Lead Product(s) : Pyridoxal Phosphate
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Medicure Receives US FDA Fast Track Designation for MC-1 for PNPO Deficiency
Details : MC-1 (pyridoxal phosphate) is a naturally occuring purinergic receptor antagonist, which is being evaluated for the treatment of Pyridox(am)Ine 5'-Phosphate Oxidase (PNPO) Deficiency.
Product Name : MC-1
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
April 23, 2024
Lead Product(s) : Pyridoxal Phosphate
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Pyridoxal Phosphate
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : FDA provides complete approval to enroll patients in its pivotal clinical trial to evaluate "MC-1" (a naturally occurring metabolite of pyridoxine) for treatment of a rare pediatric disease called pyridox(am)ine 5′-phosphate oxidase deficiency.
Product Name : MC-1
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
November 23, 2023
Lead Product(s) : Pyridoxal Phosphate
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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