The first new treatment for lupus in more than 50 years won the blessing of a federal regulatory panel today, providing good news for patients and the drug's developers.
The U.S. Food and Drug Administration panel voted that the drug, known as Benlysta, is safe and effective. Benlysta works well enough to outweigh risks of suicide, infection and cancer, and should be approved, the panel recommended.
The full FDA is scheduled to decide on the drug next month, and usually follows the recommendation of its advisory panels.
Analysts say the drug is likely to be approved because it addresses an unmet medical need, and some expect annual sales to exceed $2 billion eventually.
“The efficacy is mild, but there is a need for a drug even with mild efficacy, given the current state of therapies,” Lenore Buckley, a professor of internal medication and pediatrics at Virginia Commonwealth University in Richmond, told Bloomberg News before today's vote.
FDA's experts stressed that Benlysta will not work for all patients and that more effective therapies are still needed. The panel was worried that the drug didn't appear to work very well in African-Americans.
“I think it's very exciting that this is the first new drug in five decades, but it's not magic bullet,” Dr. Maria Suarez-Almazor of the MD Anderson Cancer Center in Houston, told the Associated Press.
The injectable drug to treat the incurable autoimmune disease would provide an important new revenue source for its developers, Human Genome Sciences and GlaxoSmithKline. Since 2006, the companies have been working together to test the drug, and will split future profits.
GSK officials would welcome positive news after a string of recent stumbles, including a $750 million settlement related to problems at a drug-manufacturing plant in Puerto Rico.
For Deirdre Connelly, the British company's top executive at its North American headquarters in Research Triangle Park, winning approval of the drug would be an important professional victory, but also a personal one. Her father suffered from lupus for 16 years and died from it in 1994.
GSK has been handling some legal, regulatory and other support work for Benlysta out of RTP, where it employs nearly 5,000 people. The clinical work has been handled in Pennsylvania.
Trading of Human Genome's shares was halted today while the FDA panel met. GSK's American depositary receipts fell 69 cents to close at $38.54 today, but rose in after-hours trading.
In August, the FDA granted the drug a prior review designation, a step the agency uses to accelerate its review of promising new treatments. The FDA is expected to issue its decision Dec. 9.
Company executives suggested the drug could be available in early 2011.