that lead the body to attack healthy cells. Benlysta isn’t recommended for patients with severe active lupus nephritis or severe active central nervous system lupus, or those taking other biologics or the chemotherapy drug cyclophosphamide, the companies said in the statement.
Developing and testing new lupus drugs poses unique challenges because no two people experience the disease in the same way, Raymond said. Symptoms range from rashes to joint swelling to kidney difficulties and tend to come and go.
A 2002 Lupus Foundation survey of more than 1,000 members found that 40 percent take at least six medications to treat their symptoms.
The steroid prednisone and some antimalarial drugs developed for other diseases won FDA approval for lupus more than a half-century ago. Immune-system suppressants used in transplant patients are among drugs prescribed for lupus without U.S. clearance, a so-called off-label use that may not be covered by health insurance plans.
Potential lupus drugs that preceded Benlysta had a history of failures. Rituxan, an arthritis and cancer drug from Basel, Switzerland-based Roche Holding AG (ROG) and Biogen Idec Inc. (BIIB) in Weston, Massachusetts, failed a lupus clinical trial in 2009.
Also falling short were Petah Tikva, Israel-based Teva Pharmaceutical Industries Ltd. (TEVA)’s Edratide, Redwood City, California-based Genelabs Technologies Inc.’s Prestara and Riquent, developed by La Jolla Pharmaceutical Co. (LJPC) and BioMarin Pharmaceutical Inc. (BMRN)
Lupus patients “witnessed the disappointment of one source of hope after another,” David Stump, Human Genome’s executive vice president for research and development, said on the conference call. “Today’s action by the FDA changes all that.”
Benlysta became the first experimental lupus drug to work in late-stage clinical trials after Human Genome and Glaxo, in collaboration with the FDA, designed a way to measure a reduction in overall disease activity.
Benlysta met its primary goal of reducing disease activity more effectively than standard treatments, the FDA said.
The FDA had evaluated Benlysta under a six-month priority review that was extended to nine months in December. While the agency typically takes at least 10 months to decide drug applications, it grants priority reviews to therapies that may provide major advances in treatment.
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