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Two Canadian provinces — Prince Edward Island, and Newfoundland and Labrador — are covering the cost of the oral formulation of <a href="https://alsnewstoday.com/radicava-edaravone-for-als/">Radicava</a> (edaravone) for eligible residents with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
Patients will have access through their provincial drug plans to <a href="https://alsnewstoday.com/radicava-edaravone-for-als/#Indications">Radicava Oral Suspension</a>, developed along with the infusion formulation by <a href="https://www.mt-pharma.co.jp/e/" rel="nofollow">Mitsubishi Tanabe Pharma</a>. The medication will be covered by special authorization under the PEI Pharmacare Formulary as well as the Newfoundland and Labrador Prescription Drug Program.
Residents typically have <a href="https://www.gov.nl.ca/hcs/prescription/nlpdp-plan-overview/">a medicine copay</a>, usually a small fee, to cover on their own.
&#8220;We would like to thank the Governments of Prince Edward Island and Newfoundland and Labrador for supporting eligible patients living with ALS to have publicly funded access to Radicava Oral Suspension,&#8221; Andy Zylak, president of Mitsubishi Tanabe Pharma Canada (MTP-CA), said in a <a href="https://www.newswire.ca/news-releases/mitsubishi-tanabe-pharma-canada-announces-that-company-s-oral-treatment-formulation-for-amyotrophic-lateral-sclerosis-als-has-been-added-to-the-provincial-drug-plans-in-prince-edward-island-newfoundland-and-labrador-810205320.html">company press release</a>.
[recommended-reading id=87664]
<h3>Oral formulation of Radicava seen as more convenient ALS treatment option</h3>
MTP-CA is a subsidiary of <a href="https://www.mt-pharma-america.com/" rel="nofollow">Mitsubishi Tanabe Pharma America</a>, which commercializes Radicava formulations in the U.S. There, the oral therapy is sold under the brand name Radicava ORS.
&#8220;To date, Radicava Oral Suspension is listed on the majority of provincial public drug plans. MTP-CA will continue to work with the remaining provincial, territorial and federal drug plans to help ensure that Radicava Oral Suspension is listed on all public formularies across the country,&#8221; Zylak added.
Edaravone, the active ingredient in both the oral and infusion therapies, is a free radical scavenger that helps to clear the body of the reactive molecules that can led to oxidative stress, a type of damage implicated in ALS. Their removal is expected to lessen the motor neuron damage that causes <a href="https://alsnewstoday.com/symptoms-of-als/">muscle weakness and other ALS symptoms</a>, helping to slow disease progression.
An intravenous formulation of Radicava was <a href="https://alsnewstoday.com/news/radicava-now-approved-to-treat-als-patients-in-canada/">approved in Canada in 2018</a>, becoming the first new ALS therapy available in the country in about 20 years. It is covered across Canadian provinces and territories, as well as through Veterans Affairs Canada and Indigenous Services Canada.
Health Canada <a href="https://alsnewstoday.com/news/radicava-oral-suspension-approved-canada-treat-als/">approved Radicava Oral Suspension</a> in 2022, providing ALS patients with a treatment seen as an easier, more convenient alternative to the infusion therapy. In addition to the two latest provinces, Radicava&#8217;s oral formulation is listed on public drug plans of Ontario, Quebec, Alberta, British Columbia, New Brunswick, Saskatchewan, and Nova Scotia.
&#8220;Access to new treatments plays a key role in helping people and their families live their best possible life in the face of an <a href="https://alsnewstoday.com/als-diagnosis/">ALS diagnosis</a>,&#8221; said Kathy MacNeill, past president of the ALS Society of Prince Edward Island.
According to MTP-CA, most private health insurance plans in the country also cover the oral medication.
&#8220;People living with ALS do not have the luxury of time to wait for access to new medications to slow the progression of their disease,&#8221; said Cheryl Power, executive director of the ALS Society of Newfoundland and Labrador. &#8220;For this reason, we&#8217;re pleased that people impacted by ALS in this province will now have publicly funded access to Radicava Oral Suspension.&#8221;

Radicava Oral Suspension added to drug plans of 2 Canadian provinces

<a href="https://www.mt-pharma-america.com/">Mitsubishi Tanabe Pharma America</a> (MTPA), which had been testing a once-daily regimen of its oral therapy <a href="https://alsnewstoday.com/radicava-edaravone-for-als/">Radicava ORS</a> (edaravone) for <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS), announced that it is discontinuing the Phase 3b trial and its extension study.
