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New Brunswick is reimbursing, through its public health insurance program, the cost of <a href="https://www.amylyx.ca/document/albrioza-product-monograph.pdf" rel="nofollow">Albrioza</a> (sodium phenylbutyrate and ursodoxicoltaurine) for eligible residents living with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
The Canadian province is the country&#8217;s fourth to provide public coverage for the oral therapy — marketed in the U.S. as <a href="https://alsnewstoday.com/amx0035/">Relyvrio</a>. Similar reimbursement decisions recently were announced in <a href="https://alsnewstoday.com/news/treat-als-ontario-quebec-add-albrioza-public-reimbursement-list/">Ontario and Quebec</a>, followed by British Columbia.
“We sincerely appreciate the New Brunswick government&#8217;s collaboration and support in providing public coverage for Albrioza, to make timely treatment possible for individuals living with ALS,” Chris Aiello, general manager and head of Canada at <a href="https://www.amylyx.com/" rel="nofollow">Amylyx Pharmaceuticals</a>, the therapy’s developer, said in a <a href="https://www.newswire.ca/news-releases/new-brunswick-to-publicly-reimburse-albrioza-tm-for-the-treatment-of-als-879073652.html">company press release</a>.
“Recognizing the speed at which this devastating disease progresses, our aim is to ensure equitable access to this treatment for all eligible Canadians, regardless of their location,” Aiello said. “We are actively working with other provincial, territorial, and federal drug plans to expedite the process and make it a reality as quickly as possible.”
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<h3>ALS oral treatment given conditional approval in Canada in 2022</h3>
Previously called AMX0035, Albrioza is an oral therapy containing a combination of two molecules that are thought to help reduce damage to nerve cells. It was granted <a href="https://alsnewstoday.com/news/amx0035-oral-als-therapy-called-albrioza-approved-canada/">conditional approval in Canada</a> for treating people with ALS in June 2022, based on clinical trial data suggesting it can <a href="https://alsnewstoday.com/news/amx0035-safe-effective-in-slowing-als-functional-decline-trial-data-shows/">slow ALS progression</a> and <a href="https://alsnewstoday.com/news/amx0035-survival-als-patients-centaur-clinical-trial-analysis-amylyx/">prolong survival</a>.
Full study results were published in <a href="https://www.nejm.org/doi/full/10.1056/NEJMoa1916945">The New England Journal of Medicine</a> and the journal <a href="https://onlinelibrary.wiley.com/doi/10.1002/mus.27569">Muscle &amp; Nerve</a>.
The medication also is <a href="https://alsnewstoday.com/news/relyvrio-amx0035-approved-us-als-treatment/">approved for ALS patients in the U.S.</a>, while a European Union committee <a href="https://alsnewstoday.com/news/chmp-opposes-approval-amx0035s-als-treatment-europe/">recommended against conditional approval</a> last month. Amylix plans to request a formal reconsideration of its application before the European Commission makes a final decision.
Albrioza has been available in Canada commercially for nearly a year, but only recently have patients begun to gain access to reimbursement formulas through public health insurance programs, run by each province and territory individually.
Announcements of reimbursement deals, in New Brunswick and elsewhere, come on the heels of Amylyx <a href="https://alsnewstoday.com/news/amylyx-agrees-deal-albrioza-als-treatment-reimbursement-canada/">finalizing an</a><a href="https://alsnewstoday.com/news/amylyx-agrees-deal-albrioza-als-treatment-reimbursement-canada/"> agreement</a> with the pan-Canadian Pharmaceutical Alliance, a government agency that collaborates with the country&#8217;s provinces and territories in establishing public drug plan funding of approved medications.
<h3>Advocates welcome news, but argue for therapy&#8217;s inclusion in plans nationwide</h3>
Advocates welcomed the decision, though they emphasized the importance of making the therapy available at lower cost to ALS patients throughout Canada.
“We applaud the government of New Brunswick for its decision to offer public coverage” for Albrioza, as “it is of utmost importance that eligible individuals can promptly access approved treatments,” said Kimberly Carter, president and CEO of the <a href="https://www.facebook.com/ALSNBNS/">ALS Society of New Brunswick and Nova Scotia</a>.
“We express our hope that other public drug plans, especially those in Atlantic Canada, will follow suit in making this treatment accessible,” Carter added. “This will ensure that Canadians dependent on public coverage will have the opportunity to benefit from this innovative treatment.”
ALS patients “critically need timely access to treatment,” said Colleen O&#8217;Connell, MD, medical director and research chief at Horizon&#8217;s Stan Cassidy Centre for Rehabilitation in New Brunswick.
“With few existing treatment options for ALS, I and others in the community are eager to hear news of Albrioza&#8217;s listing on other public drug plans to enable wider access to this treatment and greater hope for people living with this disease,” O&#8217;Connell added.
In Canada, prescription drugs are provided at no charge to all hospitalized patients. For others, each province and territory&#8217;s <a href="https://www.canada.ca/en/health-canada/services/health-care-system/pharmaceuticals/access-insurance-coverage-prescription-medicines.html">public health insurance plan</a> can cover at least part of a prescription drug that&#8217;s included in its plan.

