Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
C1 esterase inhibitor liquid · 1 trial · 1 indication
The angioedema attacks were recorded in the electronic patient diary. The investigator completed a separate angioedema eCRF for each attack based on the review of the patients diary. Time-normalized number of angioedema attacks was expressed as the number of attacks per month (ie, 30.4 days) of exposure. NNA=30.4 \* (number of attacks during treatment period) / (days of treatment period).
| Arm | Type | Description |
|---|---|---|
| Experimental/Placebo | EXPERIMENTAL | Subjects will be randomized to receive C1 Esterase Inhibitor in the 1st Treatment period and then switch to Placebo in the 2nd treatment period. |
| Placebo/Experimental | EXPERIMENTAL | Subjects will be randomized to receive a placebo treatment in the 1st Treatment period and then switch to receive C1 Esterase Inhibitor in the 2nd treatment period. |
| Experimental/ Experimental | EXPERIMENTAL | Subjects will be randomized and receive C1 Esterase Inhibitor in both 1st as well as the 2nd treatment period |
| Name | Type | Description |
|---|---|---|
| C1 esterase inhibitor [human] liquid | DRUG | C1 Esterase Inhibitor \[Human\] Liquid administered Subcutaneously as specified on specified days |
| Placebo | DRUG | Placebo |
The maximum duration of participation is approximately 9 months. Patients will complete a screening period of up to 21 days. Following screening, eligible patients will be randomly assigned to 1 of 3 treatment sequences. Each patient will undergo 2 14-week treatment periods for a total of 28 weeks (...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Intellia Therapeutics, Inc. | NTLA | 3 | PHASE3 | NTLA-2002, Normal Saline Administration |
| BioCryst Pharmaceuticals, Inc. | BCRX | 2 | PHASE3 | Berotralstat |
| Ionis Pharmaceuticals, Inc. | IONS | 1 | PHASE3 | Donidalorsen |
| Pharvaris N.V. | PHVS | 1 | PHASE2 | deucrictibant |
| BioMarin Pharmaceutical Inc. | BMRN | 1 | PHASE1 | Dose 1 of BMN 331 |
| Astria Therapeutics, Inc. | ATXS | 1 | PHASE2 | STAR-0215 |
C1 esterase inhibitor liquid is used for the prevention of angioedema attacks in adolescents and adults with Hereditary Angioedema (HAE). It is an investigational therapy being developed by Takeda Pharmaceutical Company Limited (TAK) and is currently in Phase 3 clinical development.
C1 esterase inhibitor liquid targets the C1 esterase inhibitor protein, which plays a role in regulating the complement system and preventing excessive activation that leads to angioedema attacks in patients with Hereditary Angioedema (HAE).
C1 esterase inhibitor liquid is being developed by Takeda Pharmaceutical Company Limited, a global biopharmaceutical company traded on the New York Stock Exchange under the ticker symbol TAK.
C1 esterase inhibitor liquid is in Phase 3 clinical development. It is an investigational drug and has not been approved by regulatory authorities. A Phase 3 trial has been completed to evaluate its efficacy and safety for preventing angioedema attacks in Hereditary Angioedema (HAE).
C1 esterase inhibitor liquid has one completed Phase 3 clinical trial, NCT02584959, which evaluated its efficacy and safety for the prevention of angioedema attacks in adolescents and adults with Hereditary Angioedema (HAE). The trial enrolled 75 participants across the United States, Canada, Germany, Hungary, Israel, Romania, and Spain.
C1 esterase inhibitor liquid is a specific formulation of the C1 esterase inhibitor protein. It is distinct from other marketed C1 esterase inhibitor products, such as plasma-derived or recombinant versions, due to its liquid formulation and subcutaneous route of administration, which is being studied for prevention of Hereditary Angioedema attacks.