This month we held an important meeting, inviting PWS Associations and stakeholders from around the world to discuss the potential for global access to VYKAT™ XR (DCCR) for treating hyperphagia in people with PWS. The session focused on the process of bringing treatments to market and how IPWSO can play a role in catalysing action to ensure faster access for all.
Professor Steffen Thirstrup, Chief Medical Officer at the European Medicines Agency explained how the drug approval process works in Europe for rare diseases. He made it clear: patient voices matter. While approval is centralised in the EU, each country still handles its own pricing and reimbursement — which can be tricky.
View Steffen Thirstrup’s presentation on our YouTube channel.
Charles Gore, Executive Director of the Medicines Patent Pool spoke honestly about the challenges of bringing new treatments for rare diseases to places like Africa, Latin America, and parts of Asia. These include high prices, complicated rules, and limited health systems. But there are possible solutions — like countries teaming up to buy the drug together at a lower price.
View Charles Gore’s presentation on our YouTube channel.
Dr. Anish Bhatnagar, Chief Executive Officer of Soleno Therapeutics reaffirmed the company’s dedication to global equity. They are actively engaging with country-level stakeholders and advocacy groups to support local regulatory submissions and explore pricing models appropriate to each region’s capacity.
Professor Tony Holland, President of IPWSO underscored that while VYKAT™ XR is a major breakthrough, best practices in environmental control, behavioural support, and professional care remain vital for improving quality of life for individuals with PWS.
This meeting was a great reminder that we need advocacy, awareness, and collaboration to make sure everyone with PWS has a fair chance at the best possible care.
As part of IPWSO’s global mission, we continue to advocate for equitable access to these life-changing treatments, working with regulatory agencies, policymakers, and the rare disease community.
We’ll keep you posted as more countries start the approval process. In the meantime, keep raising awareness and supporting your local PWS groups — your voice really does make a difference.
With hope,
The IPWSO Team