Activated PI3Kdelta Syndrome (APDS) – Market outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2020 To 2030
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). 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 neurodevelopment delay and growth retardation. APDS due to gain-of-function mutations in the genes encoding p110δ (PIK3CD) and p85α (PIK3R1) are termed APDS1 and APDS2, respectively. 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 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.
The competitive
landscape of Activated PI3Kdelta Syndrome (APDS) includes country-specific
approved and pipeline therapies. Any asset/product-specific designation or
review and Accelerated Approval are tracked and supplemented with analyst
commentary.
KOLs insights of Activated
PI3Kdelta Syndrome (APDS) across 8 MM market from the center of Excellence/
Public/ Private hospitals participated in the study. Insights around current
treatment landscape, epidemiology, clinical characteristics, future treatment
paradigm, and Unmet needs.
Activated
PI3Kdelta Syndrome (APDS) Market
Forecast: Patient Based Forecast Model (MS. Excel Based Automated
Dashboard) which Data Inputs with sourcing, Market Event, and Product Event,
Country specific Forecast Model, Market uptake and patient share uptake, Attribute
Analysis, Analog Analysis, Disease burden, and pricing scenario, Summary, and
Insights.
S. No Asset Company Stage
1 leniolisib Pharming
Group N.V Phase 2/3
2 Nemiralisib GlaxoSmithKline Phase 2
3 CDZ173 Novartis
Pharmaceuticals Phase 2/3

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