When Is a Drug Worth a Fortune
When is a drug worth more than one hundred forty-five thousand dollars a year? For some, if it can prolong their lives. The new drug, Edaravone is only one of two drugs approved to treat amyotrophic lateral sclerosis, commonly known as ALS or Lou Gehrig's disease.
ALS is a progressive neurological disorder and is fatal within three to five years after diagnosis. It affects the motor neurons, which are nerve cells that control the voluntary muscles we use to chew, walk, breathe, and talk. In ALS, these motor neurons die and stop sending signals to muscles. Eventually these muscles waste away and people die from not being able to breathe. What makes ALS tragic is that the person is aware because they do not lose any of their higher brain function.
Until Edaravone was recently approved by the FDA, more than twenty years had passed without any significant new treatments for the disease. The federal agency had asked the drug's Japanese manufacturer to apply for approval in the US when it saw the effectiveness of the drug. In the clinical trial in Japan, Edaravone significantly reduced the progression of ALS in patients. It's intravenously infused daily for fourteen days followed by fourteen days of no medication. This cycle is repeated monthly.
Edaravone may be counteracting something called oxidative stress, which may be involved in the death of neurons in ALS. Oxidative stress is generated when cells metabolize and if not eliminated can damage and kill cells. Even though studies show the drug didn't benefit people in late stages of ALS, it worked best on those diagnosed within two years.
More Information
FDA approves drug to treat ALS
The U.S. Food and Drug Administration today approved Radicava (edaravone) to treat patients with amyotrophic lateral sclerosis (ALS), commonly referred to as Lou Gehrig's disease. 'After learning about the use of edaravone to treat ALS in Japan, we rapidly engaged with the drug developer about filing a marketing application in the United States,' said Eric Bastings, M.D., deputy director of the Division of Neurology Products in the FDA's Center for Drug Evaluation and Research. 'This is the first new treatment approved by the FDA for ALS in many years, and we are pleased that people with ALS will now have an additional option.'..
FDA approves second drug to treat ALS
The Food and Drug Administration announced in May it approved a new drug to help treat amyotrophic lateral sclerosis, commonly known as ALS or Lou Gehrig's disease...
ALS Fact Sheet
Amyotrophic lateral sclerosis (ALS) is a group of rare neurological diseases that mainly involve the nerve cells (neurons) responsible for controlling voluntary muscle movement. Voluntary muscles produce movements like chewing, walking, and talking. The disease is progressive, meaning the symptoms get worse over time. Currently, there is no cure for ALS and no effective treatment to halt, or reverse, the progression of the disease...