FDA Approves New Drug to Treat ALS

FDA Approves First ALS Drug in 22 Years

Anyone who was on social media in the summer of 2014 knows what the Ice Bucket Challenge was.  Videos of people completing the challenge littered most people’s social media feeds, all in hopes of promoting awareness and raising money for amyotrophic lateral sclerosis (ALS), which is “a rare disease that attacks and kills the nerve cells that control voluntary muscles.”

FDA Approves 1st ALS Drug in 22 Years

On Friday, May 5th, the FDA approved Radicava (edaravone), a drug indicated for the treatment of patients with ALS, more commonly known as Lou Gehrig’s disease.  Radicava is manufactured by the Japanese company Mitsubishi Tanabe Pharma, but US promotion of the product will be handled by MT Pharma America, a sales subsidiary of Mitsubishi Tanabe Pharma.

Radicava was granted orphan drug designation by the FDA; and, according to a recent article from Fox 5 DC, the product was not expected to be available for another two to three years, but the FDA approved the product’s application less than a year after it was submitted.  Fox’s article states that the product’s speedy approval is partly due to the funds raised by the Ice Bucket Challenge.  This goes to show that, although many people may have doubted about how this seemingly “silly” challenge would help those suffering from ALS, we can now see how the small efforts made by so many people nearly three years ago will positively impact the lives of those suffering from this crippling disease.

About Radicava

“Radicava is an intravenous infusion given by a health care professional. It is administered with an initial treatment cycle of daily dosing for 14 days, followed by a 14-day drug-free period. Subsequent treatment cycles consist of dosing on 10 of 14 days, followed by 14 days drug-free.”

Efficacy of Radicava

Mitsubishi Tanabe Pharma demonstrated efficacy of Radicava for the treatment of ALS in a six-month clinical trial conducted with 137 participants in Japan.  During the study, the participants were randomized to receive either the drug or a placebo.  According to FDA’s recent press release, “at week 24, individuals receiving edaravone declined less on a clinical assessment of daily functioning compared to those receiving a placebo.”

Side Effects of Radicava

The most common side effects reports by clinical trial participants include bruising (contusion) and gait disturbance.  FDA also notes that there are a number of more serious side risks associated with the use of Radcava; these risks require immediate medical care, and include:

  • Hives
  • Swelling
  • Shortness of breath
  • Allergic reactions to sodium bisulfite (an ingredient in the drug).

As this is the first drug approved by the FDA for the treatment of patients with ALS in 22 years, Radicava’s approval is a monumental step for both these patients and their families.  Deputy director of CDER’s Division of Neurology Products, Eric Bastings, MD, states that “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 is committed to providing patients with new and innovative therapies, and Radicava’s approval is just another way the Agency confirmed that commitment.

For additional information, view FDA’s announcement and Mitsubishi Tanabe Parma’s press release.

Are you in the process of developing a product for a rare disease?  We can help you get your product approved by the FDA in the least amount of time possible.  To learn more about our services and how we can help you, contact us today.