Commercial-stage biopharmaceutical company, Pharming Group, has been granted a positive opinion by the European Medicines Agency (EMA) for its Paediatric Investigation Plan (PIP) for leniolisib. Leniolisib is a phosphoinositide 3-kinase (PI3K) inhibitor that Pharming is currently developing to treat activated PI3Kdelta syndrome (APDS). So far, patients aged 12 years and older have been included in the ongoing registration of the Phase II/III study evaluating the therapy. However, as APDS affects younger children as well, the PIP agreed upon by EMA will enable the company to enroll children as young as one year old in future studies.

Source: Pharming Group N.V.

Activated PI3Kdelta Syndrome (APDS), also known as PASLI disease, is a combined immunodeficiency resulting 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). APDS due to gain-of-function mutations in the genes encoding p110δ (PIK3CD) and p85α (PIK3R1) are termed APDS1 and APDS2, respectively. These mutations result in the hyperactivation of the PI3K/AKT/mTOR/S6K signally pathways in immune cells. Patients with APDS may develop immune-deficient and immune-dysregulation features, including recurrent respiratory tract infections, bronchiectasis, herpesvirus infections, autoimmunity, non-neoplastic lymphoproliferation, and lymphoma, as well as neurodevelopment delay and growth retardation. There is no curative therapy for APDS. However, Treatment regimes for APDS patients include antibiotic prophylaxis and immunoglobulin replacement therapy. Hematopoietic stem cell transplantation (HSCT) has succeeded in several APDS patients and can be a treatment option, especially in young patients.

• In the United States, 1.75 to 2.3 patients are being diagnosed per million populations. Patients with APDS had a high incidence and wide range of inflammatory/autoimmune manifestations.

However, the current Activated PI3Kdelta Syndrome (APDS) treatment market share, market uptake, attribute analysis concerning the most potential emerging therapies (leniolisib, Nemiralisib, and CDZ173) have 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 Activated PI3Kdelta Syndrome (APDS) treatment. Pharming Group N.V, GlaxoSmithKline, and Novartis Pharmaceuticals are the key companies in the advanced development stage.

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market insight report on Activated PI3Kdelta 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 2020 to 2030.

Thelansis specializes in pharmaceutical market research and market Insight Report Company, published reports across the therapeutic area, including rare / ultra-rare and mainstream indication. Over the period, we have built a robust repository of 6,000+ Bio-pharma reports that cover Epidemiology studies and Market forecasting based on the KOL opinions.

Competitive intelligence and track of trial results throughout the phases of development executed by a team of a mix of Scientific and Business backgrounds. As an organization, the primary focus is to provide real-world data evidence and market insight to pharmaceutical companies for their decision-making

• Delivery Office:
  B-1030, C Wing Vrindavan tech village, Outer ring road
  Bangalore- 560037
  India+91(124)404-1731
  clientsupport@thelansis.com
• Sales office:
  183 Asylum Street Hartford,
  CT-06103, USA
  Contact no. +1 (302) 380-3552
  m.berg@thelansis.com