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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.amylyx.com/">Amylyx Pharmaceuticals</a> is facing a new hurdle — a second round of questions from a European Medicines Agency (EMA) committee — in its quest to have <a href="https://alsnewstoday.com/amx0035/">AMX0035</a> approved as a treatment for <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) in Europe.
The questions, from the Committee for Medicinal Products for Human Use, known as CHMP, come after the company completed a scientific advisory group meeting, in which experts come together to evaluate a specific drug and provide recommendations for approval. However, the committee said there were still certain major objections to AMX0035 that should be addressed.
Amylyx will now respond to the questions, and expects an opinion from the committee by mid-2023. The EMA will consider that opinion in its decision on AMX0035&#8217;s potential approval.
While the company had previously said it was expecting a decision from the regulatory authority <a href="https://alsnewstoday.com/news/phoenix-trial-approved-als-therapy-relyvrio-amx0035-enrolled/">in the first half of 2023</a>, updated timelines now point to &#8220;the third quarter of 2023 at the earliest,&#8221; Amylyx stated in <a href="https://www.businesswire.com/news/home/20230313005731/en/Amylyx-Pharmaceuticals-Reports-Fourth-Quarter-and-Full-Year-2022-Financial-Results">press release</a> announcing its financial results for 2022.
“We continue to engage with the European Medicines Agency as it reviews our marketing authorisation application for AMX0035,&#8221; said Joshua Cohen and Justin Klee, co-CEOs at Amylyx. &#8220;We remain focused on our efforts &#8230; to drive the broadest coverage possible for this important new therapeutic option.”
[recommended-reading id=79299]
<h3>&#8216;Much work remains,&#8217; Amylyx CEOs say</h3>
AMX0035 is an oral fixed-dose combination of two small molecules — tauroursodeoxycholic acid and sodium phenylbutyrate — that protect nerve cells from damage induced by cellular stress.
The treatment is <a href="https://alsnewstoday.com/news/amx0035-albrioza-oral-als-therapy-available-canada/">commercially available</a> to treat people with ALS in Canada, under the name Albrioza. In the U.S., it was <a href="https://alsnewstoday.com/news/relyvrio-amx0035-approved-us-als-treatment/">approved in September</a> of last year and is marketed as Relyvrio.
“2022 was an exceptionally exciting year for Amylyx, culminating with the approval of Relyvrio in the U.S. following the approval with conditions for Albrioza in Canada, marking an important step toward delivering much-needed advancements in treatment options to the ALS community,” Cohen and Klee said.
Approvals of AMX0035 in North America were supported by data from <a href="https://clinicaltrials.gov/ct2/show/NCT03127514">CENTAUR (NCT03127514)</a>, a Phase 2 trial in which the therapy was found to significantly slow functional decline and extend survival in people with the neurodegenerative disease. That trial involved 137 people with recently diagnosed and rapidly progressing ALS.
Now, the company is conducting the <a href="https://clinicaltrials.gov/ct2/show/NCT05021536">PHOENIX Phase 3 trial (NCT05021536)</a> to test the therapy in a larger group of patients. <a href="https://alsnewstoday.com/news/phoenix-trial-approved-als-therapy-relyvrio-amx0035-enrolled/">Enrollment was completed</a> last month, with a total of 664 participants who will receive the medication or a placebo for 48 weeks (nearly one year).
The trial’s main goals are to assess AMX0035&#8217;s safety and changes in disease progression, as measured with the ALS Functional Rating Scale-Revised (ALSFRS-R). Secondary measures include changes in lung function, quality of life, and survival.
After completing the trial, patients will be able to join an open-label extension part and continue receiving treatment for two more years.
[quote align=&#8221;center&#8221;]2022 was an exceptionally exciting year for Amylyx, culminating with the approval of Relyvrio in the U.S. following the approval with conditions for Albrioza in Canada, marking an important step toward delivering much-needed advancements in treatment options to the ALS community.[/quote]
Earlier this year, Amylyx entered an <a href="https://alsnewstoday.com/news/amylyx-select-neopharm-market-amx0035-middle-east/">exclusive license and distribution agreement</a> with <a href="https://www.neopharmisrael.co.il/">Neopharm</a>, which, pending its approval, would make the Israel-based company responsible for regulatory submissions and commercialization of AMX0035 in certain Middle East regions. Those regions include Israel, Gaza, the West Bank, and the Palestinian Authority.
“Our commercial launch is off to a strong start, and we are encouraged by the engagement we have seen from physicians, people living with ALS, and payors,” Cohen and Klee said.
“While we are thrilled with this progress, we are also cognizant that much work remains to meet our goal of quick and efficient access for every eligible person living with ALS,” they added.

