Thank You and See You in Mallorca!

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We extend our heartfelt thanks to everyone who made IPIC2025 an unforgettable success!

With over 900 attendees, IPIC2025 was a truly inspiring gathering of our global community—filled with learning, collaboration, and shared commitment to advancing care for people with primary immunodeficiencies.

As we close this remarkable chapter, we are already looking ahead with excitement to our next congress: IPIC2027, taking place in beautiful Mallorca, Spain, from November 3–5, 2027.

In 2027, our congress will elevate primary immunodeficiency care as we learn, connect, and make lasting memories on the vibrant island of Mallorca.

Thank you once again for your continued support and engagement. We can’t wait to welcome you to Mallorca in 2027 for what promises to be another milestone event for the PI community. Until then, stay connected—and keep the spirit of collaboration alive!

🎥 Relive the highlights of IPIC2025 by watching our recap video here.

 

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IPOPI Applauds EMA’s Positive Opinion on Waskyra™ for Wiskott-Aldrich Syndrome

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IPOPI has welcomed the positive opinion issued by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). The opinion recommends granting marketing authorisation in the European Union for Waskyra™, a groundbreaking ex vivo gene therapy for Wiskott-Aldrich Syndrome (WAS), a rare and life-threatening primary immunodeficiency.

“This marks a significant milestone for gene therapy in the European Union, offering new hope for patients with Wiskott-Aldrich Syndrome,” said Johan Prévot, Executive Director of IPOPI and Board Member of the AGORA Foundation. “This positive opinion will not only increase access to life-saving therapies for those affected by WAS but could also pave the way for the development of more innovative treatments for other primary immunodeficiencies and rare disorders,” added Martine Pergent, IPOPI President. For more information, see here.

 

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The IPOPI Jose Drabwell Research Grant Programme Winners

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The IPOPI Jose Drabwell Research Grant Programme is an initiative aimed at promoting scientific and clinical research in the field of primary immunodeficiencies.

The second grant call in 2025, supported by Grifols and Kedrion, ran from April to August, inviting applicants from around the world to seize the opportunity to fuel innovation in the field with patient-centred and knowledge-generating projects.

IPOPI is pleased to announce the winners of the IPOPI Jose Drabwell Research Grant Programme 2025, who will benefit from funding for 2 years to carry out the following projects:

 

  • Nguyen Thi Thanh Hiep

Affiliation: University of Medicine and Pharmacy, Ho Chi Minh City, Vietnam

Project Title: Evaluation of the diagnostic value and the correlation between TREC, KREC concentrations and lymphocyte counts in pediatric patients suspected of inborn errors of immunity

📌 This project evaluates whether measuring TREC and KREC levels can serve as cost-effective tools to support early screening and diagnosis of inborn errors of immunity in Vietnamese pediatric patients.

 

  • Dr Zuzana Parackova

Affiliation: Department of Immunology, Faculty Hospital in Motol and 2nd Faculty of Medicine, Charles University, Prague, Czech Republic

Project Title: METSTAT: Metformin as a Novel Immunomodulator in STAT1 Gain-of-Function Interferonopathy

💊 This project will test whether the widely used drug metformin can be repurposed to safely improve immune function and quality of life in patients with the rare disorder STAT1 gain-of-function.

 

  • Dr Helen L. Leavis

Affiliation: University Medical Center Utrecht, Department of Rheumatology and Clinical Immunology and Center of Translational Immunology, Utrecht University, Utrecht, The Netherlands

Project Title: European multicenter diagnostic trial of CVID enteropathy

🔬 This project will establish a multicenter cohort of CVID patients to develop and validate diagnostic tools for CVID-associated enteropathy, aiming to improve early recognition and guide safer, more effective treatments.

 

  • Prof Nicolette Nabukeera-Barungi

Affiliation: Department of Paediatrics and Child Health, School of Medicine, College of Health Sciences, Makerere University, Kampala, Uganda

Project Title: Improving Access to Quality Care for Inborn errors of immunity Using Low-Cost Techniques at Mulago National Referral Hospital, Uganda

🌍 This project in Uganda aims to improve early detection and care for children with inborn errors of immunity by implementing screening techniques, training healthcare workers and caregivers, and collecting baseline prevalence data through a national referral hospital study.

