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Epidermolysis Bullosa

8
Pipeline Programs
9
Companies
13
Clinical Trials
1 recruiting
0
Approved Products

Pipeline by Development Stage

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

Competitive Landscape

8 companies ranked by most advanced pipeline stage

RHEACELL
RHEACELLGermany - Heidelberg
2 programs
2
allo-APZ2-OTSPhase 31 trial
allo-APZ2-OTSPhase 31 trial
Active Trials
NCT05464381Active Not Recruiting91Est. Jan 2027
NCT05838092Active Not Recruiting9Est. Jul 2026
Chiesi
ChiesiBrazil - Santana de Parnaíba
1 program
1
Oleogel-S10Phase 31 trial
Active Trials
NCT06917690Recruiting6Est. Sep 2029
Amicus Therapeutics
Amicus TherapeuticsPA - Philadelphia
1 program
1
SD-101Phase 34 trials
Active Trials
NCT02670330Terminated152Est. Sep 2018
NCT02384460Completed169Est. Jul 2017
NCT02090283Terminated42Est. Sep 2018
+1 more trials
Vyne Therapeutics
Vyne TherapeuticsIsrael - Ness Ziona
2 programs
2
Serlopitant TabletPhase 21 trial
VPD-737Phase 21 trial
Active Trials
NCT03836001Completed28Est. Jun 2022
NCT02654483Completed14Est. Jul 2018
RegeneRx Biopharmaceuticals
1 program
1
Thymosin Beta 4Phase 21 trial
Active Trials
NCT00311766Terminated30Est. Nov 2012
Castle Creek Biosciences
1 program
1
diacerein 1% ointmentPhase 21 trial
Active Trials
NCT03389308Completed51Est. Apr 2020
Takeda
TakedaTOKYO, Japan
1 program
CX501PHASE_21 trial
Active Trials
NCT00987142Completed12Est. May 2010
Organogenesis
OrganogenesisCANTON, MA
1 program
ApligrafPHASE_41 trial
Active Trials
NCT01619670Terminated3Est. Jan 2015

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
OrganogenesisApligraf
ChiesiOleogel-S10
RHEACELLallo-APZ2-OTS
RHEACELLallo-APZ2-OTS
Amicus TherapeuticsSD-101
Amicus TherapeuticsSD-101
Vyne TherapeuticsSerlopitant Tablet
Castle Creek Biosciencesdiacerein 1% ointment
Vyne TherapeuticsVPD-737
Amicus TherapeuticsSD-101
Amicus TherapeuticsSD-101
TakedaCX501
RegeneRx BiopharmaceuticalsThymosin Beta 4

Clinical Trials (13)

Total enrollment: 655 patients across 13 trials

NCT01619670OrganogenesisApligraf

A Observational Study to Evaluate Apligraf(R) in Nonhealing Wounds of Subjects With Epidermolysis Bullosa

Start: Jun 2012Est. completion: Jan 20153 patients
Phase 4Terminated
NCT06917690ChiesiOleogel-S10

A Study to Learn About the Safety and Efficacy of the Drug Oleogel-S10 in Japanese Patients With Epidermolysis Bullosa

Start: Apr 2025Est. completion: Sep 20296 patients
Phase 3Recruiting
NCT05838092RHEACELLallo-APZ2-OTS

Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III)

Start: Oct 2023Est. completion: Jul 20269 patients
Phase 3Active Not Recruiting
NCT05464381RHEACELLallo-APZ2-OTS

Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III, Cross-over)

Start: Jun 2023Est. completion: Jan 202791 patients
Phase 3Active Not Recruiting
NCT02670330Amicus TherapeuticsSD-101

Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa

Start: Jun 2015Est. completion: Sep 2018152 patients
Phase 3Terminated
NCT02384460Amicus TherapeuticsSD-101

ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa

Start: Mar 2015Est. completion: Jul 2017169 patients
Phase 3Completed
NCT03836001Vyne TherapeuticsSerlopitant Tablet

A Neurokinin-1 Receptor Antagonist for the Treatment of Pruritus in Patients With Epidermolysis Bullosa

Start: Apr 2019Est. completion: Jun 202228 patients
Phase 2Completed
NCT03389308Castle Creek Biosciencesdiacerein 1% ointment

Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex

Start: Dec 2017Est. completion: Apr 202051 patients
Phase 2Completed
NCT02654483Vyne TherapeuticsVPD-737

Neurokinin-1 Receptor Antagonist for the Treatment of Itch in EB Patients

Start: Aug 2016Est. completion: Jul 201814 patients
Phase 2Completed
NCT02090283Amicus TherapeuticsSD-101

Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis Bullosa

Start: Mar 2014Est. completion: Sep 201842 patients
Phase 2Terminated
NCT02014376Amicus TherapeuticsSD-101

Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa

Start: Jan 2014Est. completion: Jun 201448 patients
Phase 2Completed
NCT00987142TakedaCX501

Trial To Assess Efficacy Of A Chimeric Skin In Patients With Epidermolysys Bullosa

Start: Dec 2006Est. completion: May 201012 patients
Phase 2Completed
NCT00311766RegeneRx BiopharmaceuticalsThymosin Beta 4

A Phase 2 Study on Effect of Thymosin Beta 4 on Wound Healing in Patients With Epidermolysis Bullosa

Start: Feb 2006Est. completion: Nov 201230 patients
Phase 2Terminated

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
1 actively recruiting trials targeting 655 patients
9 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.