Cyclic pyranopterin monophosphate

FDA Approves First Treatment for Molybdenum Cofactor Deficiency Type A

Retrieved on: 
Friday, February 26, 2021
Chemistry, Molybdenum cofactor deficiency, Organophosphates, Physical sciences, Cofactors, Pteridines, Organic compounds, Cyclic pyranopterin monophosphate, Pterin, Molybdenum, CPMP, MOCS1

Patients with Molybdenum Cofactor Deficiency Type A experience severe and rapidly progressive neurologic damage including intractable seizures, feeding difficulties and muscle weakness from the accumulation of toxic sulfite metabolites in the central nervous system.

Key Points: 
  • Patients with Molybdenum Cofactor Deficiency Type A experience severe and rapidly progressive neurologic damage including intractable seizures, feeding difficulties and muscle weakness from the accumulation of toxic sulfite metabolites in the central nervous system.
  • Before today's approval, the only treatment options included supportive care and therapies directed towards the complications arising from the disease.
  • Patients with Molybdenum Cofactor Deficiency Type A cannot produce a substance known as cyclic pyranopterin monophosphate (cPMP).
  • The effectiveness of Nulibry for the treatment of Molybdenum Cofactor Deficiency Type A was demonstrated in 13 treated patients compared to 18 matched, untreated patients.

BridgeBio Pharma’s Origin Biosciences Initiates Rolling Submission of New Drug Application with the U.S. FDA for BBP-870 for the Treatment of MoCD Type A

Retrieved on: 
Tuesday, December 3, 2019
Chemistry, Physical sciences, Organophosphates, Chemical elements, Molybdenum cofactor deficiency, Cofactors, Pteridines, Cyclic pyranopterin monophosphate, CPMP, Pterin, Molybdenum, MOCS1

Currently, there are no approved therapies that alter the course of MoCD Type A, which results in severe and irreversible neurological injury.

Key Points: 
  • Currently, there are no approved therapies that alter the course of MoCD Type A, which results in severe and irreversible neurological injury.
  • As we begin our first rolling new drug application with the FDA, we are taking a significant step forward for patients by targeting MoCD Type A at its source through the provision of the monophosphate cPMP.
  • Together with patients and physicians, the company aims to bring a safe, effective treatment for MoCD Type A to market as quickly as possible.
  • Efficacy and safety of cyclic pyranopterin monophosphate substitution in severe molybdenum cofactor deficiency type A: a prospective cohort study.

BridgeBio Pharma’s Origin Biosciences Presents New Data on the Natural History of Molybdenum Cofactor Deficiency (MoCD) Type A at the Society of the Study of Inborn Errors of Metabolism (SSIEM) Conference

Retrieved on: 
Thursday, September 5, 2019
Chemistry, Enzymes, Physical sciences, Organophosphates, Cofactors, Pteridines, Molybdenum cofactor deficiency, Cyclic pyranopterin monophosphate, Pterin, Molybdenum, Tyrosine hydroxylase, MOCS1

The majority of patients had MoCD Type A (n=37), followed by MoCD Type B (n=16) and Other (n=12).

Key Points: 
  • The majority of patients had MoCD Type A (n=37), followed by MoCD Type B (n=16) and Other (n=12).
  • Nearly all patients (n=57) had first presenting symptoms by Day 28 with median onset for MoCD Type A patients at 2days.
  • 2Mechler K, Mountford WK, Hoffmann GF, Ries M. Ultra-orphan diseases: a quantitative analysis of the natural history of molybdenum cofactor deficiency.
  • Efficacy and safety of cyclic pyranopterin monophosphate substitution in severe molybdenum cofactor deficiency type A: a prospective cohort study.