 

We wish the winners the best of luck and success on these projects and would like to thank everyone who applied.

 

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IPOPI Renews Mandate at EMA Patients’ Working Party

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IPOPI is proud to announce that it has renewed its mandate as a member of the Patients’ and Consumers’ Working Party (PCWP) at the European Medicines Agency (EMA). This new term highlights the strong commitment to speaking up for people living with primary immunodeficiencies (PIDs). It also shows IPOPI’s dedication to supporting patient-centered healthcare policies across Europe.

The Role of the PCWP in European Medicines Regulation

The PCWP plays an important role at the EMA. It makes sure that patients’ and consumers’ views are part of how medicines are evaluated, regulated, and supervised. As a long-standing member of this group, IPOPI shares valuable knowledge drawn from the real-life experiences of people with primary immunodeficiencies in Europe and around the world.

Including patient perspectives helps ensure that new medicines and treatments truly meet patients’ needs. It is also crucial for improving safety, quality of care, and trust in the healthcare system.

Working Together for Better Access to Treatments

Thanks to its membership in the PCWP, IPOPI can work closely with other patient and consumer organisations. It also engages directly with EMA representatives and key stakeholders in healthcare and the pharmaceutical industry. This collaboration helps advocate for policies that improve access to safe, effective, and high-quality medicines for people with rare diseases, including primary immunodeficiencies.

Moreover, IPOPI aims to raise awareness about the importance of early diagnosis and better treatment options for PIDs. Through its work, the organisation strives to improve the quality of life for people affected by these rare conditions.

Strengthening Patient Engagement in the Regulatory Process

IPOPI believes that patients must play an active role in the life cycle of medicinal products. This includes drug development, clinical trials, regulatory approval, and post-marketing monitoring. By sharing the patient experience, IPOPI helps make sure that regulatory decisions focus on what truly matters to people living with rare diseases.

Dedicated Representatives for IPOPI

In this renewed mandate, IPOPI will be represented by Otilia Stanga, IPOPI Board member and Treasurer, as the full member of the PCWP. Leire Solis, IPOPI’s Health Policy and Advocacy Director, will serve as the alternate member. Their experience and dedication will help IPOPI bring the voice of PID patients to important discussions about regulatory science, patient safety, and new therapies.

Looking Ahead

IPOPI remains committed to working with the EMA and other partners to promote patient-focused policies and improve access to essential treatments. The organisation will continue to ensure that the voices of people living with primary immunodeficiencies are heard in Europe and beyond.

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1 - With Juan Garcia Burgos from EMA

IPOPI at EMA’s 30-Year Scientific Conference

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Juan Garcia Burgos (EMA) and Johan Prévot (IPOPI)

On 25 June 2025, IPOPI proudly took part in the European Medicines Agency (EMA)’s 30th anniversary scientific conference, held at EMA’s headquarters in Amsterdam, the Netherlands. The event marked three decades of groundbreaking achievements in medicines regulation and regulatory science, bringing together key partners and stakeholders to reflect on the past and look towards the future.

The conference gathered representatives from the European institutions and agencies, the European medicines regulatory network, international partners, patient and healthcare professional organisations, industry organisations, the Dutch government, and other esteemed guests. Participants explored the latest trends, innovations, and challenges in the regulation of medicines, as well as public and animal health.

IPOPI was represented by our Executive Director, Johan Prévot. Of particular interest for people living with primary immunodeficiencies (PIDs) and other rare diseases, the meeting was an opportunity to delve into the evolution of regulatory science and discuss how it can continue to advance the development and availability of safe and effective medicines, taking into account disruptive technologies,  the need to keep the public trust in science and a fast moving environment

IPOPI has long been active at the EMA level been and since 2006 formally involved in the EMA’s work, ensuring that the patient voice is considered in regulatory processes and decisions. This longstanding collaboration underscores our commitment to supporting patient-centred regulatory science and improving access to essential treatments.

IPOPI congratulates EMA on its 30 years of dedicated work in safeguarding public and animal health across Europe and beyond. We look forward to continuing our collaboration in the years to come.

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