European approval of AMX0035 for ALS faces new hurdle in review

A large Phase 3 trial designed to confirm the safety and efficacy of the approved therapy <a href="https://alsnewstoday.com/amx0035/">Relyvrio</a> (sodium phenylbutyrate and taurursodiol) for <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) is now fully enrolled.
<a href="https://www.amylyx.com/">Amylyx Pharmaceuticals</a> announced the completion of patient recruiting for the trial, called <a href="https://clinicaltrials.gov/ct2/show/NCT05021536">PHOENIX (NCT05021536)</a>, which will test the oral therapy in a total of 664 people at dozens of sites across the U.S. and Europe. Top-line data is expected in 2024.
“We are grateful for the people living with ALS and their families who are participating in PHOENIX and the dedication of the study investigators,” Joshua Cohen and Justin Klee, co-CEOs of Amylyx, said in a <a href="https://www.businesswire.com/news/home/20230202005258/en/Amylyx-Pharmaceuticals-Announces-Completion-of-Enrollment-in-Global-Phase-3-PHOENIX-Trial-of-AMX0035-in-ALS">company press release</a>.
“We remain committed to continuing research and exploring the full potential of [Relyvrio] as part of our mission to one day end the suffering caused by ALS and other neurodegenerative diseases,” they added.
Relyvrio, formerly called AMX0035, is a fixed-dose combination of two compounds — tauroursodeoxycholic acid and sodium phenylbutyrate — that block stress signals involved in nerve cell damage and ALS progression.
[recommended-reading id=79235]
<h3>Confirming the effectiveness of Relyvrio post-approval</h3>
The treatment&#8217;s effectiveness and safety was originally investigated in a Phase 2 clinical trial called <a href="https://clinicaltrials.gov/ct2/show/NCT03127514">CENTAUR (NCT03127514)</a>. That trial, completed in 2019, involved 137 adults with recently diagnosed and rapidly progressing ALS.
The results showed that Relyvrio significantly <a href="https://alsnewstoday.com/news-posts/2020/09/03/amx0035-safe-effective-in-slowing-als-functional-decline-trial-data-shows/">slowed functional decline</a> and <a href="https://alsnewstoday.com/news-posts/2020/10/19/amylyxs-amx0035-significantly-extends-als-patients-lives-long-term-trial-data-show-also-slows-functional-decline/">extended survival</a>, compared with a placebo.
Amylyx used that data to support its application for Relyvrio&#8217;s <a href="https://alsnewstoday.com/news/relyvrio-amx0035-approved-us-als-treatment/">approval in the U.S.</a> and its <a href="https://alsnewstoday.com/news/amx0035-oral-als-therapy-called-albrioza-approved-canada/">conditional approval in Canada</a> — where the therapy is marketed under the brand name Albrioza. In Canada, more data is needed to support full regulatory approval of Relyvrio.
The PHOENIX trial was designed to confirm results from CENTAUR in a larger group of ALS patients. Participants are randomly assigned to receive Relyvrio or a placebo for 48 weeks (about one year), followed by open-label treatment for up to two years. In the open-label part, both patients and researchers know exactly what therapy participants are receiving/
The trial’s main goals are to assess the therapy&#8217;s safety and changes in the rate of functional decline, as measured with the ALS Functional Rating Scale-Revised (ALSFRS-R). Other goals include changes in lung function, quality of life, and survival.
[quote align=&#8221;center&#8221;]We anticipate that PHOENIX will help us generate further data about the safety and efficacy of [Relyvrio].[/quote]
The study is being conducted at 69 sites in the U.S. and Europe as part of collaborations with the Northeast ALS Consortium (NEALS) and the Treatment Research Initiative to Cure ALS (TRICALS), respectively.
“The Phase 3 PHOENIX trial [is] an excellent collaboration between European and United States Centers for excellence in ALS research and care,” said Leonard H. van den Berg, MD, PhD, professor of neurology at the University Medical Center Utrecht and chair of the TRICALS.
&#8220;We anticipate that PHOENIX will help us generate further data about the safety and efficacy of AMX0035,&#8221; van den Berg added.
The trial is expected to conclude in 2024 and its results could further support Amylyx’s regulatory applications — including in Europe, where the medication is <a href="https://alsnewstoday.com/news/amylyxs-amx0035-now-under-review-approval-europe/">under regulatory review</a>. Amylyx expects a decision from the European Medicines Agency in the first half of 2023.
The company said the results of the ongoing trial also would likely be needed for full approval of the therapy in the European Union if conditional marketing authorization is granted.
“We are pleased to share this milestone from the PHOENIX trial, a collaborative effort to advance our scientific understanding of ALS and build upon the positive data from our Phase 2 CENTAUR trial,” said Sabrina Paganoni, MD, PhD, principal investigator of CENTAUR and a member of the NEAL&#8217;s executive committee.
Relyvrio also is being tested as a potential treatment of other neurodegenerative conditions, such as <a href="https://alzheimersnewstoday.com/what-is-alzheimers-disease/">Alzheimer’s disease</a> and Wolfram syndrome.

Tag: Amylyx Pharmaceuticals

Amylyx agrees to deal for Albrioza reimbursement in Canada for ALS

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