Synonyms Dean-Barnes syndrome, Deficiency, Ppox, Mixed hepatic porphyria + [48] |
Introduction An autosomal dominant porphyria that is due to a deficiency of protoporphyrinogen oxidase (EC 1.3.3.4) in the LIVER, the seventh enzyme in the 8-enzyme biosynthetic pathway of HEME. Clinical features include both neurological symptoms and cutaneous lesions. Patients excrete increased levels of porphyrin precursors, COPROPORPHYRINS and protoporphyrinogen. |
Target |
Mechanism ALAS1 inhibitors [+1] |
Active Org. |
Originator Org. |
Active Indication |
Inactive Indication |
Drug Highest PhaseApproved |
First Approval Ctry. / Loc. US |
First Approval Date20 Nov 2019 |
Target |
Mechanism MC1R agonists |
Active Org. |
Originator Org. |
Active Indication |
Inactive Indication |
Drug Highest PhaseApproved |
First Approval Ctry. / Loc. EU [+3] |
First Approval Date22 Dec 2014 |
Start Date17 May 2023 |
Sponsor / Collaborator |
Start Date28 Mar 2023 |
Sponsor / Collaborator |
Start Date28 Mar 2023 |
Sponsor / Collaborator |