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 treating acute attacks in patients with hereditary angioedema, a genetic disorder. It has also been studied for prophylaxis of hereditary angioedema attacks. The drug is being developed by Pharming Group N.V. and is currently in Phase 2 clinical development.
Recombinant Human C1 Inhibitor works by replacing the deficient or dysfunctional C1 inhibitor protein in patients with hereditary angioedema. This protein normally regulates the complement system and prevents excessive activation that leads to swelling. By restoring C1 inhibitor activity, the drug helps control acute attacks and may prevent future episodes.
Recombinant Human C1 Inhibitor is developed by Pharming Group N.V., a biopharmaceutical company traded under the ticker PHAR. The company is focused on developing innovative therapies for rare diseases, with this drug being a key asset in its pipeline for hereditary angioedema.
Recombinant Human C1 Inhibitor is currently in Phase 2 clinical development. It has completed three clinical trials, including a Phase 3 study, but is not yet approved by regulatory authorities. The drug remains investigational and is being evaluated for safety and efficacy in treating hereditary angioedema.
Recombinant Human C1 Inhibitor has been studied in three completed trials. NCT00225147 was a Phase 2 trial treating acute attacks in 77 patients. NCT00262301 was a Phase 3 trial with 75 patients. NCT00851409 assessed safety and immunogenicity in 25 patients, and NCT02247739 was a Phase 2 prophylaxis study with 32 patients.
Recombinant Human C1 Inhibitor is also known as Ruconest, a brand name for the drug. It is a recombinant form of the human C1 esterase inhibitor protein. This medication is used to treat acute attacks of hereditary angioedema in patients, and it is being developed by Pharming Group N.V.