New Brunswick adds Albrioza for ALS to its public health plan

<a href="https://www.amylyx.ca/">Amylyx Pharmaceuticals Canada</a> has entered into product listing agreements with Quebec and Ontario to publicly reimburse <a href="https://www.amylyx.ca/document/albrioza-product-monograph.pdf">Albrioza</a> (sodium phenylbutyrate and ursodoxicoltaurine), which was recently approved for <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
The oral therapy, which is marketed in the U.S. as <a href="https://alsnewstoday.com/amx0035/">Relyvrio</a>, has been added to the list of medications reimbursed under the Regie de l&#8217;assurance maladie du Quebec and Ontario&#8217;s Exceptional Access Program.
Previously called AMX0035, the therapy has been shown to help slow disease progression and extend survival in people with ALS, a disorder that affects some 3,000 residents of Canada.
The drug became available through Ontario&#8217;s Drug Benefit Program June 22, shortly after Amylyx completed negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA), which negotiates prices with manufacturers on behalf of public payers.
Negotiations began after the treatment was <a href="https://alsnewstoday.com/news/amx0035-oral-als-therapy-called-albrioza-approved-canada/">conditionally approved</a> by Canadian authorities last year. Albrioza was made <a href="https://alsnewstoday.com/news/amx0035-albrioza-oral-als-therapy-available-canada/">commercially available</a> in Canada in August 2022, but patients couldn&#8217;t be reimbursed because it wasn&#8217;t yet listed on most public health plans.
&#8220;We are thrilled Ontarians with public insurance who are living with ALS will now have access to Albrioza, and we are grateful for the collaboration of the Ontario government to make timely access possible,&#8221; Chris Aiello, general manager and head of Canada at Amylyx, said in a <a href="https://finance.yahoo.com/news/ontario-publicly-reimburse-albrioza-treatment-173000313.html">company press release</a>.
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<h3>Efforts to extend access to Albrioza underway</h3>
Tammy Moore, CEO of the <a href="https://als.ca/?gclid=CjwKCAjwwb6lBhBJEiwAbuVUSlSGBZ85o5pxjkqSwQ9xOLMulkJnNlsXFzy_CM4RpxQnbNNBz8ozahoC9LcQAvD_BwE">ALS Society of Canada</a>, said: &#8220;We congratulate the Ontario government on its decision to reimburse this treatment in such a timely manner following pCPA negotiations.&#8221;
Quebec became the second province to provide public reimbursement of Albrioza under an agreement that became effective July 6. Amylyx is working with other provinces and territories, as well as federal drug plans, to make the therapy available to more people in Canada.
&#8220;We are grateful for the Quebec government&#8217;s collaboration and its support of people living with ALS by providing public coverage for Albrioza,&#8221; Aiello said in another <a href="https://www.newswire.ca/news-releases/government-of-quebec-to-cover-albrioza-tm-for-the-treatment-of-als-820413417.html">press release</a>. &#8220;Understanding the speed at which this disease progresses, our goal is to ensure that any eligible Canadian, no matter where they live, will have equitable access to this treatment. We are working closely with all other public drug plans to make this happen as soon as possible.&#8221;
Albrioza is a fixed-dose combination sodium phenylbutyrate and tauroursodeoxycholic acid that&#8217;s thought to protect nerve cells from the cellular stress-induced damage implicated in ALS. It&#8217;s taken twice daily by mouth or via a feeding tube.
&#8220;The decision to include Albrioza on Quebec&#8217;s list of publicly covered medications is welcome news,&#8221; said Genevieve Matte, assistant clinical professor, department of neurosciences a the University of Montreal. &#8220;There have been so few treatment options for ALS and so it&#8217;s vital that people living with ALS have timely access to new treatment that can slow disease progression and loss of functional decline. My hope is that access will be extended across Canada to all those who may benefit from this treatment.&#8221;
The decision to approve Albrioza in Canada was based on data from the <a href="https://classic.clinicaltrials.gov/ct2/show/NCT03127514">CENTAUR Phase 2 clinical trial (NCT03127514)</a> and its <a href="https://classic.clinicaltrials.gov/ct2/show/NCT03488524">open-label extension study (NCT03488524)</a>, which indicated it significantly slowed functional decline in patients with rapidly progressing disease. It also extended survival by 6.9 months over a placebo, indicating a 43% lower risk of death.
&#8220;Our work is by no means done,&#8221; Aiello said. &#8220;We are dedicated to ensuring that all who rely on public coverage in Canada can also access the treatment as soon as possible. People living with ALS are on a different clock, and thus we are moving urgently to obtain timely, equitable access for all eligible Canadians.&#8221;
&#8220;I look forward to news of Albrioza&#8217;s listing on other public drug plan formularies for eligible Canadians coast to coast,&#8221; said Christen Shoesmith, MD, medical director of the ALS clinic at the London Health Sciences Centre and current Canadian ALS Research Network (CALS) chair.

