Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Recombinant human C1 inhibitor · 4 trials · 3 indications
The time to beginning of relief of symptoms has been assessed by using a patient-reported visual analogue scale ("VAS") ranging from 0 mm (no symptoms at all) to 100 mm (extremely disabling). Time to beginning of relief of symptoms at the location that showed first "VAS" score decrease of at least 20 mm from baseline score (t= 0 min) to the next assessment time-point). Assessment time-points were taken on pre-scheduled time-points after drug administration: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours, 48 hours. Time to beginning of relief has been calculated as median time, by using the exact time-points on which each assessment was performed.
Average number of HAE attacks normalized to a 28 day period
Prior to the treatment period, patients enrolled in the study, were asked about the amount of "HAE" attacks in the past 2 years, (calculated to attacks/week), this number is defined as "Historical". During the treatment period, patients received a dose of 50 IU/kg of "rhC1INH" administered by slow "IV" injection over 4 to 5 minutes, once a week during an eight week period. The amount of attacks during this period is defined as "Prophylaxis" (calculated to attacks/week).
| Arm | Type | Description |
|---|---|---|
| 100 IU/kg "rhC1INH" | EXPERIMENTAL | 100 IU/kg recombinant human C1 inhibitor |
| Saline | PLACEBO_COMPARATOR | Saline solution |
| rhC1INH twice weekly | EXPERIMENTAL | rhC1INH administered twice weekly |
| rhC1INH once weekly | EXPERIMENTAL | rhC1INH administered once weekly |
| Placebo (Saline) twice weekly | PLACEBO_COMPARATOR | Placebo (Saline) administered twice weekly |
| Recombinant Human C1 Inhibitor | OTHER | Weekly administration of 50 IU/kg Recombinant Human C1 Inhibitor |
| 100 IU/kg rhC1INH | EXPERIMENTAL | 100 IU/kg Recombinant human C1 inhibitor |
| 50 IU/kg rhC1INH | EXPERIMENTAL | 50 IU/kg Recombinant human C1 inhibitor |
| Name | Type | Description |
|---|---|---|
| recombinant human C1 inhibitor | DRUG | IV |
| Placebo | DRUG | IV |
Inclusion Criteria: * Clear clinical and laboratory diagnosis of HAE * Baseline plasma level of functional C1INH of less than 50% of normal * Evidence for exacerbation or development of a severe abdominal, oro-facial/ pharyngeal/ laryngeal, genito-urinary and/or peripheral HAE attack Exclusion Cri...
| 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 |
Recombinant Human C1 Inhibitor is used for the treatment of acute attacks in patients with Hereditary Angioedema, a rare genetic disorder. It is also being studied for prophylaxis of Hereditary Angioedema attacks. The drug is currently in clinical development and is not yet approved by regulatory authorities.
Recombinant Human C1 Inhibitor is being developed by Pharming Group N.V., a biopharmaceutical company listed on the stock exchange under the ticker symbol PHAR. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with Hereditary Angioedema.
Recombinant Human C1 Inhibitor is in Phase 2 and Phase 3 clinical trials. The drug is investigational and has not been approved by the FDA or other regulatory agencies. It is being studied for the treatment of acute attacks and prophylaxis in patients with Hereditary Angioedema.
Recombinant Human C1 Inhibitor has been studied in several completed clinical trials, including NCT00225147 and NCT00262301 for acute attacks in Hereditary Angioedema, NCT00851409 for safety and immunogenicity, and NCT02247739 for prophylaxis. These trials enrolled a total of 184 patients across multiple countries.
Yes, Recombinant Human C1 Inhibitor is also known as rhC1INH, as referenced in clinical trial titles. This abbreviation is used in studies such as NCT00851409, which examined the safety and immunogenicity of repeated rhC1INH administration in patients with Hereditary Angioedema.