The FDA has rejected the new multiple sclerosis drug Lemtrada, saying the drugmaker didn't show the drug's benefits outweigh some serious risks.
The agency asked the maker, Genzyme, to test the drug in more clinical trials.
The drug was to be used for relapsing-remitting MS, the most common type.
MS affects about 400,000 people in the U.S. Blurred vision, balance problems, slurred speech, and memory problems of MS can be disabling.
Genzyme says in a news release it plans to appeal the FDA's decision on Lemtrada (alemtuzumab).
The drug has been approved in Canada, the European Union, and Australia. In the U.S. it received a mixed review from an FDA advisory committee in November. At that time, several members raised concerns about its safety.
The FDA had earlier approved the drug to treat leukemia, marketed as Campath.
Lemtrada is given as an infusion. It was expected to be used in MS patients who did not respond to other treatments.
Lemtrada's Side Effects a Concern
"We're disappointed by the decision," says Timothy Coetzee, PhD, chief advocacy, services, and research officer for the National Multiple Sclerosis Society. He says he's concerned about what this means for U.S. patients with MS.
In two clinical trials, Lemtrada reduced relapse rates over 2 years by about half compared to patients on another MS drug, Rebif (interferon beta-1a), Coetzee says. That reduction in relapses, he says, "is a pretty big deal."
A patient who relapses must go to the doctor and take time off work. Recovering from a relapse can also take some time, Coetzee says.
But the powerful impact of Lemtrada also can have downsides.
One potential side effect is called immune thrombocytopenic purpura, or ITP. This causes low platelet counts in the blood and can lead to dangerous bleeding. In the clinical trials, one patient died of ITP.
Genzyme put a special patient safety monitoring program in place after the death.
In the later research studies, most patients treated with the drug had mild to moderate infusion-related reactions, Coetzee says, including headache, rash, nausea, and fever. He says about 30% of those on Lemtrada developed thyroid-related problems. But those conditions, if caught early, can often be treated, he says.
Infections are another potential side effect, including shingles and upper respiratory, urinary tract, and sinus infections.
Coetzee says the National Multiple Sclerosis Society will continue to monitor the situation and to gather more information about the FDA's decision.
SOURCES: Timothy Coetzee, PhD, chief advocacy, services, and research officer, National Multiple Sclerosis Society. News release, Genzyme. Michael Racke, MD, professor and chair of neurology, Ohio State University, Columbus. FDA: "Genzyme Alemtuzumab Advisory Committee Briefing Document."©2013 WebMD, LLC. All Rights Reserved.
