• Skip to primary navigation
  • Skip to main content
  • Skip to primary sidebar
  • Skip to footer
  • Email
  • Facebook
  • LinkedIn
  • Twitter
  • YouTube

International Alliance of ALS/MND Associations

  • Members' Login
  • Contact
  • Join the Alliance
  • Donate
  • What is ALS/MND
  • Find a Member Association
  • Support for PALS & CALS
    • Fundamental Rights for People with ALS/MND and Caregivers
    • Research
      • Voice Preservation
      • Open Science
      • Expanded Access
      • Understanding ALS/MND Research
      • Improving Regulatory Pathways
      • Right to Try
      • US FDA Orphan Drug Designation
      • Unproven (Off-Label) Treatments
      • Open Label Extension
    • Advocacy
      • Advocacy Toolkit
      • Emergency Preparedness Toolkit
      • Equitable Access to Therapies
      • Recommendations for Trial Sponsors
    • Clinical Care
      • Genetic Counselling & Testing
      • Mental Health Support
      • Nursing and Symptom Management
      • Nutrition and Swallowing
      • Occupational Therapy and Activities of Daily Living
      • Physiotherapy and Mobility
      • Respiratory Care
      • Speech Therapy and Communication
      • Support for Family & Caregivers
      • Technology
      • Global Clinic Locator
    • Drugs in Development
      • AB Science – Masitinib
      • BrainStorm Cell Therapeutics – NurOwn
      • Clene Nanomedicine – CNM-Au8
      • Collaborative Medicinal Development – CuATSM
      • ILB – Tikomed
      • Kadimastem – AstroRx
      • Mitsubishi Tanabe Pharma America – Oral Edaravone
      • Neuronata-R/Lenzumestrocel
      • NeuroSense – PrimeC
      • Neuvivo – NP001
      • Prilenia Therapeutics – Pridopidine
      • T Regulatory Cell Therapies
      • SOD1 Therapies & Trials
    • Approved Drugs
      • Nuedexta
      • Radicava/Edaravone
      • Riluzole/Tiglutik
      • Rozebalamin/Methylcobalamin
      • Tofersen/Qalsody
    • Drugs No Longer in Development
      • Amylyx – AMX0035
      • Collaborative Medicinal Development – CuATSM
      • Cytokinetics – Reldesemtiv
      • Orphazyme – Arimoclomol
      • TUDCA Trial
  • Support for Health Professionals
    • Breaking the News in ALS/MND
    • Diagnostic Delay (in development)
  • Events/Programs
    • Calendar of Events/Programs
    • Alliance Meeting
    • Allied Professionals Forum
    • Alliance Webinars
    • ALS/MND Connect
    • Global Day
    • March of Faces
    • Patient Fellows Program
    • Global CRLI
    • International Symposium
  • About
    • Who We Are
    • ALS/MND Health Literacy Map
    • Board of Trustees
    • Advisory Councils/Committees
      • Scientific Advisory Council
      • PALS and CALS Advisory Council
      • Innovation and Technology Council
      • Advocacy and Public Policy Forum
      • Research Directors Forum
      • Governance Committee
      • Finance Committee
    • Staff
    • History
    • Archives
      • Newsletters
      • Meetings
    • Awards
      • Forbes Norris Award
      • Humanitarian Award
      • Allied Health Professional Award
      • Student Innovation Award
  • Members
    • Member Registration
    • Forgot Password

Orphazyme – Arimoclomol

BACKGROUND

Arimoclomol is an oral capsule drug that enhances a mechanism known as the heat shock response. When a cell of the body is exposed to stress or damage, the proteins required for the cell’s normal functions can change their shape or “fold” and either lose their ability to do their job or become toxic. Cells protect themselves from this misfolding of proteins by stimulating production of heat shock proteins (Hsps) that are designed to manage the proper protein refolding.

Misfolded and clumped proteins have long been a hallmark of ALS/MND and it is believed that these may contribute to multiple disease processes. Unlike other cells, neurons, and particularly motor neurons, have an impaired ability to produce an effective heat shock response. Therefore, drugs that can enhance the production of Hsps may have therapeutic value in ALS/MND.