That decision comes after an interim analysis by an independent data monitoring committee (IDMC) showed the once-daily treatment is not likely to work better than the approved on/off regimen.
Still, the IDMC’s recommendation to stop the study was not due to safety or efficacy concerns, and this decision will not impact the commercial availability of Radicava ORS, according to the company.
“Research in ALS has always been extraordinarily difficult, but our continued efforts to advance our knowledge is critical to discover the full potential of oral edaravone as an approved treatment for people with ALS,” Gustavo A. Suarez Zambrano, MD, MTPA’s vice president of medical affairs, said in a <a href="https://www.mt-pharma-america.com/media/news/press-releases/2023/07/31/mitsubishi-tanabe-pharma-america-announces-update-on-global-phase-3b-mt-1186-a02-postmarketing-study-of-oral-edaravone-in-als">company press release</a>.
“We are incredibly grateful to all the study participants, investigators, caregivers and clinical trial staff for their participation, as we remain steadfastly committed to the ALS community,” Suarez Zambrano added.
[recommended-reading id=79659]
<h3>Post-marketing study fulfills MTPA commitment to FDA</h3>
Edaravone, the active ingredient in Radicava and Radicava ORS, is designed to offset oxidative stress, a type of cellular damage implicated in ALS. The therapy aims to prevent damage to motor neurons — nerve cells that control body movement — thereby slowing disease progression.
The into-the-vein (intravenous) formulation of Radicava <a href="https://alsnewstoday.com/news/fda-approves-radicava-first-new-als-therapy-in-20-years-and-cause-for-hope/">earned</a> FDA approval in 2017, and the oral formulation was <a href="https://alsnewstoday.com/news/fda-approves-radicava-ors-oral-suspension-edaravone-als/">approved in the U.S.</a> last year. A liquid suspension taken either orally or via a feeding tube, Radicava ORS was designed to offer the same efficacy as the original therapy but in a more convenient and less burdensome formulation for patients.
Both are given in 28-week or roughly seven-month cycles. In an initial treatment cycle, patients receive daily treatment for 14 consecutive days, followed by two weeks without treatment. Then, the therapies are taken daily over 10 of the next 14 days, followed by another two-week break.
The FDA&#8217;s approval of Radicava ORS was supported by <a href="https://alsnewstoday.com/news/als-treatment-radicava-ors-fares-well-phase-3-clinical-trial/">clinical trial results</a> demonstrating that 105 mg of the oral formulation delivered similar amounts of edaravone to the body and had the same efficacy in slowing disease progression as the 60 mg used in the intravenous version.
The now-discontinued <a href="https://clinicaltrials.gov/ct2/show/NCT04569084">Phase 3b MT-1186-A02 study (NCT04569084)</a> was conducted to fulfill a post-marketing commitment that MTPA made to the FDA after the regulatory agency approved Radicava’s intravenous formulation. It was designed to evaluate the approved Radicava ORS on/off dosing regimen versus once-daily dosing.
The study <a href="https://alsnewstoday.com/news/post-marketing-als-trial-approved-radicava-ors-fully-enrolled/">enrolled 384 ALS patients</a>, ages 18-75, whose symptoms had started less than two years prior. The trial’s main goal was to evaluate changes in disease progression, as assessed by the ALS Functional Rating Scale-Revised (ALSFRS-R), over 48 weeks, or about one year.
The trial&#8217;s IDMC conducted its pre-planned interim analysis after 190 patients — half of the planned study population — reached the 48-week assessment.
Based on the interim data from the primary endpoint, the IDMC concluded that there is a low statistical probability for the once-daily dosing regimen to be more effective than the current on/off regimen. As a result, study discontinuation was recommended.
[quote=&#8221;center&#8221;]Our learnings from this study will support further research, including real-world evidence and biomarker data generation.[/quote]
Nonetheless, the preliminary data did show that the efficacy of the on/off dosing regimen of Radicava ORS was consistent with data from the clinical trials that supported FDA approval of both Radicava formulations. Also, no new safety concerns were reported.
“Our learnings from this study will support further research, including real-world evidence and biomarker data generation,” Suarez Zambrano said.
