On May 5, 2017, the Food and Drug Administration (FDA) approved Radicava (or edaravone) as a new drug for amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease. It is extremely important news for those with the condition as it’s only the second drug the FDA has ever approved for ALS. Riluzole was the first, but its approval happened over two decades ago.
It took a long time to find an alternative drug, but the wait for people living with ALS is over. While Riluzole increases a patient’s survival time by up to three months, there is no information on whether Radicava will also boost lifespan. Patients receive Radicava via intravenous infusion, and it costs just over $145,000 per annum.
What Is ALS?
ALS is an extremely rare disease that affects anywhere between 12,000 and 15,000 people in the United States, according to the Centers for Disease Control and Prevention (CDC). Also called Lou Gehrig’s disease, it gets the name from the famous baseball player who retired in 1939 due to the condition. It attacks and kills the body’s nerve cells that control its voluntary muscles that produce movements such as walking, talking, breathing, and chewing.
The nerves become unable to activate specific muscles, and over time, the muscles grow weak; paralysis is the result. ALS is a progressive disease, which means it gets worse over time. The majority of people with the condition die from respiratory failure within 3-5 years. Doctors diagnose approximately 5,000 new instances of ALS per year with the majority of people in the 55-75 age range. To date, scientists have not determined the cause of most ALS cases.
What Does the Science Say About Radicava?
Patients receive Radicava through an intravenous infusion provided by a healthcare professional. Initially, patients receive daily doses for 14 days followed by a 14-day drug-free period. After that, patients receive one dose for 10 of the next 14 days followed by another 14 days without the drug. Each infusion takes one hour and costs $1,100, which equates to over $145,000 in a year.
Radicava received approval after results of a six-month clinical study in Japan showed significantly reduced rates of physical decline. The study included 137 participants who received either Radicava or a placebo. A trial extension compared patients on Radicava for 48 weeks with those who took a placebo for 24 weeks before switching to the drug for another 24 weeks.
According to measurements of the ALS Functional Rating Scale-Revised, patients on Radicava for the entire 48 week period showed an almost 60 percent lower relative risk of death or certain disease progression events along with a much lower rate of overall physical decline. For the record, an example of a disease progression event is the loss of your ability to use your arms.
Potential Side Effects
Although the effect is relatively modest at present, experts on the disease suggest it is a significant development. Radicava will almost certainly become a popular drug on the marketplace despite its price tag. In contrast, Riluzole costs $405 for 30 pills, and as patients take two a day, it works out at $27 a day or $9,855 per annum. Radicava is almost 15 times more expensive.
However, Radicava appears to carry fewer adverse side effects. Riluzole can cause asthma, nausea, vomiting, blurred vision, persistent anorexia, and many more issues. In contrast, Radicava’s side effects are mild and include gait disturbance and bruising. Side effects that are more serious include allergic reactions and breathlessness, but such problems are extremely rare.
The director of the Eleanor and Lou Gehrig ALS Center at Columbia University Medical Center, Dr. Neil A. Shneider, said that the drug is very safe. However, he admitted that he would prefer Radicava if it came in pill form, as Riluzole does. Administering the drug is a long and difficult process for the patient. As patients require an intravenous line, the risk of infection is always present. Shneider also said the drug would not necessarily make patients feel stronger or better, nor will they experience an improvement in symptoms.
MT Pharma America is the drug’s manufacturer, and the company is a subsidiary of a Japanese firm called Mitsubishi Tanabe Pharma Corporation. The manufacturer says it will offer co-pay assistance to those with commercial insurance and aid patients who face coverage delays or are currently uninsured. It may even treat certain patients at no cost.
Radicava offers new hope for people with ALS, which is something they have not had in a long time. It enters a tiny market by the standards of the pharmaceutical industry. The ALS drug market was valued at $38 million in 2013. It is set to fall to $16 million in 2020 as Riluzole will lose its patent status and the market will see new generic competition.
The hope is that Radicava offers some form of relief to those with ALS, and perhaps it will pave the way for the launch of new ALS drugs. The efforts of companies to produce effective ALS drugs previously suffered numerous late-stage clinical failures. Maybe Radicava will be a breakthrough.