Pharming Group's Joenja® receives FDA approval for the treatment of rare APDS condition in Adults and Pediatric patients

Pharming Group N.V. has received FDA approval for Joenja® (leniolisib) to treat activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in patients aged 12 and above. Joenja® is a selective PI3Kδ inhibitor, administered orally, and is the first and only treatment approved for APDS in the US, which is a rare and progressive primary immunodeficiency. The FDA reviewed the Joenja® application for APDS under Priority Review.

Publish Date: 24-03-2023 Source: Pharming Group N.V.

Activated PI3K delta Syndrome (APDS), also referred to as PASLI disease, is a combined immunodeficiency that arises from dominant, gain-of-function mutations in the genes encoding p110δ (PIK3CD) and p85α (PIK3R1), the catalytic and regulatory subunits of phosphoinositide 3-kinase δ (PI3Kδ). This condition results in the hyperactivation of the PI3K/AKT/mTOR/S6K signaling pathways in immune cells. Patients with APDS can experience immune-deficient and immune-dysregulation features such as recurrent respiratory tract infections, bronchiectasis, herpesvirus infections, autoimmunity, non-neoplastic lymphoproliferation, and lymphoma, as well as neurodevelopmental delay and growth retardation. APDS1 and APDS2 are terms used to distinguish between APDS due to gain-of-function mutations in the genes encoding p110δ (PIK3CD) and p85α (PIK3R1), respectively. Unfortunately, there is currently no curative therapy available for APDS. However, certain treatment regimens can be implemented for APDS patients, such as antibiotic prophylaxis and immunoglobulin replacement therapy. Hematopoietic stem cell transplantation (HSCT) has proven to be successful in several APDS patients and may be a viable treatment option, especially in young patients.

Activated PI3K Delta Syndrome (APDS) incidence in the United States is approximately 1.75 to 2.3 patients per million population. Individuals with APDS often experience a wide range of inflammatory and autoimmune manifestations at a high frequency.

However, the current Activated PI3K Delta Syndrome (APDS) treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (Nemiralisib, CDZ173, etc..) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for the Activated PI3K Delta Syndrome treatment. The key companies in the advanced development stage are GlaxoSmithKline, Pharming Technologies B.V., etc..

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Activated PI3K Delta Syndrome (APDS) to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2021 to 2032.

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