Arimoclomol was first published to have an effect on elevating Hsps and delaying disease progression in an ALS/MND mouse model in 2004. In 2008, it was tested for safety, tolerability and pharmacokinetics in 84 people living with ALS/MND by the Northeast ALS Consortium (NEALS), indicating it could be dosed safely up to three times daily at 100 mg per dose and that it effectively crossed the blood-brain barrier. A follow up academic (non pharma, investigator-initiated) clinical trial led by Dr. Michael Benatar, examined arimoclomol in a double-blind, placebo-controlled study of 38 people with fast-progressing ALS caused by SOD1 mutations at 200 mg/day over 12 months. Again, arimoclomol was deemed safe and tolerable but the indication that the drug may slow down disease progression and prolong survival was not statistically significant, and conclusions could not be drawn about its efficacy in ALS/MND. Arimoclomol was considered safe and well-tolerated, with only one person stopping treatment due to skin rash.

Orphazyme was founded in Denmark in 2009 based on work demonstrating that Hsps could also correct an abnormality in a cellular structure called the lysosome, implicated in diseases called lysosomal storage diseases. In advancing arimoclomol as an Hsp-inducing drug for these diseases, the company also initiated an ALS program, using the substantial groundwork to initiate a phase 3 clinical trial.

The multicenter, randomized, double-blind, placebo-controlled study was started in 2018, enrolling 245 people living with ALS, with a 2:1 treatment to placebo ratio and studied over 76 weeks. A measure called the Combined Assessment of Function and Survival (CAFS) was used as the primary means of determining if arimoclomol is effective in ALS, while other common measures like ALSFRS-R, survival and slow vital capacity (SVC) were also evaluated. Arimoclomol is an oral capsule that was taken three times daily for the duration of the study.

TRIAL DESIGN & RESULTS

On May 7, 2021, an Orphazyme press release stated that the “pivotal trial did not meet primary and secondary endpoints evaluating impact on function and survival”. This indicates that arimoclomol will not be further pursued as a treatment for ALS/MND. In May 2024, a publication on Lancet Neurology confirmed what stated in the press release, with further data suggesting an increase of adverse reactions in the treated compared to the placebo group. 

SUMMARY

Given the available data, it is the opinion of the SAC that there is enough evidence to conclude that at the studied dosage, Arimoclomol did not show overall benefit in individuals living with ALS/MND.

International Alliance of ALS/MND Associations
October 2024


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

Primary Sidebar

  • Luis Antonio Pimenta Lima, Brazil

    Luis Antonio Pimenta Lima, Brazil

  • Olga Cosentino, Diagnosed 2013,  Asociación ELA Argentina

    Olga Cosentino, Diagnosed 2013, Asociación ELA Argentina

  • Aida Trzmiel de Guterman, Asociacion ELA Argentina, Diagnosed 2007, Argentina

    Aida Trzmiel de Guterman, Asociacion ELA Argentina, Diagnosed 2007, Argentina

  • Lin Yong Yi, Taiwan MND Association, Diagnosed 2004

    Lin Yong Yi, Taiwan MND Association, Diagnosed 2004

  • March of Faces Photo Submission_OLGA_ELA ARGENTINA

    March of Faces Photo Submission_OLGA_ELA ARGENTINA

  • Alberto Baez Murillo, Colombia

    Alberto Baez Murillo, Colombia

  • Zelina Brito, Diagnosed 2018, Brazil

    Zelina Brito, Diagnosed 2018, Brazil

  • Margarita Pizarro, Asociacion ELA Argentina, Diagnosed 2017, Argentina

    Margarita Pizarro, Asociacion ELA Argentina, Diagnosed 2017, Argentina

  • JP

    JP

  • Jon Newsome, Les Turner ALS Foundation, USA

    Jon Newsome, Les Turner ALS Foundation, USA

  • David Bishop

    David Bishop

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

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

  • Andrea Zicchieri, Associazione conSLAncio Onlus, Italy

    Andrea Zicchieri, Associazione conSLAncio Onlus, Italy
    AndreaZicchieri_conSLAncioItaly

