Report on the European Medicines Agency’s Assessment of Pridopidine for Huntington’s Disease

Executive Summary

This report details the refusal of the European Medicines Agency (EMA) to grant marketing authorization for the drug pridopidine, developed by Prilenia for the treatment of Huntington’s disease (HD). This decision, announced in July 2025, represents a significant setback in the global effort to achieve Sustainable Development Goal 3 (SDG 3): Good Health and Well-being, specifically concerning the provision of treatments for neurodegenerative conditions. Despite this outcome, corporate commitments to future research demonstrate resilience in pursuing SDG 9 (Industry, Innovation, and Infrastructure).

Drug Profile and Clinical Development History

Pridopidine Mechanism and Therapeutic Goals

Pridopidine is an experimental oral medication investigated for Huntington’s disease and ALS. Its development aligns directly with the objectives of SDG 3. The therapeutic mechanism of action is understood to be the activation of the sigma-1 receptor (S1R), a protein integral to managing cellular stress and maintaining neuronal health.

Summary of Clinical Trial Outcomes

The journey of pridopidine through clinical trials illustrates the challenges inherent in achieving the innovation targets of SDG 9. A review of major trials reveals a consistent pattern:

• Initial Trials (HART, MermaiHD, PRIDE-HD): These studies established that pridopidine was safe and well-tolerated by patients. However, they failed to meet their primary endpoints related to improving motor symptoms.
• Post-Hoc Analyses: Subsequent analysis of trial data suggested potential benefits in Total Functional Capacity (TFC), a measure of a person’s ability to perform daily activities. This finding provided the rationale for continued development.
• Phase 3 PROOF-HD Trial: This trial was specifically designed to measure functional outcomes rather than motor symptoms. It did not meet its primary endpoint in the overall study population. Exploratory analyses suggested favorable signals on function, cognition, and motor skills in a subgroup of patients not taking other dopamine-altering medications, but these results were deemed inconclusive for regulatory approval.

Regulatory Application and Strategic Partnerships

Marketing Authorization Application to the EMA

In September 2024, Prilenia submitted a marketing authorization application to the EMA. This action was based on the potential benefits observed in a specific sub-population of HD patients. The application process itself is a key component of the infrastructure required by SDG 9 to translate scientific research into accessible healthcare solutions.

The Role of SDG 17: Partnerships for the Goals

Subsequent to the EMA application, Prilenia announced a strategic partnership with Ferrer, a Spanish pharmaceutical company. This collaboration exemplifies SDG 17 (Partnerships for the Goals), uniting international entities to develop and commercialize a novel therapy. Such partnerships are vital for mobilizing the resources needed to address complex global health challenges and advance SDG 3.

EMA Decision and Implications for Sustainable Development

Refusal of Marketing Authorization

In July 2025, Prilenia and Ferrer confirmed that the EMA’s Committee for Medicinal Products for Human Use (CHMP) had refused the marketing authorization application for pridopidine. As a result, pridopidine will not be made available for the treatment of HD in Europe at this time.

Impact on Health and Innovation Goals

This regulatory outcome has direct consequences for several Sustainable Development Goals:

1. SDG 3 (Good Health and Well-being): The decision is a direct setback for the HD community and the broader goal of providing effective treatments for all. It delays progress in managing a debilitating neurological disease.
2. SDG 10 (Reduced Inequalities): The lack of a new therapeutic option perpetuates the health inequalities experienced by individuals and families affected by rare diseases like HD.
3. SDG 9 (Industry, Innovation, and Infrastructure): The refusal underscores the high-risk, high-cost nature of pharmaceutical innovation and the rigorous standards that must be met to ensure public safety and drug efficacy.

Future Outlook and Continued Research Efforts

Commitment to Further Research

Despite the regulatory setback in Europe, Prilenia and Ferrer have affirmed their continued commitment to the HD and ALS communities. They have announced plans to initiate a new, “potentially registrational global HD study.” This demonstrates a resilient approach to innovation (SDG 9) and a long-term dedication to achieving health-related goals (SDG 3).

The Broader HD Research Landscape

Progress toward SDG 3 is not dependent on a single therapeutic candidate. The broader HD research field remains active and promising, with multiple initiatives underway that support the goal of finding effective treatments. Notable advancements in 2025 include:

• Positive updates from trials by uniQure and PTC Therapeutics.
• Advancement of a trial by Skyhawk Therapeutics.
• Initiation of Phase 1 trials by Spark Therapeutics and Alnylam.

These diverse and innovative approaches underscore the industry’s collective and persistent effort to address the challenge of Huntington’s disease, ensuring that the path to progress, while not linear, continues to move forward.