Quebec, Ontario add Albrioza to public reimbursement list

The Committee for Medicinal Products for Human Use (CHMP) has issued an opinion opposing <a href="https://www.amylyx.com/" rel="nofollow">Amylyx Pharmaceuticals</a>’ bid for conditional European Union approval of AMX0035, its oral treatment to slow the progression of <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
The negative recommendation from the advisory committee, an arm of the European Medicines Agency (EMA), is not binding. But the EMA will consider the opinion when making its final approval decision, while Amylyx intends to request that CHMP reconsider its findings.
According to the EMA, <a href="https://www.ema.europa.eu/en/medicines/human/summaries-opinion/albrioza">the committee had concerns</a> that data from the <a href="https://clinicaltrials.gov/ct2/show/NCT03127514">CENTAUR trial (NCT03127514)</a>, on which the application was based, did not &#8220;convincingly show&#8221; that AMX0035 was effective at <a href="https://alsnewstoday.com/living-with-als-2/">slowing ALS progression</a>.
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<h3>Oral ALS treatment has conditional approval in US and Canada, not Europe</h3>
Survival findings also were &#8220;unreliable,&#8221; the opinion stated, given how these data were collected and analyzed. In support of its decision, <a href="https://alsnewstoday.com/news/european-approval-amx0035-als-faces-new-hurdle-review/">advice from expert groups</a>, including neurology specialists and patient representatives, was considered.
The therapy is approved in the U.S. under the name <a href="https://alsnewstoday.com/amx0035/">Relyvrio</a> (sodium phenylbutyrate and taurursodiol) and in Canada as Albrioza — the brand name that will be used in Europe should it be approved.
“We are confident in the strength of our CENTAUR trial data, which we believe meets the criteria for conditional approval. These data were also the basis of the full approval received from the U.S. Food and Drug Administration and the approval with conditions from Health Canada,” Tammy Sarnelli, global head of regulatory affairs and clinical compliance at Amylyx, said in a <a href="https://www.businesswire.com/news/home/20230622200968/en/Amylyx-Pharmaceuticals-Receives-CHMP-Negative-Opinion-on-its-Conditional-Marketing-Authorisation-Application-for-AMX0035-for-the-Treatment-of-ALS-in-the-European-Union">company press release</a>. “We disagree with the CHMP’s opinion and will request a formal re-examination procedure of the current Marketing Authorisation Application (MAA).”
Filing for a formal reconsideration of the application starts a process that takes about four months. Based on an evaluation by a different set of examiners than those of the initial assessment, the process will close with CHMP adopting a final opinion.
“We will continue to engage with CHMP and EMA through the re-examination process with the goal of making Albrioza available in Europe as it is in the United States and Canada,” said Joshua Cohen and Justin Klee, Amylyx’s co-CEOs. “We know how precious time is for people with ALS and their families.”
Although it is not clear how AMX0035 works, its two active ingredients, sodium phenylbutyrate and ursodoxicoltaurine (taurursodiol in the U.S), are thought to reduce the nerve cell damage that <a href="https://alsnewstoday.com/causes-of-als/">causes ALS</a> and slow the progression of <a href="https://alsnewstoday.com/symptoms-of-als/">disease symptoms</a>.
<h3>Findings of Phase 3 trial of AMX0035 in ALS patients likely in mid-2024</h3>
Data from CENTAUR, a Phase 2 trial that enrolled 137 adults with rapidly progressing ALS, supported its <a href="https://alsnewstoday.com/news/amx0035-albrioza-oral-als-therapy-available-canada/">conditional approval in Canada</a> and <a href="https://alsnewstoday.com/news/relyvrio-amx0035-approved-us-als-treatment/">its U.S approval.</a>
Meeting its primary goal, the trial showed that AMX0035 significantly <a href="https://alsnewstoday.com/news-posts/2020/09/03/amx0035-safe-effective-in-slowing-als-functional-decline-trial-data-shows/">slowed disability progression</a> compared with a placebo. Data spanning CENTAUR and its open-label extension study also supported a <a href="https://alsnewstoday.com/news/amx0035-survival-als-patients-centaur-clinical-trial-analysis-amylyx/">10-month survival benefit</a> in people assigned to AMX0035 from the start.
A Phase 3 trial called <a href="https://clinicaltrials.gov/ct2/show/NCT05021536">PHOENIX (NCT05021536)</a> is underway to confirm AMX0035’s safety and efficacy in 664 ALS patients at sites across the U.S. and Europe. The new <a href="https://alsnewstoday.com/news/phoenix-trial-approved-als-therapy-relyvrio-amx0035-enrolled/">trial is fully enrolled</a>, with top-line data expected by June 2o24.
“While our MAA continues to be under review, we will also work towards completing our global Phase 3 PHOENIX study, with topline results anticipated in mid-2024, which will provide important additional data on the efficacy and safety profile of Albrioza,” said Stéphanie Hoffmann-Gendebien, general manager at Amylyx. “There have been no new innovations approved in Europe for this devastating disease in over 25 years, and we recognize the urgent need of the ALS community in Europe to access new treatment options.”
CHMP’s negative opinion will not affect patients currently enrolled in clinical trials of AMX0035, Amylyx stated. It encourages study participants needing further information to speak with their clinical trial physician.