Supported by the findings, MTPA was released from its post-marketing commitment to the FDA. The company is now notifying trial investigators at study sites of these findings.
MT-1186-A02’s extension study, the <a href="https://classic.clinicaltrials.gov/ct2/show/NCT05151471">Phase 3b MT-1186-A04 trial (NCT05151471)</a>, also will be canceled. Complete trial data will be communicated at a future date, the company noted in its release.

MTPA to stop Phase 3 trial of approved Radicava ORS for ALS

A Phase 3b study evaluating the long-term safety and effectiveness of two dosing regimens of <a href="https://alsnewstoday.com/radicava-edaravone-for-als/">Radicava ORS</a> (edaravone) — an approved oral formulation of the medicine — in people with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) is now fully enrolled.
The study was designed to fulfill a post-marketing commitment that <a href="https://www.mt-pharma-america.com/">Mitsubishi Tanabe Pharma America</a> (MTPA) made to the U.S. Food and and Drug Administration (FDA) after the regulatory agency <a href="https://alsnewstoday.com/news/fda-approves-radicava-first-new-als-therapy-in-20-years-and-cause-for-hope/">approved</a> the into-the-vein (intravenous) formulation of Radicava in 2017.
The medication&#8217;s oral formulation, Radicava ORS, <a href="https://alsnewstoday.com/news/fda-approves-radicava-ors-oral-suspension-edaravone-als/">earned</a> its U.S. approval in 2022.
&#8220;We&#8217;re very pleased to announce the completion of trial enrollment in our Phase 3b study of Radicava ORS, representing an important achievement in our ongoing commitment to the fight against ALS,&#8221; Atsushi Fujimoto, president of MTPA, said in a <a href="https://www.prnewswire.com/news-releases/mitsubishi-tanabe-pharma-america-announces-enrollment-completion-for-global-phase-3b-study-of-radicava-ors-edaravone-in-als-301779833.html">company press release</a>.
&#8220;We are deeply grateful to the study participants and their families, as well as study investigators and clinical staff for working so diligently to help us meet this milestone,&#8221; Fujimoto added.
[recommended-reading id=82739]
<h3>Phase 3b study testing 2 different doses of oral therapy</h3>
Radicava ORS is a liquid suspension taken either orally or via a feeding tube. As with the intravenous formulation, it is given in 28-day cycles.
The first cycle consists of daily treatment for 14 days followed by a two-week period (a second 14 days) without the medication. In subsequent cycles, Radicava is taken daily for 10 of the next 14 days, followed again by two weeks without treatment.
Approval of Radicava ORS was based on <a href="https://alsnewstoday.com/news/als-treatment-radicava-ors-fares-well-phase-3-clinical-trial/">clinical trial data</a> indicating that a 105 mg dose of the oral treatment delivers similar amounts of edaravone — Radicava&#8217;s active ingredient — to the body and is as good at slowing disease progression as the intravenous version (60 mg).
Now, the global <a href="https://clinicaltrials.gov/ct2/show/NCT04569084">MT-1186-A02 study (NCT04569084)</a> is designed to compare the approved Radicava ORS on/off dosing regimen against once-daily dosing.
A total of 384 ALS patients, ages 18-75, whose symptoms emerged in the two years prior to the trial, were enrolled across 80 sites in the U.S., Canada, Europe, and Asia.
After an eight-week screening period, participants will either receive Radicava ORS every day, or be given the medication and a placebo. In the medication/placebo group, patients will receive Radicava ORS for 14 days and a placebo for 14 days in the first 28-day cycle, followed by 10 days of treatment and 18 days on a placebo for the next cycles.
The trial&#8217;s main goal is to evaluate changes in disease progression, as assessed by the ALS Functional Rating Scale-Revised (ALSFRS-R), over 48 weeks, or nearly one year. Secondary goals include changes in lung function and in measures of patient-reported quality of life, as well as the time patients live without requiring ventilatory support.
Top-line results from the trial, which already is underway, are expected in 2024.
&#8220;We look forward to seeing how the results of this study evaluating an alternative dosing regimen for Radicava ORS build upon our existing data to support its long-term efficacy and safety profile for people living with ALS, and how it may increase our understanding of the full potential of the medication as a treatment option,&#8221; said Michael D. Weiss, MD, of the University of Washington Medical Center, who is the study&#8217;s lead investigator.
