(NEW YORK) -- Last Friday, after more than four years of review, the U.S. Food and Drug Administration approved the first and only therapy designed to improve symptoms of Pseudobulbar Affect (PBA), a neurological disorder that causes involuntary laughter and sudden, uncontrollable crying in patients.
The new medication, Nuedexta from Avanir Pharmaceuticals in Aliso Viejo, Calif., combines the over-the-counter cough suppressant dextromethorphan with quinidine, a generic drug used to restore normal rhythms to erratically beating hearts.
In clinical trials, Nuedexta was safe, reduced the frequency and severity of PBA episodes, and showed a significant advantage over a placebo. But in 2006, the FDA expressed concerns that higher doses of the drug combination raise the risks of dangerous cardiac rhythms. By reducing the doses of quinidine from 30 milligrams to 10 milligrams, Avanir satisfied the FDA's concerns about cardiac risks. In a Phase III clinical trial of the drug with MS patients, half the study participants who got the drug reported no PBA episodes in their last two weeks of the study.
"This is wonderful news for all the patients who suffer from PBA," said Dr. Erik Pioro, a Cleveland Clinic neurologist who specializes in ALS and related disorders. "They will now have an effective, safe, and well-tolerated treatment for this distressing and extremely isolating condition."
In the absence of something better, doctors have treated PBA with off-label prescriptions for antidepressants or levodopa, which boosts levels of the neurotransmitter dopamine. But these have a range of side effects and haven't been subjected to large studies in PBA patients.
Avanir plans to make the drug available in the first quarter of 2011, said CEO Keith Katkin, and will start by providing 30-day samples to select doctors who treat PBA. Avanir estimates the drug will run $3,000 to $5,000 a year for patients, or about $250 a month. Patients with limited incomes will get the drug free through a patient assistance program, Katkin said.
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