EMA branch opposes conditional approval of AMX0035 in Europe

Albrioza, the approved treatment for <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) formerly known as AMX0035, is one giant step closer to being eligible for reimbursement under public drug plans in Canada, according to its developer, Amylyx Pharmaceuticals.
<a href="https://www.amylyx.com/">Amylyx</a> has reached an agreement with the pan-Canadian Pharmaceutical Alliance, or pcPA — a government partnership in Canada that collaborates on various public drug plan initiatives — for terms under which ALS patients in the country can be reimbursed for Albrioza.
The negotiations began after the therapy, marketed as <a href="https://alsnewstoday.com/amx0035/">Relyvrio</a> in the U.S., was <a href="https://alsnewstoday.com/news/amx0035-oral-als-therapy-called-albrioza-approved-canada/">conditionally approved</a> by Canadian authorities a year ago. While Albrioza became <a href="https://alsnewstoday.com/news/amx0035-albrioza-oral-als-therapy-available-canada/">commercially available</a> in the country in August 2022, it hasn&#8217;t yet been listed on most public health plans — meaning patients on those plans couldn&#8217;t be reimbursed for its costs.
Amylyx has now signed off on its intentions to honor the new agreement in a letter of intent (LOI), which, if finalized, will enable eligible patients to avoid high out-of-pocket costs for the medication.
“Today marks a major milestone for people living with ALS and their caregivers in Canada. With this signed LOI, eligible Canadians living with this rapidly progressive disease who rely on public coverage are one step closer to accessing Albrioza,” Chris Aiello, general manager and head of Canada at Amylyx, said in a <a href="https://www.businesswire.com/news/home/20230615926630/en/Amylyx-Pharmaceuticals-Completes-Negotiation-Process-and-Signs-Letter-of-Intent-with-the-pCPA-for-ALBRIOZA%E2%84%A2-for-the-Treatment-of-ALS">company press release</a>, adding, however, “Our work is by no means done.”
[recommended-reading id=78571]
<h3>Advocates cite lengthy delay in getting treatment for ALS covered</h3>
With the LOI now in place, the company will move forward to work individually with federal, provincial, and territorial drug plans to finalize the agreements, which will enable Albrioza to be listed for reimbursement on those plans.
Although the news is welcome, some believe it did not come fast enough for patients, many of whom see rapid declines in the first few years after disease onset.
“While it is good news that Canadians living with ALS are closer to accessing this innovative treatment, some people living with ALS have been waiting a year to access this therapy, and they will continue to wait,” said Tammy Moore, CEO of the <a href="https://als.ca/">ALS Society of Canada</a>.
“People with ALS do not have the luxury of time,” Moore said.
[quote align=&#8221;center&#8221;]We believe there are opportunities to streamline the multiple steps and lengthy processes involved in achieving public reimbursement for treatments like Albrioza in Canada. The time is now for equitable and expedited access to approved treatments for ALS, and we will continue advocating on behalf of the ALS community to improve their access to care.[/quote]
The process of a therapy being listed on public health plans involves sometimes lengthy discussions between pcPA and the company that markets the therapy. The pcPA has to negotiate the price of the treatment on behalf of public drug payers, including both federal and local governments, along with determining which patients will be eligible for reimbursement.