Radicava works by combating oxidative stress, a type of cellular damage that&#8217;s been implicated in ALS. In doing so, it&#8217;s expected to protect nerve cells from damage and slow disease progression.
Intravenous Radicava also is approved in Japan, South Korea, Canada, and Switzerland, among other countries. The oral version has approvals in the U.S., Canada, and Japan.

Post-marketing trial of 2 Radicava ORS doses now fully enrolled

Radicava ORS, an oral formulation of <a href="https://alsnewstoday.com/radicava-edaravone-for-als/">Radicava</a> (edaravone), is now available to U.S. veterans living with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS), the therapy’s developer, <a href="https://www.mt-pharma-america.com/" rel="nofollow">Mitsubishi Tanabe Pharma America</a> (MTPA), announced.
The therapy was added to the U.S. Department of Veterans Affairs National Formulary (VANF) in June 2022, shortly after its May <a href="https://alsnewstoday.com/news/fda-approves-radicava-ors-oral-suspension-edaravone-als/">approval by the U.S. Food and Drug Administration</a> (FDA). This was an initial step in making Radicava ORS available to service members who are part of the Veterans Affairs&#8217; (VA) integrated healthcare system.
Approval at the national level also is required for <a href="https://www.va.gov/formularyadvisor/drugs/4041481-EDARAVONE-SUSP-ORAL">dispensing this medication</a>, and that process now is &#8220;in place,&#8221; MTPA reported, making the treatment available to patients within the VA system.
[recommended-reading id=82505]
<h3>Radicava in an oral suspension formulation can be taken at the home</h3>
&#8220;Given the devastating nature of ALS and the demand for additional treatment options, we are thrilled to see Radicava ORS added to the VA&#8217;s National Formulary,&#8221; Atsushi Fujimoto, MTPA’s president, said in a <a href="https://www.prnewswire.com/news-releases/radicava-ors-edaravone-added-to-us-department-of-veterans-affairs-national-formulary-301768724.html">company press release</a>.
The VANF comprises a <a href="https://www.pbm.va.gov/PBM/nationalformulary/VA_National_Formulary_FEB_2023.xlsx">list of prescription medications</a> and products available across all VA facilities. Its goal is to ensure a therapy is appropriate and safe for patient use, with criteria that follows the FDA&#8217;s approval label for a treatment.
&#8220;Some studies have shown that military veterans are twice as likely to develop ALS than those who haven&#8217;t served in the military,&#8221; said Juan Reyes, a U.S. veteran with ALS and patient ambassador for MTPA.
&#8220;The addition of Radicava ORS to the VA National Formulary symbolizes not only the dedication and recognition of veterans by MTPA and the VA, but also a significant step forward for those in the ALS community in need of an alternate treatment option,&#8221; Reyes added.
Radicava ORS is an oral formulation of edaravone, a medication that works to slow ALS progression by reducing oxidative damage to cells. Oxidative stress is believed to drive nerve cell death, as neurons are particularly sensitive to it.
FDA approval was based on data showing that Radicava ORS delivers similar amounts of edaravone to the body as the intravenous (into-the-vein) infusion formulation of Radicava, and seems to be equally effective at slowing disease progression.
As a therapy that can be self-administered at home, taken by mouth or via feeding tube, it can be more convenient and easier to use less than Radicava.
The oral therapy follows the same dosing regimen as the infusion treatment, with both administered in 28-day cycles. In a first treatment cycle, Radicava ORS is taken daily for 14 days, followed by 14 days without treatment. Subsequent treatment cycles consist of daily dosing for 10 of 14 consecutive days, followed by 14 days (two weeks) of no treatment.
Patients are advised to take Radicava ORS in the morning, after overnight fasting, and to not eat — drinking only water, if wanted — for one hour after the drug&#8217;s administration.
The oral formulation of edaravone is also <a href="https://alsnewstoday.com/news/radicava-oral-suspension-approved-canada-treat-als/">approved in Canada</a>, where it is sold as Radicava Oral Suspension, and Japan, where it is marketed as Radicut Oral Suspension 2.1%.

Radicava ORS, oral form of edaravone, available to US veterans

Note: This story was updated March 10, 2023, to correct a secondary headline to note the therapy was tracked for less than a year. The story also clarifies the timing of results from a Phase 3 clinical trial and that edaravone is believed to reduce oxidative stress.