  • Susan Keldani, Les Turner ALS Foundation, USA

    Susan Keldani, Les Turner ALS Foundation, USA

  • Brian Parsons

    Brian Parsons

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

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

  • Jose Rivero Muñoz, Diagnosed 2015, FYADENMAC, Mexico

    Jose Rivero Muñoz, Diagnosed 2015, FYADENMAC, Mexico

  • Jorge Melo, ABrELA, Brazil

    Jorge Melo, ABrELA, Brazil

  • Mahmood Anwar, UK

    Mahmood Anwar, UK

  • Juvenal Bayona Romero

    Juvenal Bayona Romero

  • Maria Santos Garcia Tellez, Diagnosed 2017 , FYADENMAC, Mexico

    Maria Santos Garcia Tellez, Diagnosed 2017 , FYADENMAC, Mexico

  • Michel Perrozzo, ARSLA, Diagnosed 2015, France

    Michel Perrozzo, ARSLA, Diagnosed 2015, France

  • Oliver Juenke, Germany

    Oliver Juenke, Germany

  • Enzo Maccarrone, AISLA ONLUS, Italy

    Enzo Maccarrone, AISLA ONLUS, Italy

  • Monica Soriano, Diagnosed 2011 ,  Asociación ELA , Argentina

    Monica Soriano, Diagnosed 2011 , Asociación ELA , Argentina

  • Carlos Alberto Báez Murillo, ACELA, Colombia

    Carlos Alberto Báez Murillo, ACELA, Colombia

  • Laurie Petit-Jean, Diagnosed 2012 , ARSLA, France

    Laurie Petit-Jean, Diagnosed 2012 , ARSLA, France

  • Shera Mukherjee, Diagnosed 2013,  Asha Ek Hope Foundation, India

    Shera Mukherjee, Diagnosed 2013, Asha Ek Hope Foundation, India

  • Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

    Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

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

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

  • Yolanda Armendariz, Diagnosed 2017 , FYADENMAC, Mexico

    Yolanda Armendariz, Diagnosed 2017 , FYADENMAC, Mexico

  • 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

  • Teddy Hanono Annie, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

    Teddy Hanono Annie, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

  • Maurice LeClerc, ALS Canada

    Maurice LeClerc, ALS Canada

  • Hollister

    Hollister
    hollister

  • Timmy, ALS Liga

    Timmy, ALS Liga

  • MNDaSG Group PALS & CALS, Motor Neurone Disease Association, Singapore (MNDaSG)

    MNDaSG Group PALS & CALS, Motor Neurone Disease Association, Singapore (MNDaSG)

  • Tison, USA

    Tison, USA

  • Rolf Mauch, Association ALS Switzerland, Diagnosed 2015

    Rolf Mauch, Association ALS Switzerland, Diagnosed 2015

  • IMG_2658

    IMG_2658

  • Nicholas (Nic) Bowman, MND Association of South Africa,  Diagnosed 2016,  Australia

    Nicholas (Nic) Bowman, MND Association of South Africa, Diagnosed 2016, Australia

  • IMG_1211

    IMG_1211

  • Liam Dwyer, England

    Liam Dwyer, England

  • Danny Reviers, Diagnosed 1979 , ALS Liga België, Belgium

    Danny Reviers, Diagnosed 1979 , ALS Liga België, Belgium

  • John and Loretta Russo, USA

    John and Loretta Russo, USA
    final3878

  • Ali Var, Turkey

    Ali Var, Turkey

  • Eric Von Schaumburg, USA

    Eric Von Schaumburg, USA

  • Mauril Bélanger, Diagnosed 2015 , ALS Canada

    Mauril Bélanger, Diagnosed 2015 , ALS Canada

  • Angie Bordaen, Diagnosed 2014,  ALS Liga België, Belgium

    Angie Bordaen, Diagnosed 2014, ALS Liga België, Belgium

  • Jose Espinosa, Argentina

    Jose Espinosa, Argentina

Learn more about the March of Faces

Latest Tweets

  • Just now

Drugs No Longer in Development

  • Amylyx – AMX0035
  • Collaborative Medicinal Development – CuATSM
  • Cytokinetics – Reldesemtiv
  • Orphazyme – Arimoclomol
  • TUDCA Trial

Footer

Subscribe to our Bi-Monthly Newsletter

Sign up to receive updates and to hear what's going on in the International Alliance of ALS/MND Associations.

"*" indicates required fields

 
This field is for validation purposes and should be left unchanged.
  • Email
  • Facebook
  • LinkedIn
  • Twitter
  • YouTube
Return to top of page

Contact | Disclaimer | Privacy Notice & Cookies | Sitemap

Copyright © 2025 The International Alliance of ALS/MND Associations. All rights reserved.


Registered in England: Charity Number 1079504 · Site built by graphics.coop · Powered by WordPress · Members' login