Trials investigate targeted APDS treatment in a paediatric setting

Cancer research

The first patient has been dosed in a Phase III study evaluating a paediatric granulated formulation of leniolisib in children with activated phosphoinositide 3-kinase delta syndrome (APDS).

Pharming’s investigational drug leniolisib is an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor.

At sites in the US, Japan, and the EU, the clinical trial will evaluate the safety, tolerability, and efficacy of leniolisib in 15 children, one to six years of age, who have a confirmed APDS diagnosis.

Dr Eveline Wu, Division Chief, Paediatric Rheumatology & Associate Professor of Paediatric Rheumatology and Allergy/Immunology at The University of North Carolina School of Medicine, commented: “As a physician who has seen how significantly APDS can affect children’s lives, I’m excited that a targeted treatment may soon become available for even younger patients. While standard supportive therapies can ease some of the progressive, immune-related symptoms that characterise APDS, leniolisib is designed to help prevent those symptoms from arising.”

Another paediatric study, announced in February 2023, is ongoing and will evaluate 15 children, four to 11 years of age, receiving leniolisib’s tablet formulation for APDS.

Leniolisib, marketed under the brand name Joenja in the US, received approval from the US Food and Drug Administration (FDA) for the treatment of APDS in adult and paediatric patients 12 years of age and older in March 2023.

Anurag Relan, Chief Medical Officer of Pharming, said: “We look forward to generating the necessary supporting data to facilitate additional regulatory filings, thereby ensuring that patients from as broad an age range as possible can potentially benefit from this disease-modifying treatment.”

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