1. Which SDGs are addressed or connected to the issues highlighted in the article?

The article discusses issues related to medical research, healthcare, innovation, and corporate partnerships, which directly connect to the following Sustainable Development Goals (SDGs):

• SDG 3: Good Health and Well-being: The entire article focuses on the development of a treatment (pridopidine) for Huntington’s disease (HD), a severe non-communicable neurological disorder. It addresses the challenges and processes involved in bringing new medicines to patients to improve their health and quality of life.
• SDG 9: Industry, Innovation, and Infrastructure: The article highlights the role of scientific research and innovation within the pharmaceutical industry. It details the long process of drug development, from understanding a drug’s mechanism (activation of the sigma-1 receptor) to conducting multiple clinical trials (HART, MermaiHD, PRIDE-HD, PROOF-HD) and seeking regulatory approval. This represents a significant investment in research and development (R&D) to advance technological and medical capabilities.
• SDG 17: Partnerships for the Goals: The article explicitly mentions the formation of a partnership to achieve a common goal. It states that “Prilenia announced a partnership with Ferrer, a Spanish pharmaceutical company. Their partnership was intended to further develop and commercialize pridopidine.” This collaboration between two private sector entities exemplifies a partnership to mobilize resources and expertise for a health-related goal.

2. What specific targets under those SDGs can be identified based on the article’s content?

Based on the article’s focus, the following specific SDG targets can be identified:

1. SDG 3: Good Health and Well-being

   • Target 3.4: “By 2030, reduce by one-third premature mortality from non-communicable diseases through prevention and treatment and promote mental health and well-being.” The effort to develop pridopidine is a direct attempt to create a treatment for Huntington’s disease, a non-communicable disease. The article mentions that post-hoc analyses suggested “possible benefits in Total Functional Capacity (TFC),” which measures a person’s ability to perform daily activities, directly relating to their well-being.
   • Target 3.b: “Support the research and development of vaccines and medicines for the communicable and non-communicable diseases…” The article is a case study of this target in action. It describes the extensive R&D process for pridopidine, including multiple clinical trials and the application for marketing authorization from the European Medicines Agency (EMA). It also mentions other companies like “uniQure and PTC Therapeutics” and “Spark Therapeutics and Alnylam” that are actively engaged in R&D for HD treatments.

2. SDG 9: Industry, Innovation, and Infrastructure

   • Target 9.5: “Enhance scientific research, upgrade the technological capabilities of industrial sectors in all countries…encouraging innovation and substantially increasing the number of research and development workers…and public and private research and development spending.” The article details the scientific research process, from the initial hypothesis about dopamine to the later understanding that pridopidine’s effects “seem to be mediated by activation of the sigma-1 receptor (S1R).” The mention of “Promising trials, innovative approaches, and new insights” and the continued commitment by Prilenia and Ferrer to initiate a new global study, despite the setback, underscore the focus on enhancing research and innovation.

3. SDG 17: Partnerships for the Goals

   • Target 17.17: “Encourage and promote effective public, public-private and civil society partnerships, building on the experience and resourcing strategies of partnerships.” The article provides a clear example of a private-private partnership formed to advance a health solution. The text states, “After Prilenia submitted their application to the EMA, they announced a partnership with Ferrer, a Spanish pharmaceutical company. Their partnership was intended to further develop and commercialize pridopidine.” This collaboration is a direct application of this target’s principle.

3. Are there any indicators mentioned or implied in the article that can be used to measure progress towards the identified targets?

Yes, the article mentions or implies several indicators that can be used to measure progress:

1. For SDG Target 3.4:

   • Total Functional Capacity (TFC) scores: The article explicitly defines this as a measure of well-being and functionality. It states, “TFC scores measure how well people can function at tasks like managing their households and finances, ability to work, drive, cook, and do other day-to-day activities.” This can serve as a direct indicator of the impact of treatments on the well-being of patients with non-communicable diseases.

2. For SDG Target 3.b:

   • Number of new medicines in clinical trials: The article names multiple trials for just one drug (“HART, MermaiHD, PRIDE-HD, and PROOF-HD”) and mentions that other companies like “Skyhawk Therapeutics,” “Spark Therapeutics and Alnylam” are also conducting trials. This count serves as an indicator of R&D activity.
   • Marketing authorization applications: The central event of the article is Prilenia’s application to the European Medicines Agency (EMA). The submission and review of such applications are key milestones and indicators of progress in drug development.

3. For SDG Target 9.5:

   • Investment in R&D: While not providing specific figures, the article implies significant private R&D spending through its description of the multi-year, multi-trial process for pridopidine. The commitment by Prilenia and Ferrer to “initiate a ‘potentially registrational global HD study’ in the near term” despite the EMA rejection indicates continued R&D investment.
   • Number of ongoing scientific research projects: The article refers to “continued scientific research” that refined the understanding of pridopidine and mentions “positive updates from uniQure and PTC Therapeutics” and new trials from others, indicating a portfolio of active research projects in the field.

4. For SDG Target 17.17:

   • Number of private-private partnerships for drug development: The article provides a specific example with the “partnership with Ferrer, a Spanish pharmaceutical company.” The formation of such collaborations can be counted as an indicator of progress towards this target.

4. Create a table with three columns titled ‘SDGs, Targets and Indicators” to present the findings from analyzing the article. In this table, list the Sustainable Development Goals (SDGs), their corresponding targets, and the specific indicators identified in the article.

Source: en.hdbuzz.net