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Oral Edaravone

Background

Mitsubishi Tanabe Pharma Corporation (MTPC) began studying an intravenous (IV) formulation (Radicava) of edaravone for ALS in 2001. Radicava was approved for the treatment of ALS in Japan and South Korea in 2015, with subsequent approvals in Canada, Switzerland, China, Indonesia, Thailand and USA (2017).

Treatment with Radicava follows a regimen of initial treatment daily for 14 days, followed by a 14-day drug free period, and subsequent cycles of daily dosing for 10 of 14 days, followed by a 14-day drug free period.

During clinical development of IV Radicava, Mitsubishi Tanabe Pharma America (a wholly owned subsidiary of MTPC) began a series of steps to determine if an oral suspension of edaravone could add to or replace the IV formulation for treatment of people living with ALS. First, bioequivalence had to be established to demonstrate that the oral edaravone could provide identical exposure as the established IV formulation. Multiple studies were performed.

The process began with a phase 1, open-label, single-dose crossover study in 42 participants, comparing 105 mg oral edaravone with 60 mg/hr IV formulation. Through comparing the pharmacokinetics (how it moves through the body) of both, it was concluded that the two formulations, at their respective doses, demonstrated equivalent exposure. This study was published (here ) in 2021.

Further, two additional phase 1 studies were performed. The first study was a placebo-controlled, randomized, single-blind study of increasing oral edaravone doses from 30 to 300 mg in 56 participants. The second study, involving 84 participants, assessed whether oral edaravone interacted with other drugs and also examined potential pharmacokinetic differences between people of different racial backgrounds. In both cases, oral edaravone was considered safe and tolerable with no significant drug interaction effects or differences between racial backgrounds. These studies can be found here.

Trial Design and Results

Following bioequivalence, the next step was to undertake a large safety and tolerability trial of oral edaravone at the intended clinical dose. On December 9, 2021, MTPA announced the results of the global phase 3 clinical trial assessing safety and tolerability of oral edaravone over 24 weeks. The study enrolled 185 participants with ALS across 50 sites in the United States, Canada, Europe and Japan and included a long-term safety extension study of up to 96 weeks. No severe treatment emergent adverse events (TEAEs) were reported.

On May 13, 2022, it was announced that the U.S. Food and Drug Administration (FDA) had approved RADICAVA ORS and it became available as of June 16. Marketing authorization for RADICAVA® Oral Suspension was granted in Canada (November 2022) and Switzerland (May 2023), and RADICUT® Oral Suspension 2.1% was granted regulatory approval in Japan in December 2022.

Simultaneously, MTPA launched a phase 3b, multi-national, randomized, doubleblind study to evaluate daily dosing of oral edaravone as compared to the current IV dosing regimen. Over 48 weeks, 380 participants either received oral edaravone once daily or for 10 days followed by an 18 day placebo suspension. This trial evaluated a comparison of change in ALSFRS-R as the primary outcome measure and additional outcomes including slow vital capacity, quality of life (ALSAQ-40).

It was set to read out late in 2023, however an interim analysis has shown that daily oral edaravone is not better than the current 2-weeks on/2-weeks off oral dosing regimen which matches the IV infusion regime. They have therefore discontinued the trial and also the associated expanded access program (EAP).

A Dutch company, Treeway, in partnership with Ferrer, conducted a Phase 3 trial (ADORE) of their own oral edaravone product, FNP122, which was taken daily. FNP122, formerly TW001, was previously announced to have bioavailability comparable to IV edaravone at doses used in treatment with Radicava. The ADORE trial was a 48-week trial that tested FNP122 against placebo in 300 patients across Europe. Patients who completed the ADORE trial had the opportunity to join an extension study where they received FNP122 for up to three years (more info on the clinical trial can be found here). In January 2024 Ferrer published a press release declaring that the Phase 3 ADORE trial did not meet primary or key secondary endpoints (press release available here). After 48 weeks daily dosing, the treated group did not show any significant difference in their ALSFRS-R score when compared to placebo. There was also no difference in the long-term survival rate between treated and placebo group after 48 or 72 weeks. This study, however, confirmed that FNP122 was safe and well tolerated. 

