Pharming Technologies B.V.

Address: Darwinweg 24

Sponsor Overview

assignment

Specializations

Primary Immunodeficiency and Immune Dysregulation

Pharming Technologies B.V. focuses clinical research on primary immunodeficiency disorders, with particular attention to common variable immunodeficiency and related immune dysregulation conditions. Its studies address the clinical burden of abnormal immune function in both adult and pediatric populations.

Common variable immunodeficiency
Immune dysregulation
Primary immunodeficiency

Research in this area includes ongoing work in patients with clinically significant immune dysfunction across multiple age groups.

Activated Phosphoinositide 3-Kinase Delta Syndrome

The sponsor has a strong therapeutic interest in activated phosphoinositide 3-kinase delta syndrome (APDS), a rare immune disorder associated with dysregulated immune signaling. Clinical activity spans pediatric patients as well as individuals with established immune dysregulation.

APDS
Immune signaling abnormalities
Pediatric immunology

This area includes research in children from early childhood through school age, reflecting a sustained focus on rare immune-mediated disease.

Pediatric Immunology

Pharming Technologies B.V. conducts research in pediatric immunology, with studies involving children affected by APDS and related immune disorders. The clinical portfolio includes younger pediatric populations, highlighting interest in age-specific immune disease management.

Children with APDS
Pediatric patients
Immune disorder care

These studies extend across early childhood and later childhood cohorts, supporting therapeutic development in rare immune conditions affecting children.

Pharmacology and Clinical Pharmacokinetics

The sponsor also maintains research interest in pharmacology and clinical pharmacokinetics related to leniolisib, including studies in healthy volunteers and individuals with altered organ function. This work supports understanding of how the therapy behaves across different patient groups.

Bioavailability
Hepatic function
Drug-drug interaction

These investigations complement the sponsor’s immune-disease portfolio by addressing treatment behavior in varied clinical settings.

Sponsored Trials

Trial Name	Country	Status
An Open-label, Single Arm Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Leniolisib in Pediatric Patients (Aged 1 to 6 Years) with APDS (Activated Phosphoinositide 3-Kinase Delta Syndrome) Followed by an Open-label Long-term Extension	Portugal Spain	Not Recruiting
An open-label, single-arm extension study to evaluate the long-term safety, tolerability, and efficacy of leniolisib for immune dysregulation in patients with primary immunodeficiency	Spain	Recruiting
Evaluation of Leniolisib on Pharmacokinetics of Caffeine, Midazolam, Rosuvastatin, Furosemide, and Metformin in Healthy Subjects with Activated PI3K Delta Syndrome	Germany	Not Recruiting
Evaluation of Leniolisib Phosphate for Safety, Tolerability, and Efficacy in Patients with Common Variable Immunodeficiency	Spain	Not Recruiting
Evaluation of Leniolisib Phosphate in Pediatric Patients Aged 4-11 with Activated Phosphoinositide 3-Kinase Delta Syndrome: Safety, Pharmacokinetics, and Efficacy	France	Not Recruiting
Evaluation of Plasma Pharmacokinetics of Leniolisib in Subjects with Impaired Hepatic Function and Normal Hepatic Function in Activated Phosphoinositide 3-Kinase Delta Syndrome	Germany Hungary	Not Recruiting
Open-label extension study of KL1333 for long-term safety, tolerability, and efficacy in adults with primary mitochondrial disease	Belgium Czechia Denmark France Germany Italy Spain	Recruiting
Relative Bioavailability Study of Leniolisib Phosphate in Various Dosages in Healthy Volunteers with Activated Phosphoinositide 3-Kinase Delta Syndrome	Germany	Not Recruiting