MHRA Approves Pharming's Joenja as First UK Treatment for Children Aged 4 to 11 With APDS

Pharming Group won UK approval to extend Joenja (leniolisib) to children aged 4 to 11 weighing at least 27 kg with activated PI3K delta syndrome, making it the first treatment approved in the UK specifically for this group. Stock Titan reported the Medicines and Healthcare products Regulatory Agency decision on November 6, 2026.

What the MHRA Extension Covers

Pharming is a Leiden-based biotechnology company that develops and commercializes medicines for rare immune and genetic diseases, with two approved products: RUCONEST for hereditary angioedema and Joenja for APDS. The new authorization adds eligible younger children to Joenja's existing UK treatment population, which had covered adults and adolescents aged 12 and older.

The approval includes 40 mg and 50 mg twice-daily dosing for eligible pediatric patients in this weight group. Joenja is an oral, selective phosphoinositide 3-kinase delta inhibitor, and Pharming said it expects the product to become available to patients covered by the expanded indication in February 2027. The company said it will work with the NHS and relevant stakeholders to support timely access.

"This approval is the latest step in our continued expansion of Joenja across age groups and geographies, giving eligible children in the U.K. aged 4 to 11 with APDS an approved treatment option," said Leverne Marsh, interim Co-CEO and Chief Commercial Officer of Pharming.

Dr Austen Worth, Pediatric Immunology Consultant at Great Ormond Street Hospital for Children, said the extension "represents a significant step forward for children and families living with APDS." Pharming said the MHRA decision was supported by data from its pediatric clinical program, conducted under the MHRA-agreed Pediatric Investigation Plan for leniolisib, along with additional clinical pharmacology and safety assessments. Leniolisib was generally well-tolerated in patients aged 4 and above, the company said.

Joenja's Earlier Regulatory Steps

According to akta.pro company records, the European Commission granted marketing authorization for Joenja in May 2026, making it the first approved treatment for APDS in the EU and valid across all 27 member states plus Norway, Iceland and Liechtenstein. Japan's Ministry of Health, Labour and Welfare approved Joenja in March 2026 for APDS patients aged 4 and older, the first approval globally for children aged 4 to 11, with OrphanPacific handling commercialization. The FDA approved a supplemental new drug application for leniolisib in December 2025 expanding APDS treatment to children aged 4 to 11.

Pharming's Recent News Flow

On November 5, 2026, Yungjin Pharm transferred Japanese rights to KL1333 to Pharming Technologies under a supplemental agreement, leaving Pharming with worldwide rights outside South Korea, The future of trading reported. Pharming reported Q3 2026 revenue of $117.6 million, up 21%, and operating profit of $29.9 million, up 89%, with RUCONEST revenue up 14% to $93.8 million, The Motley Fool reported on November 4, 2026. On October 14, 2026, Pharming presented Phase II topline data for leniolisib in genetically identifiable primary immunodeficiencies with immune dysregulation at ESID 2026, showing a mean 26.4% spleen volume reduction, Stock Titan reported.

When Joenja Reaches UK Children

Pharming said Joenja is expected to become available to patients covered by the expanded indication in February 2027. The company also said leniolisib is under regulatory review for APDS in several other countries.