UK Regulator Approves Pharming’s Joenja for Rare Immune Disorder APDS, Leveraging FDA Review

Approval Date:
The UK's Medicines and Healthcare products Regulatory Agency (MHRA) approved Joenja (leniolisib) on September 25, 2024, for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents aged 12 years and older who weigh 45 kg or more.

Regulatory Process:
The MHRA used the International Recognition Procedure (IRP) pathway, which allows for faster approval by leveraging the review conducted by trusted regulatory partners, in this case, the FDA's approval in March 2023.

Clinical Trial Results:
The approval was based on a 12-week placebo-controlled study that showed significant improvements in reducing lymph node size and normalizing immunophenotype in patients treated with Joenja compared to those receiving placebo.

Availability:
While approved, the availability of Joenja on the NHS will depend on a positive review by the National Institute for Health and Care Excellence (NICE), expected in December 2024, with potential availability by March 2025.

Manufacturer:
Pharming Group N.V. is the manufacturer of Joenja and has expressed its commitment to making the treatment available to patients with APDS, a rare and progressive primary immunodeficiency.