Based on the lack of efficacy of oral edaravone FNP122, Ferrer announced that its open label extension study (ADORETX, more info here) will be concluded. Full publication and results for the phase 3 trial and extension study of FNP122 are still pending.

To address community concerns, MTPA issued a statement discussing the differences between the results and products used in the trials by both companies (available here).

Summary

Considering the available evidence, it is the opinion of the SAC that oral edaravone appears to be safe and tolerable, which underscores our commitment to prioritizing patient safety. However, the efficacy of oral edaravone has been brought into question following the results of the ADORE trial, prompting the need for a critical re-evaluation of its therapeutic benefits and potential impact on patient outcomes.

While the safety and tolerability profile of oral edaravone is encouraging, the findings from the ADORE trial highlight the need for a reassessment of its role in the treatment landscape. The SAC recognizes the importance of transparently addressing these uncertainties and engaging in constructive dialogue to better understand their implications for patient care. This is further complicated because oral edaravone is a currently marketed product in some countries, but not others, and full results for ADORE are currently pending.

We understand that this news may raise concerns and uncertainties within the community. It is essential for people living with ALS to maintain open communication with healthcare providers and explore available treatment options.

International Alliance of ALS/MND Associations

March 2024


The original language of communication is English and any translation cannot be guaranteed for accuracy of messaging.

Primary Sidebar

Drugs in Development

  • AB Science – Masitinib
  • BrainStorm Cell Therapeutics – NurOwn
  • Clene Nanomedicine – CNM-Au8
  • Collaborative Medicinal Development – CuATSM
  • ILB – Tikomed
  • Kadimastem – AstroRx
  • Methylcobalamin
  • Mitsubishi Tanabe Pharma America – Oral Edaravone
  • Neuronata-R/Lenzumestrocel
  • NeuroSense – PrimeC
  • Neuvivo – NP001
  • T Regulatory Cell Therapies
  • Prilenia Therapeutics – Pridopidine
  • SOD1 Therapies & Trials

  • Andrietta

    Andrietta

  • Norm MacIsaac,  ALS Society of Canada,  ALS Society of Quebec,  Diagnosed 2014

    Norm MacIsaac, ALS Society of Canada, ALS Society of Quebec, Diagnosed 2014

  • Catherine Pearce, Australia

    Catherine Pearce, Australia

  • Marco Antonio Alvarez Mercado, Mexico

    Marco Antonio Alvarez Mercado, Mexico

  • Cath Muir

    Cath Muir
    Cath

  • Osiel Mendoza, Diagnosed 2016 ,  ALS Therapy Development Institute, USA

    Osiel Mendoza, Diagnosed 2016 , ALS Therapy Development Institute, USA

  • Jean Waters, Diagnosed 2004, MND Association of England, Wales and N Ireland

    Jean Waters, Diagnosed 2004, MND Association of England, Wales and N Ireland

  • Kris Van Reusel, Belgium

    Kris Van Reusel, Belgium

  • David Solomon, Diagnosed 2015, MND Association of England, Wales and N Ireland

    David Solomon, Diagnosed 2015, MND Association of England, Wales and N Ireland

  • 83

    83

  • Luis Antonio Pimenta Lima, Brazil

    Luis Antonio Pimenta Lima, Brazil

  • Wilfried Leusing

    Wilfried Leusing

  • Dick Dayton, USA

    Dick Dayton, USA

  • Karl Hughes, Diagnosed 2010 , IMNDA,  Ireland

    Karl Hughes, Diagnosed 2010 , IMNDA, Ireland

  • IMG_1211

    IMG_1211

  • Jose Espinosa, Argentina

    Jose Espinosa, Argentina

  • Guido De Mets, Belgium

    Guido De Mets, Belgium

  • Tammy Moore and Eddy Lefrancois

    Tammy Moore and Eddy Lefrancois

  • Ailsa Malcolm-Hutton, Diagnosed 2013,  MND Association of England, Wales and N Ireland

