Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ORGN001 · 2 trials · 1 indication
Patients with a confirmed diagnosis of MOCD Type A, treated with ORGN001 and still alive at last observation.
Treatment Emergent Serious Adverse Events
| Arm | Type | Description |
|---|---|---|
| ORGN001 (formerly ALXN1101) | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| ORGN001 (formerly ALXN1101) | DRUG | - |
Patients must meet all of the following inclusion criteria to be considered for enrollment in this study: 1. Male or female neonatal patient (1 to 28 days of age \[inclusive\] at the time of ORGN001 administration, with day 1 of age corresponding to the day of birth) or infant (29 days to \<2 years...
ORGN001 is an investigational small molecule being developed for Molybdenum Cofactor Deficiency, Type A, a rare genetic disorder. It is currently in Phase 2 clinical development and has not been approved by the FDA. The drug is intended to address the underlying metabolic defect in patients with this condition.
ORGN001 is being developed by BridgeBio Pharma, Inc., a biopharmaceutical company traded on NASDAQ under the ticker BBIO. The company is conducting clinical trials to evaluate the safety and efficacy of ORGN001 in patients with Molybdenum Cofactor Deficiency, Type A.
ORGN001 is in Phase 2 clinical development. It is an investigational drug and has not been approved by regulatory authorities. Two Phase 2 trials have been completed, with a total of 13 participants enrolled across multiple countries.
ORGN001 has been studied in two completed Phase 2 trials. NCT02047461 enrolled 8 pediatric patients with Molybdenum Cofactor Deficiency Type A, while NCT02629393 enrolled 5 neonates, infants, and children with the same condition. Both trials were uncontrolled and not blinded.
Yes, ORGN001 was formerly known as ALXN1101. The clinical trials NCT02047461 and NCT02629393 both reference the drug as ORGN001, formerly ALXN1101, indicating that the names refer to the same investigational compound.