--FDA approves J&J's Sirturo but requires prominent warning label
--Drug is unlikely to be a big moneymaker but may accrue goodwill to J&J
--Fewer than 100 Americans suffer from the disease, multidrug resistant TB
(Adds details on safety risks in paragraph eight, and comments from consumer group Public Citizen and FDA official in paragraphs 13-16.)
By Joseph Walker and Nathalie Tadena
The U.S. Food and Drug Administration approved Johnson & Johnson's (JNJ) drug to treat a form of resistant tuberculosis that is uncommon in the U.S. but growing globally.
The drug, Sirturo, will treat patients with multidrug resistant tuberculosis, or MDR-TB, a possibly fatal disease that affects as many as 630,000 people world-wide who can't be cured with existing therapies alone. Tuberculosis is a bacterial infection that mostly involves the lungs.
Sirturo, also known as bedaquiline, is not expected to be a large moneymaker for Johnson & Johnson (JNJ), one of the world's largest health-care conglomerate, because the disease is prevalent in poorer countries unable to afford high prices for the treatment. Providing the drug, though, could help the company create goodwill among regulators, governments and patients around the world.
"The commercial opportunity is very limited," said Pamela Van Houten, a J&J spokesperson. "This is part of our commitment to advance innovative medicines that help address serious public health issues."
The company said it expects to begin selling Sirturo in the second quarter of 2013, and won't announce its sales price until then. Fewer than 100 Americans have MDR-TB, according to J&J, but FDA approval is likely to strengthen the chances for approval in regions where the disease is more prevalent, including India, China and Eastern Europe.
However, the drug's potential side effects, including an increased risk of death, has raised concerns among members of the FDA. The agency will require that a prominent warning label be placed on the drug, informing consumers that patients taking Sirturo in clinical trials saw increased rates of fatalities.
"Because the drug also carries some significant risks, doctors should make sure they use it appropriately and only in patients who don't have other treatment options," said Edward Cox, an FDA official.
About 11.4% of patients taking Sirturo died in during clinical trials, compared to 2.5% taking placebos.
The FDA granted Sirturo accelerated approval, which is a type of temporary approval for diseases that don't have viable treatment options, based on less clinical data than required for traditional drug approvals.
The accelerated approval was based on data from a pair of Phase 2 studies--one of which showed 77.6% of patients in the treatment group reaching treatment success after 24 weeks compared with 57.6% of those in the placebo group.
It is still unclear, though, whether Sirturo caused the increase death rate, said Mel Spigelman, chief executive of the Global Alliance for TB Drug Development, which is partnering with J&J to develop Sirturo in more common strains of tuberculosis.
"It looked more likely that this was just a bad coincidence," Dr. Spigelman said. "But you have to be careful, and the monitoring will have to be rigorous."
Last month, an FDA panel supported a type of early approval for J&J's experimental drug; however, panel members were divided on whether Sirturo played a role in deaths seen in clinical studies.
"A greater number of deaths in the bedaquiline arm is concerning," FDA Deputy Director of Anti-Infective Products, said in a presentation to the panel. "The role of bedaquiline in deaths where hepatoxocity and cardiac failure are contributory could not be ruled out."
The consumer group Public Citizen, which had been strongly opposed to approval, said Monday that it was disappointed in the FDA's decision.
"There were five times more deaths in the drug group than in the placebo group," said Sidney Wolfe, director of health research at Public Citizen. "I don't see how [the FDA] can be so dismissive about it."
Tuberculosis usually is treated with a combination of four drugs for at least six months, according to the National Heart, Lung and Blood Institute. Some forms of tuberculosis, however, can't be treated with currently available drugs, which the FDA has said "threatens to erode the progress made in the world-wide control of tuberculosis."
Sirturo is designed to work in a new way to treat tuberculosis and would be used with other drugs.
Shares of J&J rose 27 cents to $69.75.
Write to Joseph Walker at firstname.lastname@example.org
Subscribe to WSJ: http://online.wsj.com?mod=djnwires