Levodopa, the gold-standard medicine for Parkinson’s disease, has helped millions of patients since the 1970s manage the neurodegenerative disease.
But there’s a caveat. Nothing is available that can slow or reverse the loss of brain cells, and what’s more, levodopa’s effects wane over time. So-called “off” episodes, when the drug isn’t working, can become more common and severe. Patients can have trouble thinking, grow anxious, or worse, “freeze” up while walking. Imagine trying to work or plan a family outing not knowing if or when your medication might wear off. “People don’t go out because they’re afraid,” says Katie Kompoliti, professor of neurological sciences at Rush University Medical Center in Chicago.
This is a problem that two new treatments, currently under FDA review or close to it, are trying to address. Inbrija, from Ardsley, NY-based Acorda Therapeutics (NASDAQ: [[ticker:ACOR]]), and APL-130277, from privately-held Marlborough, MA-based Sunovion Pharmaceuticals, are reformulated versions of well-known Parkinson’s drugs developed to rescue patients from these off episodes. Acorda just presented the latest data from Inbrija at the American Academy of Neurology’s yearly meeting on Tuesday; its drug could be approved by October.
Sunovion filed for FDA approval in March, and hasn’t yet disclosed the full details from its successful Phase 3 studies. “These drugs are going to be a big deal for patients” who don’t have convenient rescue therapies right now, says Rachel Dolhun, the vice president of medical communications for the nonprofit Michael J. Fox Foundation.
Parkinson’s experts interviewed by Xconomy and not involved with either program are optimistic that both drugs can fill a need, but they question how much additional benefit the drugs will offer compared to levodopa, especially given how easy it is to take levodopa and its relatively mild side effects. They also want to see if Parkinson’s patients, with their impaired movement, will actually be able to use the drugs when needed, and whether the drugs will work fast enough to be worth the high prices they’re both likely to command.
“The clinical trial results are promising, but real-world experience will give us our best answer of how successful these drugs are going to be,” says Meredith Spindler, an assistant professor of clinical neurology at the University of Pennsylvania School of Medicine’s Parkinson’s Disease and Movement Disorders Center.
Parkinson’s affects an estimated 1 million people in the U.S., according to the nonprofit Parkinson’s Foundation. Acorda estimates that about 350,000 of these patients experience “off” periods. The currently available options for patients: apomorphine (Apokyn), an injectable rescue drug; taking more levodopa pills or extended release versions; deep brain stimulation; and Duopa, a surgically implanted infusion pump from AbbVie (NYSE: [[ticker:ABBV]]) that delivers levodopa directly into the small intestine for 16 straight hours.
Some patients use other tricks, like dissolving levodopa in water or drinking it with soda on an empty stomach, hoping for a quick effect.
All of these methods have problems, experts say. Deep brain stimulation, for instance, only works in people who respond well to levodopa, says Kompoliti. There’s no guarantee that people will respond to more pills; responding could depend on a variety of factors, like what patients eat or drink before taking the drugs. And even though the injectable apomorphine is very effective and fast-acting, patients tend to shy away. “It’s very hard for these people to give themselves injections,” Kompoliti says. It also causes side effects like nausea, light-headedness, and low blood pressure.
If Inbrija and APL-130277 can
