On May 5, 2017, the FDA approved a new drug — Edaravone — to treat ALS.  This is the first approval of an ALS treatment that slows progression since Riluzole was approved 22 years before, and incredibly good news for people living with ALS and the ALS field in general. 

Patients naturally have many questions about the treatment and how it works. We will try to answer them here.

Are all people with ALS eligible to be treated with Edaravone?
Yes. Edaravone is an FDA-approved drug for all people with ALS; it is similarly approved in Japan for all people with ALS.
What benefits can patients expect from the treatment?
The pivotal trial reported a 33 percent slowing of loss of function in the participants in the trial. The study was six months in duration, so unfortunately we do not have data /information on longer term effects.
Are there any known side effects?
Overall Edaravone has proven to be very safe in the six month trial.  There is some increased risk of contusion and gait imbalance, and a risk of allergic reaction in people with sulfite allergies.
How is the treatment administered?
Edaravone is an intravenous drug - it is given daily over 60 minutes for a 60 mg dose (two 30 mg IV bags) for 14 days in month one, then 14 days off, then 10 days in the next month over two weeks; then two weeks off; and then repeated each month. It can be administered in a home setting. We will likely administer the first treatments in our infusion center.
Is it available at Mass General?
How hopeful are you that it will make a difference in the lives of people living with ALS?
We are very hopeful. This is really good news for our patients and for the ALS field. There are some unanswered questions still — its effects on survival and whether it will be effective for everyone, regardless of how long someone has had illness. Still, this is a major step forward. Hopefully there will be more studies on this drug and related drugs to understand mechanisms and develop more therapies. There are also a lot of other therapies in trials or in preclinical testing that are very hopeful.
Are there other treatments/cures for ALS on the horizon?
Yes, including several in testing at the Healey Center for ALS. Learn more about participating in ALS clinical trials and studies.
What does the FDA approval of Edaravone signify about future ALS treatments?
This is, if not the first, one of the first therapies to be approved in US that did not have a clinical trial here in the US. It is also the first drug approved where trials were six months in duration. This is a paradigm shift in ALS and sets perhaps a new regulatory pathway that is more streamlined. We are very happy for our patients, for the field and look forward to many more options for our patients! And we will keep working hard to find them — there is still much to do.
For people with additional questions about Edaravone, who should they contact?

For our patients, they should discuss Edarvone with their clinician.  For specific questions about Radicava™, please feel free to contact the following services set up by the company:

• MT Pharma's Medical Affairs: 888-292-0058
• Searchlight Support™: 844-772-4548
• You can also find more information about Radicava™ at radicava.com.