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MHRA approves Joenja for children aged 4-11 with APDS in the U.K., expanding existing indication
PHARPharming Group N.V.
The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has approved an extension of the marketing authorization for Pharming’s Joenja (leniolisib) to include children aged 4 to 11 years weighing at least 27 kg with activated PI3K delta syndrome (APDS). This approval makes Joenja the first treatment specifically authorized in the U.K. for this pediatric age group, adding 40 mg and 50 mg twice-daily dosing options. The expanded indication is expected to become available to patients covered by the NHS in February 2027. Pharming Group N.V. is a global biotechnology company that develops and commercializes innovative medicines for people living with rare immune and genetic diseases.