On 9/29/2022, the US Food and Drug Administration’s (FDA) announced that it approved RELYVRIO (AMX0035) as a new treatment for ALS.
As we celebrate this ground-breaking development in the fight against ALS, we want to thank everyone who worked tirelessly to make this monumental moment happen! While we still have a lot of work to find cures for ALS, this new treatment is a significant step toward our ultimate goal.
We know that there are a lot of questions that people have, in light of this exciting news. Here are answers to some of the frequently asked questions that we are fielding from our ALS community.
What can you tell us about this new treatment for ALS?
A study published in the New England Journal of Medicine in 2020 showed that AMX0035 is safe and effective at treating ALS. Earlier this year, AMX0035 trial investigators reported long-term results showing that it can slow disease progression, extend life by 10 months, and reduce the risk of hospitalization.
As reported in the New York Times, the Phase 2 trial involved patients considered to have fast-progressing disease. Two-thirds of participants received AMX0035. Over 24 weeks, they experienced a 25 percent slower decline than participants receiving placebo — declining 2.32 points less on a 48-point ALS scale that rates 12 physical abilities, including walking, speaking, swallowing, dressing, handwriting and breathing.
The open-label extension study involved 90 of those patients, including 34 from the placebo group, who began taking the medication about seven months after those who received it from the beginning. Patients who received the treatment the longest had a median of about 6.5 months more time before being hospitalized, being put on a ventilator or dying, Amylyx reported. Researchers later published another analysis that suggested additional benefit.
It was already officially approved for use in Canada in June of 2022 for people living with ALS. On 9/29/2022, the FDA announced that it approved AMX0035, now called RELYVRIO, as a new treatment for ALS.
How does RELYVRIO work?
RELYVRIO is a combination of two existing drugs, sodium phenylbutyrate and taurursodiol, which acts to prevent nerve cell death by blocking stress signals in cells. Unlike other treatments in development, RELYVRIO does not target the root cause of ALS. Instead, it aims to preserve the motor neurons that are progressively lost in ALS patients, slowing clinical decline.
How is RELYVRIO administered?
RELYVRIO is an oral therapy (a suspension in water, taken by swallowing or via PEG tube). Your doctor will tell you how much RELYVRIO to take and when to take it. The recommended dosing is to take 1 packet once a day for the first 3 weeks. After the first 3 weeks, take 2 packets per day, 1 packet in the morning and 1 packet at night.
How should I decide whether RELYVRIO is right for me?
As with any decision regarding your treatment and/or disease management plan, talk to your doctor about whether or not RELYVRIO is right for you.
How can adults with ALS access RELYVRIO?
Amylyx has launched two websites to provide information to the ALS community.
First, you and your health care provider can visit www.relyvrio.com for specific details about this medication. In addition, you can utilize the Amylyx Care Team (ACT) Support Program to help people living with ALS access the therapy and answer many questions about insurance reimbursement and other programs. To find out more, please visit: www.amylyxcareteam.com or call 1-866-318-2989.
If you and your doctor determine that RELYVRIO is right for you, your doctor will complete the RELYVRIO Enrollment Form with you, which enrolls you in the ACT Support Program and also serves as your prescription.
Will RELYVRIO be covered by my insurance?
Amylyx is working with all insurers, including Medicare, Veterans Affairs (VA), and commercial health insurance companies, to educate them on RELYVRIO and asking them to cover it. Drug coverage decisions will be made by Medicare, VA, and commercial private insurers. Securing coverage could take up to 45 days or longer, especially within the first 6 months of product approval. Call ACT to see if there are any programs that may be available for commercially insured eligible adults living with ALS while your insurance is being secured.
What if I can’t afford RELYVRIO?
ACT can help you understand your out-of-pocket insurance cost. If you are unable to afford RELYVRIO, ACT can help you learn about programs that you may be eligible for. To learn more about possible financial assistance options, visit AmylyxCareTeam.com or call 1-866-318-2989 Monday-Friday, 8:00 am-8:00 pm ET.
I have a feeding tube. Can I use RELYVRIO?
Yes. RELYVRIO can be administered through a feeding tube, as directed by your doctor.
How do I transition from an Amylyx clinical trial, Early Access Program, or Compassionate Use Program to the commercial product (RELYVRIO)?
Amylyx is committed to ensuring adults living with ALS have equal access to receiving RELYVRIO. If you are currently enrolled in a clinical trial, the Early Access Program, or the Compassionate Use Program, please contact your Site Coordinator or doctor to transition to the commercially available product, if you and your doctor decide to continue taking RELYVRIO.
How did the ALS community and the Golden West Chapter contribute to this victory?
The Golden West Chapter worked closely with Amylyx and many of our families to provide direct feedback and personal stories that ultimately helped contribute to this outcome. Thousands of people living with ALS, their loved ones, and advocates sent emails to the FDA urging the agency approve the treatment. The Chapter mobilized ALS advocates who participated in several meetings with FDA officials.
The Chapter recognized Amylyx with our 2021 Commitment to Cures Award for their efforts to champion drug discovery and development in the relentless pursuit of treatments and cures for people with ALS and related diseases.
The Golden West Chapter shares a close affiliation with the Certified Treatment Centers of Excellence responsible for the AMX0035 clinical trials based in California. In addition to supporting and staffing those clinics, the Chapter voluntarily conducted outreach to the ALS community to help populate the trials effectively and efficiently.
How did the ALS Ice Bucket Challenge in 2014 contribute to this drug’s development?
If you took the Ice Bucket Challenge, you helped deliver RELYVRIO. This is the first approved ALS treatment funded by Ice Bucket Challenge donations. In 2016, The ALS Association invested $2.2 million to help develop and test RELYVRIO.
Amylyx is working on making RELYVRIO available as soon as possible. We encourage you to talk with your doctor, and learn more on their website or contact the Amylyx Care Team (ACT) at 1-866-318-2989. The Golden West Chapter will continue to provide the very latest information as it becomes available.