The Medicines and Healthcare products Regulatory Agency (MHRA) has approved Pharming Group’s Joenja (leniolisib) to treat patients with the rare immune disease activated phosphoinositide 3-kinase delta syndrome (APDS).
Joenja is now the first drug to be approved in the UK for APDS and has been indicated for use in adults and adolescents aged 12 years and older who weigh at least 45kg.
Approximately 30 to 40 people in the UK are affected by APDS, an inherited disorder where patients are unable to fight infections because their immune systems do not work properly.
The main symptoms usually occur in the first two years of life and include repeated lung infections, as well as a failure to grow and develop normally. Patients are also more prone to develop blood cell cancers, such as lymphoma.
Taken orally twice daily, Joenja is a small molecule phosphoinositide 3-kinase delta inhibitor that was approved for the same patient population in the US last year.
The drug’s latest approval occurred through the International Recognition Procedure (IRP), a fast-track approval process that takes into account prior authorisations from other regulatory partners.
The MHRA’s decision was supported by results from a 12‑week placebo-controlled study of 31 APDS patients. By day 85 of the trial, patients randomised to receive Joenja saw a reduction in lymph node size and a 37% improvement in naïve B cells counts compared to placebo.
Julian Beach, interim executive director of healthcare quality and access, MHRA, said: “Approval of [Joenja] through the IRP means we have facilitated access for people suffering from this serious disease to an effective medicine to fight infections and reduce symptoms of the disease… As with all medicines, we will keep its safety under close review.”
Also commenting on the authorisation, Pharming’s chief executive officer, Sijmen de Vries, said: “The MHRA approval of Joenja, the first treatment option specifically indicated for APDS, is an important milestone for people in the UK living with this debilitating disease.”
Joenja is now being evaluated by the National Institute for Health and Care Excellence regarding reimbursement on the NHS, and applications are currently under review with other regulatory authorities globally.