    Ailsa Malcolm-Hutton, Diagnosed 2013, MND Association of England, Wales and N Ireland

  • Sharon Corosanite, Diagnosed 2014 , ALS Hope Foundation, USA

    Sharon Corosanite, Diagnosed 2014 , ALS Hope Foundation, USA

  • Graham Johnson, MND Australia

    Graham Johnson, MND Australia

  • Monica Soriano, Diagnosed 2011 ,  Asociación ELA , Argentina

    Monica Soriano, Diagnosed 2011 , Asociación ELA , Argentina

  • Andrea Zicchieri, Associazione conSLAncio Onlus, Italy

    Andrea Zicchieri, Associazione conSLAncio Onlus, Italy
    AndreaZicchieri_conSLAncioItaly

  • Anthony (Tony) Lynch, MND New South Wales, Diagnosed 2016, Australia

    Anthony (Tony) Lynch, MND New South Wales, Diagnosed 2016, Australia

  • Denis Blais, Diagnosed 2015 , ALS Canada

    Denis Blais, Diagnosed 2015 , ALS Canada

  • Sally Pauls, Diagnosed 2006 , Les Turner ALS Foundation

    Sally Pauls, Diagnosed 2006 , Les Turner ALS Foundation

  • Michel Perrozzo, ARSLA, Diagnosed 2015, France

    Michel Perrozzo, ARSLA, Diagnosed 2015, France

  • Paul Launer, USA

    Paul Launer, USA

  • Margreth Burger-Saile, Diagnosed 2011,  ALS Schweiz,  Switzerland

    Margreth Burger-Saile, Diagnosed 2011, ALS Schweiz, Switzerland

  • Purningam Jacob, Diagnosed 2012 , Asha Ek Hope Foundation, India

    Purningam Jacob, Diagnosed 2012 , Asha Ek Hope Foundation, India

  • Francisco Perez Palop, Diagnosed 2013 , FUNDELA, Spain

    Francisco Perez Palop, Diagnosed 2013 , FUNDELA, Spain

  • Chris McCauley, Diagnosed 2015 , ALS Canada

    Chris McCauley, Diagnosed 2015 , ALS Canada

  • Xian-Zhang Niu, Diagnosed 2006 , Shaanxi ALS Association, China

    Xian-Zhang Niu, Diagnosed 2006 , Shaanxi ALS Association, China

  • Frank "Papa" Taylor

    Frank “Papa” Taylor

  • Michael Lee, Australia

    Michael Lee, Australia

  • Bob Simonds and Drew O'Neil, USA

    Bob Simonds and Drew O’Neil, USA

  • Duncan Bayly , MND Australia

    Duncan Bayly , MND Australia

  • Shay Rishoni

    Shay Rishoni

  • Shay Rishoni, Diagnosed 2011 , Prize4Life, Israel

    Shay Rishoni, Diagnosed 2011 , Prize4Life, Israel

  • David Watson,  MND Scotland,  Diagnosed 2018

    David Watson, MND Scotland, Diagnosed 2018

  • Mauril Belanger

    Mauril Belanger

  • Kirsty Gerlach, MND New Zealand, Diagnosed 2017

    Kirsty Gerlach, MND New Zealand, Diagnosed 2017

  • Roxana Canova, Diagnosed 2012 ,  Asociación ELA Argentina

    Roxana Canova, Diagnosed 2012 , Asociación ELA Argentina

  • Marcel R. Wernard, Diagnosed 2016,  ALS Patients Connected,  The Netherlands

    Marcel R. Wernard, Diagnosed 2016, ALS Patients Connected, The Netherlands

  • Art Eggert, USA

    Art Eggert, USA

  • Greg Heydet, ALS Hope Foundation, USA

    Greg Heydet, ALS Hope Foundation, USA

  • Hollister

    Hollister
    hollister

  • Peng Yi-Wen

    Peng Yi-Wen

  • David Bishop

    David Bishop

  • Bruno Leanza Mantegna, Diagnosed 1999 , AISLA Onlus, Italy

    Bruno Leanza Mantegna, Diagnosed 1999 , AISLA Onlus, Italy

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