Winner
Pharming® UK launches a targeted therapy in the UK to revolutionize care for those with activated PI3K delta syndrome (APDS)
Summary of work
Pharming® UK’s launch of the first and only medicine designed to correct the underlying immune defect in activated PI3Kδ syndrome (APDS; an ultra-rare primary immunodeficiency [PID]) was a critical achievement. With just 12 years from disease characterization to availability of a targeted therapy, Pharming rapidly established a presence in the UK PID community, mapping specialist immunology centres, partnering stakeholders, and maximising resources. A sharp, insight-driven launch mindset secured unprecedented launch milestones. Rapid regulatory review and pre-agreed interim funding led to the first patient receiving funded therapy ~6 months post-approval and the NHS becoming the first European healthcare service to offer this medicine. Significant media coverage of its life-changing impact was generated: 9,810 Google hits “[product name] news”; 33,900 Google hits “activated PI3K delta syndrome news” (Aug 2025). BBC-interviewed patient: “This treatment brought me hope and joy, but it is bittersweet because my family passed away before they could benefit. All I’ve known is medication, needles, appointments. Now, I can find out who I truly am.” James Palmer (NHSE): “Another example of NHS commitment to offering access to innovative medicines for rare conditions.”
Judges’ comments
The work from Pharming® and Only The Brave was a compelling launch in a difficult area with many pre-launch technical barriers successfully overcome through strong focus and persistence, notably in a small team rare disease environment with limited commercial potential. It had a sharp unmet needs focus, strong partnerships, and tangible impact within a tiny patient population.
