Complement Deficiencies (CD) – Market outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2020 To 2030
Complement deficiencies cause susceptibility not only to bacterial infection but also to autoimmune disease. Genetically determined MBL deficiencies are observed in autoimmune diseases such as systemic lupus erythematosus (SLE) and rheumatoid arthritis (RA). Observation of the low concentrations of MBL and other complement proteins (e.g., C1q, C1r, C4) and poor complement system activation are also characteristic of inactive SLE. Complement activation almost inevitably results in tissue destruction, as seen in rheumatic diseases such as SLE, RA, vasculitis, and antiphospholipid antibody syndrome. Interest in complement in inflammatory disease has led to the development of pharmacologic complement inhibitors such as eculizumab, a humanized monoclonal anti-C5 antibody approved for paroxysmal nocturnal hemoglobinuria. Inflammatory conditions also result from hereditary or acquired deficiencies of complement regulatory proteins. For example, lack of the C1q inhibitor protein leads to undue complement activity under metabolic stress and the development of intermittent and potentially life-threatening angioedema.
The
estimated C2 deficiency is the most common complement deficiency in the USA,
with frequency estimates of 0.5 to 1 in 10,000 for homozygous C2-deficient
patients. Complement deficiencies cause susceptibility not only to bacterial
infection but also to autoimmune disease. Patients with specific complement
deficiencies are prone to SLE; lupus develops in approximately 90% of C1q-null,
75% of C4-null, and up to 30% of C2-null individuals.
The
competitive landscape 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 Complement
Deficiencies (CD) 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.
Complement
Deficiencies (CD) 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 rMenB+OMV Novartis Phase 3
2 CSL312 CSL
Behring Phase 3
3 BMN 331 BioMarin
Pharmaceutical Phase 1/2
4 NTLA-2002 Intellia
Therapeutics Phase 1/2
5 Berotralstat BioCryst
Pharmaceuticals Phase 3
6 PHA-022121 Pharvaris
Netherlands B.V. Phase 2
7 KVD824 KalVista
Pharmaceuticals, Ltd. Phase 2
8 GNR-038 AO
GENERIUM Phase 1
9 Donidalorsen Ionis
Pharmaceuticals, Inc. Phase 3
10 rhC1INH Pharming
Technologies B.V. Phase 2
Comments
Post a Comment