Molybdenum Cofactor Deficiency Type

Top drugs studied for this disease

All Approvals Safety alerts Label changes Trial results Research Guidance Withdrawals

CLINICAL_TRIALS trial_result · 2023-10-17
Clinical Trial Update: Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A [Phase 2, Phase 3]
A Phase 2/3 study evaluating ORGN001 (formerly ALXN1101) in neonates, infants, and children with Molybdenum Cofactor Deficiency Type A has c…
CLINICAL_TRIALS trial_result · 2023-10-17
Clinical Trial Update: Safety & Efficacy Study of ORGN001 (Formerly ALXN1101) in Pediatric Patients With MoCD Type A Currently Treated With rcPMP [Phase 2]
Phase 2 clinical trial update on ORGN001 (formerly ALXN1101) in pediatric MoCD Type A patients on rcPMP, completed with results posted in 20…
FDA approval · 2022-10-27
FDA Approved: NULIBRY — Supplemental Application (Sentynl Therapeutics, Inc.)
The FDA approved a supplemental application for NULIBRY (fosdenopterin hydrobromide) from Sentynl Therapeutics, Inc.
FDA approval · 2021-02-26
FDA Approved: NULIBRY — Original Application (Sentynl Therapeutics, Inc.)
FDA approved NULIBRY (fosdenopterin hydrobromide) under an original application from Sentynl Therapeutics for the treatment of molybdenum co…
CLINICAL_TRIALS withdrawal · 2011-02-01
Clinical Trial Update: Study of cPMP (Precusor Z) to Treat Molybdenum Cofactor Deficiency (MoCD) Type A [Phase 1, Phase 2]
The Phase 1/2 clinical trial of cPMP (Precursor Z) for Molybdenum Cofactor Deficiency Type A has been withdrawn by the sponsor.
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