Les Turner ALS Research and Patient Center at Northwestern Medicine
On May 5, the FDA approved a new drug to treat ALS. Radicava™ (also known as Edaravone) is the first approved ALS-specific drug since Riluzole, which was approved 22 years ago. This is exciting news for the ALS community. While much is still unknown about the drug, its availability and administration, we hope to answer some of your questions here. As we learn more about Radicava, we will keep you informed. And while we hope these answers are informative, we urge you to speak with your clinician about Radicava and whether or not it is appropriate for you.
Who is eligible to be treated with Radicava?
Radicava is an FDA-approved drug for all people with ALS; it is similarly approved in Japan for all people with ALS. It is important to note that the FDA’s approval of Radicava is based on a clinical trial conducted in a small subset of patients with early onset ALS who had well preserved respiratory function.
How effective is Radicava?
According to the FDA, the pivotal Radicava trial showed a 33 percent slowing of loss of function in the participants rated on the ALS Functional Rating Scale (ALSFRS-R). The study was six months in duration and we do not have information on longer term effects. Survival benefit was not measured.
Is there any safety information patients should be aware of?
Before receiving Radicava, please discuss with your healthcare provider all of your medical conditions. The following medical conditions should be explored:
• Allergy history.
• Pregnancy or plan to become pregnant.
• Breastfeeding or plan to breastfeed.
• Medication history: prescription and over-the-counter medicines, vitamins, and herbal supplements.
What are the possible side effects of Radicava?
Radicava was generally well tolerated during clinical trials with the most common side effects including bruising, problems walking (gait disturbance) and headache.
Occasional serious side effects, including hypersensitivity (allergic) reactions and sulfite allergic reactions, have been reported. Radicava contains sodium bisulfite, a sulfite that may cause a type of allergic reaction that can be serious and life-threatening. Sodium bisulfite can also cause less severe asthma episodes in certain people. Patients with a history of asthma appear to be more susceptible to sulfite sensitivity.
Your healthcare provider may decide to give you the first treatment in the hospital to be able to monitor you for signs and symptoms of serious side effects or allergic reactions.
How is the drug treatment administered?
Radicava is an intravenous drug – it is given daily over 60 minutes for a 60 mg dose (two 30 mg IV bags) for 14 consecutive days in month one, then 14 days off. Monthly follow up treatments are given 10 days over a two weeks period; then two weeks off and repeated each month thereafter. Length of treatment should be discussed with your clinician.
Can the drug be taken with Riluzole?
Many of the participants in the clinical trial were taking Riluzole at the same time Radicava was being administered. Please speak with your doctor about taking Radicava and Riluzole at the same time.
When will it be available at the Lois Insolia ALS Clinic?
MT Pharma America anticipates the drug will be available in the United States in August 2017. The Lois Insolia ALS Clinic at the Les Turner ALS Research and Patient Center at Northwestern Medicine is investigating potential delivery options which may include administering the drug through an in-home setting and/or infusion center. We are working hard to ensure the drug can be provided to our patients by prescription as soon as it’s available here in the US.
How does this drug make a difference in the lives of people living with ALS?
While this is a major step forward for the ALS community and we are hopeful, there are many unanswered questions including Radicava’s effects on survival, if any, and whether it will be effective for everyone, regardless of how long someone has had ALS. We anticipate there will be more studies on this drug and related drugs to understand mechanisms and develop more therapies and that Radicava will become an option as part of an overall treatment plan for ALS.
Are there other treatments/cures for ALS on the horizon?
Yes, in addition to Tirasemtiv and Masitinib, which are currently in phase 3, several treatments are in phase 2, including NP001 and NurOwn. It is a very exciting time in ALS drug development with a rich pipeline of new agents targeting multiple mechanisms of action.
Who do I contact with additional questions about Radicava?
Patients should discuss questions about Radicava with their clinician. For specific questions about Radicava™, please feel free to contact the following services set up by MT Pharma:
• MT Pharma’s Medical Affairs 1-888-292-0058
• Searchlight Support™1-844-SRCHLGT (1-844-772-4548) (available after the drug can be prescribed, most likely in July)
• You can also sign up to receive updates about Radicava™, including when it will be available for your healthcare provider to prescribe at: https://www.radicava.com/patient/.
For more information about the Les Turner ALS Research and Patient Center visit www.feinberg.northwestern.edu/sites/alsresearch/
For more information about the Les Turner ALS Foundation visit www.lesturnerals.org