MHRA Approves Joenja as First Drug for Rare APDS

The Medicines and Healthcare products Regulatory Agency (MHRA) has granted approval for Pharming Group’s Joenja (leniolisib) to treat patients suffering from activated phosphoinositide 3-kinase delta syndrome (APDS), a rare immune disorder. This approval marks Joenja as the first medication in the United Kingdom specifically aimed at treating APDS, and it is intended for use in adults and adolescents aged 12 and above who weigh a minimum of 45 kilograms.

APDS is a genetic condition that affects approximately 30 to 40 individuals in the UK. Patients with this disorder have impaired immune systems, making them vulnerable to infections. The symptoms of APDS typically emerge within the first two years of life and include recurrent lung infections, stunted growth, and developmental delays. Additionally, patients with APDS are at an increased risk of developing blood cell cancers, such as lymphoma.

Joenja, an orally administered medication taken twice daily, functions as a small molecule inhibitor of the phosphoinositide 3-kinase delta enzyme. It received approval for the same patient demographic in the United States the previous year. The latest approval by the MHRA was facilitated through the International Recognition Procedure (IRP), an expedited approval process that considers previous authorizations from other regulatory bodies.

The MHRA’s decision was supported by the findings of a 12-week placebo-controlled study involving 31 patients with APDS. By day 85 of the trial, patients who were randomly assigned to receive Joenja exhibited a reduction in lymph node size and a 37% improvement in naïve B cell counts compared to those who received a placebo.

Julian Beach, interim executive director of healthcare quality and access at MHRA, remarked: "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."

Sijmen de Vries, chief executive officer of Pharming, also commented on the authorization, stating: "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 currently under evaluation by the National Institute for Health and Care Excellence for potential reimbursement through the NHS. Additionally, applications for approval are under review by other regulatory authorities worldwide.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!