Additional discussions then ensue with each individual public drug payer to work out the details.
“We believe there are opportunities to streamline the multiple steps and lengthy processes involved in achieving public reimbursement for treatments like Albrioza in Canada,” Moore said.
“The time is now for equitable and expedited access to approved treatments for ALS, and we will continue advocating on behalf of the ALS community to improve their access to care,” Moore added.
[recommended-reading id=82805]
<h3>Treatment for ALS facing regulatory hurdles in EU</h3>
Albrioza is a fixed-dose oral combination of two small molecules — tauroursodeoxycholic acid and sodium phenylbutyrate — that together are thought to protect nerve cells against forms of cellular stress implicated in ALS. As such, the therapy is expected to slow ALS progression.
Canadian authorities conditionally approved the treatment based on findings from the <a href="https://clinicaltrials.gov/ct2/show/NCT03127514">Phase 2 CENTAUR trial (NCT03127514)</a> and its <a href="https://clinicaltrials.gov/ct2/show/NCT03488524">extension study (NCT03488524)</a>. Their findings indicated that Albrioza significantly <a href="https://alsnewstoday.com/news-posts/2020/09/03/amx0035-safe-effective-in-slowing-als-functional-decline-trial-data-shows/">slowed functional declines</a> and <a href="https://alsnewstoday.com/news-posts/2022/05/06/amx0035-survival-als-patients-centaur-clinical-trial-analysis-amylyx/">extended survival time</a> for ALS patients.
A full, final approval in the country is contingent upon results from the global <a href="https://clinicaltrials.gov/ct2/show/NCT05021536">Phase 3 PHOENIX trial (NCT05021536)</a>, which is now seeking to confirm the benefits of Albrioza against a placebo in a larger group of patients. That trial, which <a href="https://alsnewstoday.com/news/phoenix-trial-approved-als-therapy-relyvrio-amx0035-enrolled/">completed enrollment</a> earlier this year, is expected to finish in 2024.
The therapy’s path to approval has not been as straightforward elsewhere.
In the U.S., it came under scrutiny by a U.S. Food and Drug Administration (FDA) advisory committee that initially voted that existing data <a href="https://alsnewstoday.com/news-posts/2022/04/01/fda-committee-votes-against-recommending-als-therapy-amx0035/">were not sufficient</a> to support the treatment’s clinical efficacy.
While that opinion is not binding, the FDA takes it into account when deciding about whether to approve a therapy.
Following a rare second review after Amylyx submitted additional trial data, the committee <a href="https://alsnewstoday.com/news/fda-committee-reversal-favors-amx0035-approval-als/">later reversed course</a>. The FDA ultimately <a href="https://alsnewstoday.com/news/relyvrio-amx0035-approved-us-als-treatment/">approved</a> the therapy, as Relyvrio, in September.
Now, the treatment faces similar hurdles in the European Union, where it&#8217;s under review. The European Medicines Agency (EMA)&#8217;s Committee for Medicinal Products for Human Use (CHMP) noted earlier this year that it was leaning toward <a href="https://alsnewstoday.com/news/amylyxs-als-therapy-amx0035s-facing-possible-no-from-chmp/">not recommending</a> the therapy for approval. In March, CHMP sought <a href="https://alsnewstoday.com/news/european-approval-amx0035-als-faces-new-hurdle-review/">more information</a> from Amylyx, noting there were still objections to the therapy that needed to be addressed.
As with an FDA advisory committee, the CHMP&#8217;s opinion is not binding, but will inform the final decision made by the EMA. The CHMP is expected to give its final opinion in coming days.