Radicava ORS, an oral formulation of <a href="https://alsnewstoday.com/radicava-edaravone-for-als/">Radicava</a> (edaravone), was well tolerated among people with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) over nearly a year of treatment in a clinical trial.
Findings from the trial had <a href="https://alsnewstoday.com/news/edaravone-oral-form-shows-good-tolerability-over-one-year-use/">been announced </a><a href="https://alsnewstoday.com/news/edaravone-oral-form-shows-good-tolerability-over-one-year-use/">previously</a> by the therapy&#8217;s developer <a href="https://www.mt-pharma-america.com/">Mitsubishi Tanabe Pharma America</a> (MTPA), and 24-week data from the trial helped form the basis of the U.S. Food and Drug Administration (FDA)&#8217;s <a href="https://alsnewstoday.com/news/fda-approves-radicava-ors-oral-suspension-edaravone-als/">approval of Radicava ORS last year</a>.
The results have now undergone peer review and were detailed in the study, &#8220;<a href="https://onlinelibrary.wiley.com/doi/full/10.1002/mus.27768">Oral edaravone demonstrated a favorable safety profile in patients with amyotrophic lateral sclerosis after 48 weeks of treatment</a>,&#8221; published in <a href="https://onlinelibrary.wiley.com/journal/10974598">Muscle &amp; Nerve</a>. The work was funded by MTPA.
[recommended-reading id=82194]
&#8220;We&#8217;re encouraged by the data that we continue to collect from the global Phase 3 trial of Radicava ORS, demonstrating a favorable safety profile after 48 weeks of treatment,&#8221; Gustavo Suarez Zambrano, MD, vice resident of medical affairs at MTPA, said in a <a href="https://www.prnewswire.com/news-releases/mitsubishi-tanabe-pharma-america-announces-48-week-results-from-global-open-label-phase-3-trial-of-radicava-ors-edaravone-in-als-301756250.html">press release</a>. &#8220;These data build upon the study&#8217;s 24-week findings that supported the FDA approval of Radicava ORS.&#8221;
While the mechanism of action of edaravone, the active agent in Radicava and Radicava ORS, is unknown, it is believed to reduce oxidative stress — a type of cell damage that’s thought to play a critical role in driving <a href="https://alsnewstoday.com/causes-of-als/">nerve cell death in ALS</a>. The original therapy was designed to be delivered intravenously (via infusion into the bloodstream), and results from clinical trials showed that it <a href="https://alsnewstoday.com/news-posts/2020/03/30/radicava-effective-slowing-als-progression-across-1-year-use-patients-trial-data/">slowed functional decline</a> in people with early ALS.
Radicava ORS was designed to deliver the same active ingredient, but in an oral form that would be more convenient for patients. Pharmacological studies have demonstrated that a 105 mg dose of oral Radicava ORS delivers the same amount of edaravone as the approved 60 mg dose of intravenous Radicava. MTPA conducted a <a href="https://clinicaltrials.gov/ct2/show/NCT04165824">Phase 3 clinical trial (NCT04165824)</a> aiming to assess the safety of the oral formulation.
The study enrolled 185 adults <a href="https://alsnewstoday.com/als-diagnosis/">diagnosed with ALS</a> at sites in the U.S., Canada, Japan, Germany, France, and Italy. The average participant age was around 60 years, and the average disease duration was about 1.5 years. About 80% of patients had <a href="https://alsnewstoday.com/forms-of-als/">limb-onset disease</a>, while the rest had bulbar-onset.
<h3>Four-week cycles</h3>
All of the patients were treated with Radicava ORS, administered in four-week treatment cycles similar to how the intravenous formulation is given: two weeks of taking the therapy, and then two weeks off in the first cycle, and then 10 days of treatment in the first two weeks, followed by two weeks off in subsequent cycles.
Most of the patients (87%) also were being treated with riluzole (sold as <a href="https://alsnewstoday.com/als-treatment/rilutek-riluzole/">Rilutek</a> and other formulations), and nearly one in five (18.4%) had been on intravenous Radicava previously.
