PM&R News   •   June 9, 2017

By now you likely have heard that there is a new FDA approved ALS drug named Edaravone (Radicava) on the market. If you haven’t seen the headlines, let’s catch you up on the latest buzz.

In 2014, a Japanese double blind placebo controlled study evaluated the efficacy and safety of Radicava. One hundred and four patients received the placebo treatment and 102 received Radicava. After 24 weeks of treatment patients’ ALS functional rating scale score decreased by 6.35 points in the placebo but only 5.70 points in the treatment group. Sounds like a small change, but take a look at the scale again.  A one point decrease could mean:

• tube feeding vs. dietary consistency changes;
• non-ambulatory vs. walking with assistance;
• or loss of useful speech vs. speech combined with non-vocal communication.

Further, adverse events between Radicava and placebo were similar.

While significant improvements were noted in the ALS functional rating scale, a post-hoc analysis supported better efficacy if given early after diagnosis.

Radicava is administered intravenously in 28-day cycles. The treatment starts with a patient receiving the therapy for 14 consecutive days, followed by a two-week drug-free period. Patients then receive an infusion during 10 of the next 14 days, followed by a two-week drug-free period.

Patients are prescribed 60 mg per treatment, and each treatment takes 60 minutes to administer.

Riluzole

On a different note, anyone remember Riluzole? Riluzole was shown to have a 9% gain in probability of surviving one year and an increased median survival from 11.8 to 14.8 months (a 3-month increased survival – hint potential Board question).  Again, a small change looking at the numbers, but could have a big impact on one’s life.

Now let’s think next steps…  What would survival, ALS-functional rating scale score, or efficacy look like if the two were combined? Everyone is excited about the new drug, but what doors have Radicava opened? Physiatry is a team speciality, maybe we are coming on the horizon of an amazing future drug combination that may act in a synergistic fashion to prolong the lives and improve function in ALS patients. 

Radicava should be available August 2017 (Mitsubishi Tanabe Pharma Corporation).