Amylyx agrees to deal for Albrioza reimbursement in Canada for ALS

<a href="https://www.neopharmisrael.co.il/" rel="nofollow">Neopharm</a> has acquired exclusive rights to commercialize AMX0035 (sodium phenylbutyrate and taurursodiol) in Israel, Gaza, the West Bank, and to the Palestinian Authority as a treatment of <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
The company&#8217;s agreement with <a href="https://www.amylyx.com/" rel="nofollow">Amylyx Pharmaceuticals</a> makes it responsible for the distribution and marketing of AMX0035 in these Middle East regions, pending its approval by regulatory authorities. Neopharm also will be in charge of the filings and duties required for regulatory review and therapy reimbursement.
“This agreement with Neopharm is an important step towards expanding AMX0035’s availability in the EMEA (Europe, Middle East, and Africa) region, if approved,” Stéphanie Hoffmann-Gendebien, general manager and head of EMEA at Amylyx, said in a <a href="https://www.businesswire.com/news/home/20230116005040/en/Amylyx-Pharmaceuticals-Announces-Exclusive-AMX0035-Distribution-Agreement-with-Israel-Based-Neopharm">company press release</a>.
[recommended-reading id=81647]
<h3>AMX0035 available in U.S. as Relyvrio and in Canada as Albrioza</h3>
AMX0035 is <a href="https://alsnewstoday.com/news/relyvrio-amx0035-approved-us-als-treatment/">approved in the U.S.</a> under the brand name <a href="https://alsnewstoday.com/amx0035/">Relyvrio</a> and in Canada as Albrioza. This is Amylyx’s first distribution agreement since Relyvrio was approved.
“Our team remains steadfast in our mission to help every eligible person living with ALS gain access to AMX0035, and working with the right partners, such as Neopharm, will help us to reach that goal as quickly and efficiently as possible,&#8221; said Hoffmann-Gendebien.
&#8220;This decision was based on a number of factors, including the ability to leverage potential approvals in other regions in the Israel Health Ministry’s review process to provide access for eligible people with ALS,” she added.
Relyvrio is an oral therapy designed to help slow disease progression in people with ALS. Taken twice daily by mouth or via a feeding tube, the treatment can be administered alone or in combination with other ALS medications.
The therapy is a fixed-dose combination of two compounds — tauroursodeoxycholic acid and sodium phenylbutyrate — that work together to inhibit stress signals involved in nerve cell damage and ALS progression.
“The addition of AMX0035 to our portfolio and the opportunity to serve the ALS community in Israel and beyond is an honor,” said Efi Shnaidman, general manager of Neopharm Israel, where the company is based. “We look forward to working with Amylyx to serve people living with ALS who may benefit from this important new treatment option.”