The study lasted 48 weeks (almost a year). About 25% of the participants did not complete the full 48 weeks of treatment, mainly due to experiencing safety issues, death due to ALS, or due to the patient&#8217;s choice. The researchers said this rate is &#8220;comparable with or slightly lower than other recent clinical trials in ALS.&#8221;
Safety-related events were reported in nearly every participant over the course of the study, though many of these events were judged to be a result of ALS disease progression and not considered related to treatment with Radicava ORS.
By 48 weeks, about one quarter (24.9%) of the participants had experienced safety-related events that were judged by study investigators to be likely side effects of the oral therapy. The most common side effects were fatigue, dizziness, headache, and constipation. No treatment-related side effects were judged to be serious.
<h3>Findings consistent with IV Radicava</h3>
These findings were generally consistent with the known safety profile of intravenous Radicava, the researchers said.
&#8220;The 48-week results from this trial suggest that RADICAVA ORS was well tolerated, and support its long-term use as a treatment for ALS. Our hope is for these data to provide additional information to physicians who are considering this oral treatment option for their patients,&#8221; said Gary Pattee, MD, a neurologist and ALS specialist who works in Nebraska.
Over the course of the study, the average forced vital capacity (FVC) — a standardized measure of lung function based on how much someone can forcibly exhale — decreased by an average of 25.1%. Again, the researchers said this is consistent with prior trial results for intravenous Radicava, where the average decline in FVC was 15.6% for those on therapy, compared to 20.4% for patients given a placebo.
&#8220;This trial provides safety data to support the use of oral [Radicava ORS], which expands on existing evidence supporting the use of FDA-approved [intravenous Radicava] and the recently approved oral suspension formulation,&#8221; the scientists concluded.

Radicava ORS fares well in Phase 3 clinical trial

Note: This story were updated Feb. 14, 2023, to correct that ICER analyses found both Radicava ORS and Relyvrio are not cost-effective for the treatment of ALS.
The recently approved therapies <a class="c-link" tabindex="-1" href="https://alsnewstoday.com/amx0035/" target="_blank" rel="noopener noreferrer" data-stringify-link="https://alsnewstoday.com/amx0035/" data-sk="tooltip_parent" data-remove-tab-index="true">Relyvrio</a> (sodium phenylbutyrate and taurursodiol) and <a class="c-link" tabindex="-1" href="https://alsnewstoday.com/radicava-edaravone-for-als/" target="_blank" rel="noopener noreferrer" data-stringify-link="https://alsnewstoday.com/radicava-edaravone-for-als/" data-sk="tooltip_parent" data-remove-tab-index="true">Radicava ORS</a> (edaravone) are not likely to be cost-effective for treating <a class="c-link" tabindex="-1" href="https://alsnewstoday.com/what-is-als/" target="_blank" rel="noopener noreferrer" data-stringify-link="https://alsnewstoday.com/what-is-als/" data-sk="tooltip_parent" data-remove-tab-index="true">amyotrophic lateral sclerosis</a> (ALS), according to a new analysis from the Institute for Clinical and Economic Review (ICER).
The report, &#8220;<a href="https://www.jmcp.org/doi/10.18553/jmcp.2023.29.2.216">The effectiveness and value of AMX0035 and oral edaravone for amyotrophic lateral sclerosis: A summary from the Institute for Clinical and Economic Review’s Midwest Comparative Effectiveness Public Advisory Council</a>,&#8221; was published in the <a href="https://www.jmcp.org/pb-assets/logos/JMCP_RGB_masthead.png">Journal of Managed Care and Specialty Pharmacy</a>.
Relyvrio and Radicava ORS were approved by the U.S. Food and Drug Administration (FDA) last year. Relyvrio contains two compounds — sodium phenylbutyrate and taurursodiol — thought to protect nerve cells from stress. Radicava ORS is a liquid suspension formulation of Radicava, which has been FDA-approved for ALS since 2017. It can be taken orally or through a feeding tube, whereas the original formulation was administered via infusion into the bloodstream.
&#8220;The oral formulation presents a lower burden for patients and eliminates the risks of intravenous administration, thus most patients are expected to switch to the oral formulation, and more patients overall are expected to begin use of edaravone now that an oral formulation is available,&#8221; the researchers wrote.
ICER is an independent nonprofit that provides evidence-based evaluations of the value of healthcare interventions. It analyzed the value of these two therapies using economic models that accounted for their cost as well as the expected benefit to patients&#8217; quality of life.
