SILVER SPRING, Md. (Reuters) - Roche Holding AG ROG.VX won a U.S. panel's backing on Tuesday for a new rheumatoid arthritis drug, a potential blockbuster that could help the Swiss company diversify its business.
By a 10-1 vote, a committee of outside experts urged the U.S. Food and Drug Administration to approve the drug, Actemra, for moderate or severe rheumatoid arthritis, a disease that causes painful joint swelling.
The FDA usually clears drugs that win support from advisory panels. A final decision is expected by Sept. 18.
Actemra works differently from other drugs on the market in that it blocks interleukin-6 (IL-6), an immune-system protein involved in inflammation. Other widely used drugs block a protein called tumor necrosis factor (TNF).
Committee members said Actemra’s ability to relieve symptoms outweighed potential risks, but some urged tracking of patients’ liver function and cholesterol levels.
“The safety concerns are real, and I think there is going to have to be monitoring,” said panel member Dr. David Felson, clinical epidemiology chief at Boston University School of Medicine.
In Roche studies, laboratory tests showed elevated liver enzymes and increased LDL or “bad” cholesterol in some patients. None of the patients showed harm from those effects, FDA reviewers said.
Felson said he was worried about the higher LDL levels.
“I’d be nervous about it, especially because most of our patients are older patients” who face a higher risk of heart disease, he said.
Some panel members were concerned by intestinal tract perforations. Roche said the rate was no greater than would be expected in rheumatoid arthritis patients.
The drug also was linked to serious infections, a risk associated with other rheumatoid arthritis drugs.
SOLD IN JAPAN
Actemra could be reserved for patients who do not respond to current drugs that block a different protein, tumor necrosis factor (TNF), some panelists said.
Known generically as tocilizumab, Actemra is sold in Japan by Roche's partner, Chugai Pharmaceutical Co Ltd 4519.T, but has yet to reach the market in the United States or Europe.
Roche bills the injectable drug as a blockbuster seller that will help it diversify and reduce its reliance on cancer medicines such as Avastin and Rituxan, sold by its U.S. partner Genentech DNA.N.
Roche now owns a majority stake in Genentech and is trying to buy the outstanding shares.
Actemra’s peak sales could top $2 billion, according to some analyst estimates.
Roche earlier this month reported total company sales of 22 billion Swiss francs ($21 billion) for the first half of 2008.
Rheumatoid arthritis, or RA, causes pain, stiffness, swelling and joint damage. More than 21 million people worldwide, including 2.5 million Americans, are estimated to have the disease, Roche said. Some cases can be disabling.
Roche said Actemra would offer an alternative to current therapies including the TNF blockers, which do not help everyone or lose effectiveness over time.
“We know that new therapies with novel mechanisms of actions are still needed ... Many times, the best we can do is get 50 percent of our patients 50 percent better,” Dr. Kenneth Bahrt, global medical director for autoimmunity for Hoffmann-LaRoche Inc, told the panel.
In company-funded studies, more patients treated with Actemra reported at least a 20 percent improvement in symptoms after 24 weeks of treatment.
About 26 percent of placebo patients saw that level of benefit, compared with 59 percent treated with the highest Actemra dose. Patients in the studies also were given older drugs for the disease.
The committee’s recommendations for patient monitoring were consistent with Roche’s own proposals, Bahrt said in an interview.
Reporting by Lisa Richwine; Editing by Ted Kerr
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