Pharming Expands Leniolisib Data in Rare Immunodeficiencies at CIS 2026

Event summary

  • Pharming presents pediatric leniolisib data in APDS at CIS 2026 Annual Meeting (May 6-9).
  • Two Phase II trials (NCT06897358, NCT06549114) evaluating leniolisib in CVID and related disorders underway.
  • Interim safety and efficacy data from long-term extension study in pediatric APDS patients (ages 4-11).
  • Clinical experience data on leniolisib use in CVID and CVID-like disorders to be shared.

The big picture

Pharming's presentations at CIS 2026 highlight its push to expand leniolisib's clinical utility beyond APDS into other primary immunodeficiencies. The focus on pediatric data and real-world clinical experience positions the drug for potential label expansions, addressing a significant unmet need in rare immunodeficiencies. The company's strategic shift toward broader PID applications could open new revenue streams if regulatory and clinical hurdles are cleared.

What we're watching

Regulatory Progress
Whether leniolisib's regulatory reviews in Canada and other countries advance in 2026.
Clinical Trial Results
The pace at which Phase II trial results for CVID and related disorders are released in H2 2026.
Market Expansion
How Pharming positions leniolisib for broader PID applications beyond APDS.