Results showed Radicava ORS provided an added 0.04 quality-adjusted life years (QALYs) and 0.05 equal value life years gained (evLYG) compared with standard of care alone. These are two measures that assess the quality and quantity of years gained with a given therapy. However, Radicava ORS was deemed not cost-effective at its list price of about $171,000 a year.
The list price for Relyvrio was not available at the time of the analysis so a placeholder price of $169,000 a year was used. Although patients gained 0.14 QALYs and 0.31 evLYGs when Relyvrio was added to standard of care therapies, results suggested the therapy also was not cost-effective at this price. The medicine’s actual list price is slightly lower, <a class="c-link" tabindex="-1" href="https://alsnewstoday.com/news/relyvrio-amx0035-approved-us-als-treatment/" target="_blank" rel="noopener noreferrer" data-stringify-link="https://alsnewstoday.com/news/relyvrio-amx0035-approved-us-als-treatment/" data-sk="tooltip_parent" data-remove-tab-index="true">at $158,000 a year</a>.
[recommended-reading id=76194]
<h3>Determining Radicava ORS, Relyvrio&#8217;s cost-effectiveness</h3>
The medicines’ cost-effectiveness analyses were based on data from clinical trials, in which both therapies slowed disease progression. However, the researchers emphasized that available clinical trial data is limited, restraining their analysis.
Relyvrio was approved based on findings from one Phase 2 clinical trial called <a href="https://clinicaltrials.gov/ct2/show/NCT03127514">CENTAUR (NCT03127514)</a> and its extension study, which tested it in 137 ALS patients with rapidly progressing disease. Results showed it <a href="https://alsnewstoday.com/news/amx0035-survival-als-patients-centaur-clinical-trial-analysis-amylyx/">slowed disease progression and appeared to extend survival</a>.
However, the researchers noted concerns about blinding — exit interviews showed most patients given a placebo could correctly guess that they weren&#8217;t getting the active treatment, likely due to the therapy&#8217;s taste and common digestive side effects — and sensitivity analyses by the FDA &#8220;demonstrated lower efficacy and less statistical persuasiveness on the outcomes of slowing disease progression and survival.&#8221;
An ongoing Phase 3 trial called <a href="https://clinicaltrials.gov/ct2/show/NCT05021536">PHOENIX (NCT05021536)</a> is expected to provide more detailed information about Relyvrio&#8217;s clinical profile.
Radicava ORS was approved based on pharmacological data showing it was biologically equivalent to the intravenous version. The FDA&#8217;s approval of the original therapy was supported by data from a small trial, called <a href="https://clinicaltrials.gov/ct2/show/NCT01492686">Study 19 (NCT01492686)</a>, that tested intravenous Radicava in 137 ALS patients with slow disease progression who were in its early stages.
&#8220;Despite its broad FDA label for treatment of all patients with ALS, there are generalizability and replicability concerns regarding the evidence given that the clinical benefit of [Radicava] was demonstrated in a single small trial of a narrow, well-defined population in Japan with ALS in the early stage that only represents 10% or less of the entire population of people living with ALS,&#8221; the researchers wrote.
An ICER advisory committee voted last year to affirm that the available evidence supports a net health benefit to adding Relyvrio to standard of care for ALS. It also voted to affirm that efficacy data for Radicava are sufficient for the specific subset of patients tested in trials, but not for all ALS patients.
The panel also affirmed that Relyvrio and Radicava ORS provided low long-term cost-effectiveness at the current list prices.
It also offered policy recommendations to improve care for the ALS community. Among them, the panel urged insurance companies to develop coverage policies for potential ALS therapies still undergoing review so patients can access them as soon as they&#8217;re approved. It also called on payers to cover home health and assistive devices.
The panel also recommended that therapy developers incorporate uncertainty about effectiveness into pricing decisions when medicines are launched and suggested that the FDA establish a process for conditional approval when evidence of efficacy is unclear.
This study was funded by Blue Cross Blue Shield of Massachusetts, California Healthcare Foundation, The Patrick and Catherine Weldon Donaghue Medical Research Foundation, Arnold Ventures, and Kaiser Foundation Health Plan.

ICER releases analysis on cost of ALS therapies

An oral suspension form of <a href="https://alsnewstoday.com/radicava-edaravone-for-als/">Radicava</a> (edaravone), an approved treatment of <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS), was favored for patient reimbursement in Canada by a branch of the Canadian Agency for Drugs and Technologies in Health (CADTH).
