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Erythropoietic Protoporphyria

9
Pipeline Programs
2
Companies
8
Clinical Trials
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
1
2
1
4
1
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Peptide
5100%
+ 3 programs with unclassified modality

On Market (1)

Approved therapies currently available

SCENESSEApproved
afamelanotide
Unknown Company
subcutaneous2019

Competitive Landscape

2 companies ranked by most advanced pipeline stage

Clinuvel Pharmaceuticals
Clinuvel PharmaceuticalsAustralia - Melbourne
6 programs
1
1
4
AfamelanotidePhase 3Peptide1 trial
AfamelanotidePhase 3Peptide1 trial
AfamelanotidePhase 3Peptide1 trial
AfamelanotidePhase 3Peptide1 trial
AfamelanotidePhase 2Peptide1 trial
+1 more programs
Active Trials
NCT06388642Completed28Est. Jan 2025
NCT01097044Completed77Est. Apr 2011
NCT04053270Completed100Est. Dec 2009
+3 more trials
Disc Medicine
Disc MedicineMA - Watertown
2 programs
1
1
DISC-1459Phase 2/31 trial
DISC-1459Phase 21 trial
Active Trials
NCT05308472Completed75Est. Aug 2024
NCT05883748Enrolling By Invitation230Est. Jun 2028

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
Clinuvel PharmaceuticalsAfamelanotide
Clinuvel PharmaceuticalsAfamelanotide
Clinuvel PharmaceuticalsAfamelanotide
Clinuvel PharmaceuticalsAfamelanotide
Disc MedicineDISC-1459
Disc MedicineDISC-1459
Clinuvel PharmaceuticalsAfamelanotide
Clinuvel PharmaceuticalsAfamelanotide 16mg implant

Clinical Trials (8)

Total enrollment: 693 patients across 8 trials

NCT01605136Clinuvel PharmaceuticalsAfamelanotide

Phase III Confirmatory Study in Erythropoietic Protoporphyria

Start: May 2012Est. completion: Jul 201393 patients
Phase 3Completed
NCT04578496Clinuvel PharmaceuticalsAfamelanotide

A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)

Start: Jul 2011Est. completion: Feb 201416 patients
Phase 3Completed
NCT00979745Clinuvel PharmaceuticalsAfamelanotide

Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)

Start: Sep 2009Est. completion: May 201174 patients
Phase 3Completed
NCT04053270Clinuvel PharmaceuticalsAfamelanotide

Multicentre Phase III Erythropoietic Protoporphyria Study

Start: May 2007Est. completion: Dec 2009100 patients
Phase 3Completed
NCT05883748Disc MedicineDISC-1459

HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP

Start: Aug 2023Est. completion: Jun 2028230 patients
Phase 2/3Enrolling By Invitation
NCT05308472Disc MedicineDISC-1459

Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP

Start: Oct 2022Est. completion: Aug 202475 patients
Phase 2Completed
NCT01097044Clinuvel PharmaceuticalsAfamelanotide

Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)

Start: Apr 2010Est. completion: Apr 201177 patients
Phase 2Completed
NCT06388642Clinuvel PharmaceuticalsAfamelanotide 16mg implant

Pharmacokinetics of Afamelanotide in Erythropoietic Protoporphyria Patients

Start: Mar 2024Est. completion: Jan 202528 patients
Phase 1/2Completed

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

4 late-stage (Phase 3) programs, potential near-term approvals
Peptide is the dominant modality (100% of programs)
2 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.