The Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion in favor of the approval of AGAMREE® (vamorolone) for the treatment of DMD patients aged 4 years and older.
Duchenne Muscular Dystrophy (DMD) is a rare neuromuscular condition. DMD has a high unmet medical need for an effective and well-tolerated treatment for all patients. The CHMP’s positive opinion recognizes the clinical significance of AGAMREE in addressing this need. This includes the noteworthy safety benefits compared to the standard corticosteroid care for DMD. This recommendation marks a crucial step towards the potential approval of AGAMREE, but also a new patient centered model for drug development in rare diseases.
DMD patient organizations played a central role in co-creating AGAMREE® (vamorolone) in all stages of development. The contributions of patient organizations as funders, the Duchenne community as active research participants, and WDO as partner in the VISION-DMD Project, supported by Horizon 2020 have been instrumental.
This collaborative approach between patient organizations, the scientific community, industry and public funding highlights the power of patient engagement in driving meaningful and successful advancements in healthcare and drug development. The result is a DMD treatment that not only addresses part of the high unmet medical need but also reflects the collective effort and dedication of all involved parties. This is paving the way for new patient centric models in research for rare diseases.
The CHMP’s positive opinion paves the way for a potential European Commission (EC) decision on marketing authorization in late 2023. This decision holds the promise of AGAMREE becoming the first drug fully approved by the European Medicines Agency (EMA) for the treatment of patients with DMD.
In parallel, the United States is also anticipating approval of vamorolone with the Prescription Drug User Fee Act (PDUFA). The action date is set for October 26, 2023, opening the door for AGAMREE’s potential launch in the U.S. in Q1-2024.
A New Model: Patient driven research and medicines development
From the early stage of the development of this drug, patient organizations were involved as funders, advisors and research participants.
Preclinical funding and Phase 1 and early Phase 2 clinical trials were supported by 16 DMD organizations. Further clinical development received funding from the European Union’s Horizon 2020 research and innovation program under grant agreement number 667078 through the VISION-DMD Project. The business model for the development of vamorolone was based on venture philanthropy. The project accessed public grant funding and partnered with stakeholder foundations to carry out the process of developing vamorolone.
The global DMD patient community, represented by the World Duchenne Organization (WDO), played a crucial role in design, development and the successful regulatory pathway of this new DMD therapy. In the VISION-DMD project, we offered meaningful patient advice on clinical trial design, the development and selection of patient relevant outcomes, data management and use, site selections and site support. Furthermore we supported patient recruitment, support and follow up, even during the challenging Covid-19 pandemic, when most other clinical trials were experiencing big delays. All these efforts contributed to improving patient retention and the success of the clinical trials.
Returning individual clinical trial results
Within the VISION-DMD project, WDO has taken on an additional role, actively promoting the return of individual clinical trial data to participants after conclusion of the trials. We supported the creation of a framework to ensure meaningful data access for participants, while also dealing with ethical and technical issues. This collaboration stems from recognizing the inherent rights of clinical trial participants to their data and the positive impact of returning such data, including greater participant engagement in their own healthcare as well as in research.
Whitepaper: Returning Individual Clinical Trial Data Back to Participants
Dr. L. Conklin, Dr. H. Peay, S. Gaglianone, S. Bakker, E. Vroom, Dr. E. Hoffman, C. Olsen
Impact of AGAMREE® (vamorolone)
If approved, AGAMREE will be the first and only medicinal product fully approved by the EMA for the treatment of DMD in all 27 member states of the European Union, as well as Iceland, Liechtenstein, and Norway.
About AGAMREE® (vamorolone)
Vamorolone is a novel drug candidate that offers hope by modifying the downstream activity of glucocorticoid receptors without the associated toxicity. This mechanism has shown the potential to ‘dissociate’ efficacy from steroid safety concerns. For this reason, vamorolone is positioned as an alternative to existing corticosteroids (e.g. Prednisone, Deflazacort), the current standard of care in children and adolescent patients with DMD.
The clinical evidence supporting AGAMREE’s efficacy and safety comes from the pivotal VISION-DMD study and other clinical trials carried out in 11 countries. Unlike the traditionally used corticosteroids, vamorolone has shown promise in providing similar benefits while maintaining the growth curve and reducing negative effects on bone metabolism.
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- Hoffman, Eric P et al (2019). Neurology. 2019 Sep 24;93(13):e1312-e1323. doi:10.1212/WNL.0000000000008168.