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Also known as ORGN001 (formerly ALXN1101)
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 being developed for the treatment of Molybdenum Cofactor Deficiency Type A, a rare inherited metabolic disorder. It is a small molecule therapeutic studied in pediatric patients, including neonates, infants and children. Both of its clinical trials enrolled patients with MoCD Type A.
ORGN001 is being developed by BridgeBio Pharma, Inc., which trades on the Nasdaq under the ticker BBIO. The program was formerly known as ALXN1101 before BridgeBio took it forward.
Yes. ORGN001 is the current name for the compound formerly called ALXN1101. Both names refer to the same small molecule, and the clinical trial records list it as ORGN001 (formerly ALXN1101).
ORGN001 is in Phase 2 development. It is an investigational small molecule and has not been approved by the FDA. Two Phase 2 trials have been conducted, and both are now completed.
ORGN001 has been studied in two completed Phase 2 trials. NCT02629393 evaluated ORGN001 in neonates, infants and children with MoCD Type A across the United States, Israel, Norway, Spain, Turkey and the United Kingdom. NCT02047461 studied pediatric patients with MoCD Type A who were already receiving rcPMP treatment.
The two completed Phase 2 trials of ORGN001 enrolled a combined total of 13 patients. NCT02629393 enrolled 5 participants, and NCT02047461 enrolled 8 participants. Both studies were uncontrolled and enrolled pediatric patients with Molybdenum Cofactor Deficiency Type A.