MHRA uses fast-track process to approve first medicine for rare immune disease APDS

The Medicines and Healthcare products Regulatory Agency (MHRA) has on Friday approved leniolisib phosphate (Joenja) to treat a rare immune disease known as activated phosphoinositide 3-kinase delta syndrome or APDS in adults and adolescents aged 12-years-old and older who weigh 45kg or more.

The medicine was approved via a fast-track approval process for medicines, known as the International Recognition Procedure (IRP), which allows the MHRA to consider the expertise and decision-making of trusted regulatory partners for the benefit of UK patients.

The decision follows an approval for the drug by the US Food and Drug Administration (FDA). This is the first time the MHRA has approved a new medicine following FDA approval.

The MHRA said it considered the assessment made by the US regulator as part of its own review, facilitating a rapid approval process.

APDS is an inherited disorder where the patient is unable to fight infections because the immune system does not work properly.

The main symptoms usually occur in the first two years of life and include repeated lung infections and a failure to grow and develop normally.

APDS is a long-term debilitating and life-threatening condition due to repeated lung infections that can lead to bronchiectasis (enlargement and inflammation of part of the airways). Patients with APDS are more prone to develop blood cell cancers, like lymphoma.

The recommended dose is 1 tablet twice daily, approximately 12 hours apart in adults and adolescents from 12 years weighing 45 kg or more.

“We are focused on providing UK patients access to safe and effective medicines in the shortest possible time,” Julian Beach, MHRA interim executive director of healthcare quality and access, said.

“Approval of leniolisib 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.”

The marketing authorisation was granted to Pharming Technologies B.V via the International Recognition Procedure.

The IRP is open to applicants that have already received an authorisation for the same product from one of MHRA’s specified trusted regulators. These are the regulatory authorities from Australia, Canada, the European Union, Japan, Switzerland, Singapore and the US.