Relyvrio&#8217;s approvals were supported by data from the <a href="https://clinicaltrials.gov/ct2/show/NCT03127514">CENTAUR Phase 2 clinical trial called (NCT03127514)</a>, which assessed the medication in 137 adults recently diagnosed with rapidly progressing ALS.
Participants were randomly assigned to either Relyvrio or a placebo, taken twice daily, for 24 weeks (about six months). Most patients then enrolled in an <a href="https://clinicaltrials.gov/ct2/show/NCT03488524">open-label extension study (NCT03488524)</a>, in which all were given Relyvrio for up to 30 months (2.5 years).
<a href="https://alsnewstoday.com/news-posts/2020/09/03/amx0035-safe-effective-in-slowing-als-functional-decline-trial-data-shows/">Top-line results</a> showed the Phase 2 trial met its main goal, with Relyvrio being significantly more effective than a placebo at slowing functional decline on the ALS Functional Rating Scale-Revised (ALSFRS-R) over the six months.
<a href="https://investors.amylyx.com/node/7001/pdf" rel="nofollow">Findings from CENTAUR and its extension</a> also demonstrated that patients randomized to Relyvrio in the main trial survived a mean of 6.9 months longer than those initially on placebo, who began treatment in the long-term extension — representing a 43% lower risk of death.
Amylyx is now sponsoring the <a href="https://clinicaltrials.gov/ct2/show/NCT05021536">PHOENIX Phase 3 trial (NCT05021536)</a> to confirm Relyvrio’s safety and efficacy in about 600 people with early-stage ALS, or those who have been experiencing symptoms for less than two years.
Participants are being randomly assigned to Relyvrio or a placebo for 48 weeks (nearly one year), followed by open-label treatment for up to two years.
The trial’s main goals are to evaluate Relyvrio&#8217;s safety and patients&#8217; rate of decline through changes in ALSFRS-R scores. Other goals include changes in lung function, quality of life, and survival.
The study is being conducted at several sites in the U.S. and Europe and is expected to finish in March 2024. Trial results could further support Amylyx&#8217;s regulatory applications.
Relyvrio is <a href="https://alsnewstoday.com/news/amylyxs-amx0035-now-under-review-approval-europe/">under regulatory review in Europe</a> and also is being tested as a potential treatment of other neurodegenerative conditions, including <a href="https://alzheimersnewstoday.com/what-is-alzheimers-disease/">Alzheimer’s disease</a> and Wolfram syndrome.

Tag: Albrioza

New Brunswick adds Albrioza for ALS to its public health plan

Quebec, Ontario add Albrioza to public reimbursement list

EMA branch opposes conditional approval of AMX0035 in Europe

Amylyx agrees to deal for Albrioza reimbursement in Canada for ALS

Amylyx Selects Neopharm to Market AMX0035 for ALS in Middle East