Approved drugs for Mitochondrial disease
As of December, 2019
| Drug | Type of disease | Country | Company |
|---|---|---|---|
|
MELAS |
Japan |
Taisho Pharmaceutical |
|
|
LHON |
EU (under exceptional circumstances) |
Santhera Pharmaceuticals |
Clinical research, clinical study, and clinical trial in Japan
As of December, 2019
| Compound | Type of disease | Status | Sponsor | |
|---|---|---|---|---|
|
Leigh Syndrome |
Clinical trial |
Active, |
Dainippon Sumitomo Pharma Co., Ltd |
|
|
MELAS |
Clinical study |
Completed |
Kawasaki Medical School |
|
|
MERRF |
Clinical study |
Completed |
Kawasaki Medical School |
|
|
Leigh Syndrome |
Clinical study |
Terminated |
Kurume University School of Medicine |
|
|
MELAS/MELA |
Clinical study |
Terminated |
Kurume University School of Medicine |
|
| Mitochondrial Disorder | Clinical study | Inquiry required | Osaka University | |
|
Mitochondrial Disease, mainly to cranial nerve symptoms |
Investigator-initiated clinical trial (DB) Phase 3 |
Active, not recruiting |
Saitama Medical University Hospital |
|
|
LHON |
Clinical study Phase 1/2 |
Active, not recruiting |
Hyogo College of Medicine |
|
|
LHON |
Clinical study Phase 1 |
Active, not recruiting |
Kobe University |
|
Clinical research, clinical study, and clinical trial around the world
As of December, 2019
| Compound | Type of disease | Status | Sponsor | Country | |
|---|---|---|---|---|---|
|
Leigh Syndrome |
Clinical trial (DB) |
Active, |
PTC Therapeutics (BioElectron/Edison Pharmaceutcals) |
USA | |
| Pearson Syndrome |
Clinical trial |
Terminated |
PTC Therapeutics (BioElectron/Edison Pharmaceutcals) |
USA | |
| Mitochondrial Disease |
Clinical trial |
Active, not recruiting |
PTC Therapeutics (BioElectron/Edison Pharmaceutcals) |
USA | |
| Mitochondrial Disorders, etc |
Clinical study (DB) |
Completed |
National Human Genome Research Institute (NHGRI) |
USA | |
| Mitochondrial Myopathy |
Clinical trial (DB) |
Completed |
Reata Pharmaceuticals Inc |
USA |
|
| Mitochondorial Disease |
Clinical trial |
Terminated |
Horizon Pharma lnc （Raptor Pharmaceuticals Inc） |
USA |
|
| Mitochondrial Myopathy | Clinical trial (DB) Phase 3 |
Active, not recruiting |
Stealth BioTherapeutics Inc |
USA |
|
| LHON | Clinical trial (DB) Phase 2 |
Active, not recruiting |
Stealth BioTherapeutics Inc |
USA |
|
| Mitochondrial Disease | Clinical trial (DB) Phase 2 |
Completed |
Stealth BioTherapeutics Inc |
USA |
|
| MELAS, MIDD, etc. |
Clinical trial (DB) |
Completed |
Khondrion BV |
Netherland |
|
| MELAS, MIDD ,etc |
Clinical trial (DB) |
Recruiting |
Khondrion BV |
Netherlands |
|
| Leigh Syndrome |
Clinical trial |
Not yet recruiting |
Aadi, LLC |
USA |
|
| GS010 (rAAV2/2-ND4) (NCT02064569) (NCT02652767) (NCT02652780) (NCT03293524) (NCT03406104) |
LHON |
Clinical trial (DB) |
Enrolling by invitation |
GenSight Biologics |
France |
| GS010 (rAAV2/2-ND4) (NCT03672968) |
LHON |
Expanded Access (Compassionate use) |
Available |
GenSight Biologics |
USA |
|
LHON |
Clinical study Phase 1 |
Recruiting | University of Miami |
USA |
|
|
LHON |
Clinical study | Recruiting |
Huazhong University of Science and Technology |
China |
|
|
Barth Syndrome |
Clinical study | Recruiting |
University Hospitals Bristol NHS Foundation Trust |
UK |
|
|
Mitochondorial Disease (Heart Rate Variability) |
Clinical study Phase 2 |
Completed |
Woodinville Psychiatric Associates |
USA |
|
| Mitochondrial Disease |
Clinical study |
Active, |
Tawam Hospital | UAE | |
|
MELAS |
Clinical study Phase 1 |
Not yet recruiting |
Baylor College of Medicine |
USA |
|
|
Multiple Mitochondrial Dysfunctions Syndrome (Carbohydrate Metabolism) |
Clinical study (DB) | Completed |
The University of Texas |
USA |
|
|
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, |
Expanded Access (Compassionate use) |
Available |
University of Pittsburgh |
USA |
|
|
LHON |
Clinical trial |
Active, |
Santhera Pharmaceuticals |
USA |
|
|
MELAS |
Clinical trial |
Completed |
Yungjin Pharm |
Korea |
|
|
Mitochondrial Disease |
Clinical trial (DB) |
Recruiting |
NeuroVive Pharmaceutical AB |
UK |
|
|
Mitochondrial Disease, |
Clinical study |
Recruiting |
Cambridge University |
UK |
|
|
Mitochondrial Myopathy |
Clinical study |
Completed |
Helsinki University |
Finland |
|
|
Mitochondrial Myopathy, etc |
Clinical study (DB) |
Completed |
Rigshospitalet |
Denmark |
|
| LHON, etc. |
Clinical trial |
Recruiting |
MD Stem Cells |
USA |
|
| MNGIE |
Clinical study |
Recruiting |
Columbia University |
USA |
|
| Non-inherited Mitochondrial Disease, Pearson Syndrome |
Clinical study |
Enrolling by invitation |
Sheba Medical Center |
Israel |
|
| Hemodialysis-Induced Symptom Mitochondrial Diseases |
Clinical study (DB) |
Completed |
Vanderbilt University |
USA |
|
| Propionic Acidemia, Carbamoyl-Phosphate Synthase I Deficiency Disease, etc |
Clinical study (DB) |
Active, not recruiting |
Children's Research Institute |
USA |
|
| Pyruvate Dehydrogenase Complex Deficiency |
Clinical study (DB) |
Recruiting |
University of Florida |
USA |
|
| Pyruvate Dehydrogenase Complex Deficiency |
Clinical trial |
Recruiting |
Fondazione Telethon |
Italia |
|
| Mitochondrial Disease |
Clinical study (DB) |
Recruiting |
University of Kansas |
USA |
|
| Thymidine (NCT03639701) |
Mitochondrial DNA Depletion Syndrome, etc | Clinical atudy Phase 1/2 |
Enrolling by invitation | Columbia University | USA |
| Thymidine Phosphorylase (EE-TP) (NCT03866954) | MNGIE | Clinical atudy Phase 2 |
Not yet recruiting |
St George's, University of London | UK |
| REN00 (NCT03862846) |
Mitochondrial Myopathy | Clinical trial Phase 1 |
Recruiting | Reneo Pharma Ltd | UK |
KOINOBORI Associate Inc.