Cold agglutinin disease (CAD) is a clonal B-cell lymphoproliferative disorder and a rare subgroup of autoimmune hemolytic anemia (AIHA). CAD accounts for 15–25% of AIHA and affects mainly middle aged or older individuals. The disease is characterized by the production of autoantibodies by bone marrow-derived clonal B cells which attach to erythrocytes when exposed to cold temperatures (0–4°C), causing agglutination and eventual immune-mediated hemolysis.
Author: Pharma Intelligence
Publisher: Datamonitor Healthcare
Published: 06 October 2022
Number of pages:27
This Market Spotlight report covers the Cold Agglutinin Disease market, comprising key marketed and pipeline drugs, clinical trials, regulatory events, recent events and analyst opinion, probability of success, and an epidemiology overview.
The only approved drug in the cold agglutinin disease space is Sanofi’s Enjaymo, which targets the complement pathway. It is administered via the intravenous route.
There are only two industry-sponsored drugs in active clinical development for cold agglutinin disease, with one drug each in Phase I and Phase III.
Therapies in development for cold agglutinin disease target the complement pathway and complement component 3 (C3). These pipeline drugs are administered via the intraocular, subretinal, subconjunctival, intravenous, and subcutaneous routes.
The overall likelihood of approval of a Phase I marrow or peripheral blood stimulator asset is 23.9%, and the average probability a drug advances from Phase III is 83.3%. Drugs, on average, take 10.6 years from Phase I to approval, compared to 9.9 years in the overall hematology space.
The distribution of clinical trials across Phase I–III indicates that the majority of trials for cold agglutinin disease have been in the early and mid-phases of development, with 64% of trials in Phase I–II, and 36% in Phase III.
Sanofi has five completed trials in cold agglutinin disease.
Sanofi leads industry sponsors with the highest overall number of clinical trials for cold agglutinin disease.
17 RECENT EVENTS AND ANALYST OPINION
17 Sutimlimab for Autoimmune Hemolytic Anemia (June 11, 2021)
19 KEY REGULATORY EVENTS
19 CHMP Adopts Positive Opinion For Enjaymo
19 Enjaymo Marks Latest Milestone In Sanofi’s Blood Disorder Effort
19 Sanofi’s Sutimlimab Bounces Back With Resubmission
19 Seventeen And Counting – Bumper Crop Of New EU Filings
21 PROBABILITY OF SUCCESS
22 CLINICAL TRIAL LANDSCAPE
23 Sponsors by status
23 Sponsors by phase
LIST OF FIGURES
13 Figure 1: Overview of pipeline drugs for cold agglutinin disease in the US
13 Figure 2: Pipeline drugs for cold agglutinin disease, by company
13 Figure 3: Pipeline drugs for cold agglutinin disease, by drug type
14 Figure 4: Pipeline drugs for cold agglutinin disease, by classification
18 Figure 5: Sutimlimab for Autoimmune Hemolytic Anemia (June 11, 2021): Phase III – Cadenza (Cold Agglutinin Disease)
21 Figure 6: Probability of success in the marrow or peripheral blood stimulator pipeline
22 Figure 7: Clinical trials in cold agglutinin disease
22 Figure 8: Top 10 drugs for clinical trials in cold agglutinin disease
23 Figure 9: Top 10 companies for clinical trials in cold agglutinin disease
23 Figure 10: Cold agglutinin disease trials status
24 Figure 11: Cold agglutinin disease trials sponsors, by phase
LIST OF TABLES
12 Table 1: Marketed drugs for cold agglutinin disease
15 Table 2: Pipeline drugs for cold agglutinin disease in the US
17 Table 3: Sutimlimab for Autoimmune Hemolytic Anemia (June 11, 2021)
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