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 Read More….