The opinion issued by the CADTH Canadian Drug Expert Committee recommended that Radicava Oral Suspension be reimbursed through the public drug plans of the country&#8217;s provinces and territories.
Certain conditions must be met, including a prescription for this formulation and the cost of oral Radicava not exceeding that of Radicava in its initial intravenous formulation.
&#8220;The positive recommendation from CADTH is an important first step in ensuring access to Radicava Oral Suspension for all eligible Canadians living with ALS,&#8221; Atsushi Fujimoto, president of <a href="https://www.mt-pharma-ca.com/" rel="nofollow">Mitsubishi Tanabe Pharma Canada</a>, said in a <a href="https://www.newswire.ca/news-releases/radicava-r-edaravone-oral-suspension-receives-positive-recommendation-from-cadth-for-the-treatment-of-patients-with-als-882129765.html">company press release</a>.
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<h3>Oral Radicava can be taken at the home by mouth or feeding tube</h3>
CADTH&#8217;s recommendation is nonbinding, with the public drug plans of each province and territory deciding whether to list the oral medication based on the agency&#8217;s recommendation and factors such as its own priorities and resources.
Mitsubishi Tanabe Pharma Canada, a subsidiary of <a href="https://www.mt-pharma-america.com/" rel="nofollow">Mitsubishi Tanabe Pharma America</a> that markets Radicava in Canada,  stated it will continue to work with insurance companies and public agencies to make Radicava Oral Suspension available to patients via public formularies and private insurance. Public formularies are lists of prescription treatments covered as benefits for those eligible.
&#8220;While we are delighted by this milestone, we know there is more work to do, and we look forward to continued discussions with stakeholders to secure coverage for Radicava Oral Suspension on public formularies,&#8221; Fujimoto said.
Oxidative stress is seen as a central mechanism in the neuronal damage taking place in ALS. Radicava is a free-radical scavenger, meaning it helps to remove the reactive molecules that can contribute to oxidative stress to lessen nerve damage and slow ALS progression.
The intravenous (IV) formulation was <a href="https://alsnewstoday.com/news/radicava-now-approved-to-treat-als-patients-in-canada/">approved in Canada</a> in 2018, and patients using it are <a href="https://als.ca/blogs/access-to-therapies-radicava-edaravone-update/">reimbursed through public formularies</a> across Canada, as well as via the Veterans Affairs Canada and Indigenous Services Canada.
Radicava Oral Suspension, <a href="https://alsnewstoday.com/news/radicava-oral-suspension-approved-canada-treat-als/#:~:text=Radicava%20Oral%20Suspension%2C%20an%20oral%20formulation%20of%20edaravone%2C,in%20Canada%20for%20treating%20amyotrophic%20lateral%20sclerosis%20%28ALS%29.">approved by Health Canada</a> in November, was developed to be as effective as Radicava IV but more convenient.  It n can be self-administered at home, either by mouth or via a feeding tube, while Radicava IV is infusion treatment administered at a center, with each infusion lasting about one hour.
Available as <a href="https://www.radicava.com/patient/taking-radicava-ors/">Radicava ORS</a> in the U.S., the medication comes in a portable bottle and includes a 5 mL oral syringe for accurate dosing. It should be taken at a dose of 105 mg (the equivalent to 5 mL of the solution) in the morning after overnight fasting. Patients are recommended to take only water for one hour after administration.
The oral formulation is given in the same dosing regimen as Radicava IV: in a first cycle, patients receive daily dosing for 14 days, followed by a two-week treatment-free period. In subsequent cycles, the medication is given for 10 of the first 14 days, followed by two weeks with no treatment.
U.S. and Canadian approvals of Radicava Oral Suspension were supported by clinical trials showing that the oral formulation delivers similar amounts of edaravone, its active ingredient, to the body.
They also were based on interim results of the open-label <a href="https://clinicaltrials.gov/ct2/show/NCT04165824">Phase 3 trial (NCT04165824)</a> in 185 ALS patients, which showed that six months of treatment with Radicava Oral Suspension was <a href="https://alsnewstoday.com/news/oral-edaravone-appears-safe-patients-interim-trial-data/">generally well tolerated and slowed functional decline</a>.

Tag: Radicava ORS