Myasthenia Gravis (MG) — RepurpOS

Disease overview

Alterations

174

Molecular (A)

100

Clinical (B–E)

Direct drugs

Via biomarker

204

Natural agents

Natural products

Merged ranked

281

With trial/literature evidence

Sources: Open Targets, HPO, ClinicalTrials.gov, DGIdb, ChEMBL, PubMed Literature, Seeds, Examine.com · Pipeline phase 7 · Updated 2026-07-06

Remission & chronicity

Spontaneous remission

Spontaneous complete stable remission (CSR) in 10-20% of patients over 10 years; generalised MG rarely resolves without treatment; ocular MG may remain limited in 50-60%

Best-intervention remission

Thymectomy in non-thymomatous MG at age <65: 50% more likely to achieve CSR at 3 years vs medical therapy (MGTX trial); eculizumab (ravulizumab): 40% reduction in exacerbations in AChR+ MG; efgartigimod alfa (Vyvgart): 67.7% MG-ADL responders (ADAPT trial); zilucoplan: 53% MG-ADL response (RAISE trial)

Drug-free remission

—

Relapse after remission

—

Chronicity

—

Gap size

Large — new complement inhibitors and FcRn antagonists represent genuine advances but cost and access are barriers; thymectomy underused in appropriate candidates

Primary barrier

access

Barrier detail: Thymectomy (MGTX trial proven benefit) is delayed or not offered in many patients with non-thymomatous MG; eculizumab ($700K/year) dramatically limits access; efgartigimod and zilucoplan more accessible but still $200K+/year; corticosteroids cause significant morbidity in MG patients managed long-term

Last SoC change: 2023 — rozanolixizumab (Rystiggo, FcRn inhibitor) approved; zilucoplan (Zilbrysq, complement C5 inhibitor) 2023; efgartigimod alfa (Vyvgart) 2021; eculizumab 2017; thymectomy MGTX trial 2016; pyridostigmine/corticosteroids standard since 1950s-1960s

Remission data layers: disease_db_100 · source-locked manual data · confidence: high

Identifiers: MONDO: MONDO:0009688

Right to Try Relevance Critical

Eligibility: This disease meets the federal and state right-to-try legal standard (21 U.S.C. § 360bbb-0a and Montana SB 535): it lacks disease-modifying therapy with indication-specific FDA approval. Patients with Myasthenia Gravis may be eligible to access investigational therapies outside of clinical trials under right-to-try frameworks.

Investigational Agents

Phase 3: 24, Phase 2: 12, Phase 1: 4

Most Advanced Phase

Phase 3

Highest dev. stage

Gene Targets

174

Actionable

Pipeline Compounds (Top 6)

Azathioprine, Batoclimab, Bq 788, Cemdisiran, Cladribine, Eculizumab

Legal Standard: Right-to-try requires the disease lacks "adequate approved therapy for that specific disease" (not symptom-management drugs). This distinction means a disease with antidepressants available still qualifies if no FDA approval exists for THAT disease indication.

View Right-to-Try Compendium

Alterations

Showing 80 of 174 · Types A (molecular) through E (functional)

Name	Type	Subtype	Direction	Frequency	Evidence	Sources	Links
Abnormal thymus morphology	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormality of the endocrine system	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormality of the immune system	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Acrocyanosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Adult onset	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anti-DCC netrin 1 receptor antibody positivity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anti-Kv1.4 antibody	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anti-Lrp4 antibody positivity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anti-muscle-specific tyrosine kinase antibody	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anti-neuromuscular Junction acetylcholine receptor antibody positivity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anti-ryanodine receptor antibody	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anti-titin antibody positivity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Autoimmunity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Bulbar palsy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Childhood onset	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Difficulty climbing stairs	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Diplopia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dysarthria	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dysphagia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dysphonia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dyspnea	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
EMG: decremental response of compound muscle action potential to repetitive nerve stimulation	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Facial palsy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Fatigable weakness	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Fatigable weakness of skeletal muscles	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Fatigue	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Glycosuria	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hashimoto thyroiditis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hearing impairment	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hemolytic anemia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hepatitis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hyperacusis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hypernasal speech	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hyperthyroidism	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Impaired mastication	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Limb muscle weakness	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Mildly elevated creatine kinase	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO LOINC
Muscle weakness	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Myositis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Neoplasm	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Ophthalmoparesis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Paresthesia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Primary adrenal insufficiency	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Proximal amyotrophy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Proximal muscle weakness	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Psychosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Ptosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Pure red cell aplasia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Raynaud phenomenon	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Respiratory failure	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Rheumatoid arthritis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Seizure	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Single fiber EMG abnormality	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Skeletal muscle atrophy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Systemic lupus erythematosus	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Thymoma	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Thymus hyperplasia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Tongue atrophy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Transient	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Type 2 muscle fiber atrophy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Weakness of facial musculature	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
ACHE acetylcholinesterase (Yt blood group)	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
AFG2A AAA ATPase AFG2A	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
AGBL1 AGBL carboxypeptidase 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ALK ALK receptor tyrosine kinase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ALS Specific Quality of Life-Revised	Scales & PROs	Clinical scale	—	—	Emerging	ClinicalTrials.gov	—
Accessibility Evaluation	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
BTK Bruton tyrosine kinase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
C5 complement C5	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CCDC169 coiled-coil domain containing 169	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CD19 CD19 molecule	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CDH10 cadherin 10	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CDKN3 cyclin dependent kinase inhibitor 3	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CFB complement factor B	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CREB5 cAMP responsive element binding protein 5	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CTLA4 cytotoxic T-lymphocyte associated protein 4	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CXCL13 C-X-C motif chemokine ligand 13	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Chalder Fatigue Scale	Scales & PROs	Clinical scale	—	—	Emerging	ClinicalTrials.gov	—
DHODH dihydroorotate dehydrogenase (quinone)	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
DOK6 docking protein 6	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets

FDA-Approved Therapies

Phase 4 agents with regulatory approval for this indication

Drug Name	Mechanism	Type
ABATACEPT		Gene-targeted
ACALABRUTINIB	inhibitor; inhibitor	Gene-targeted
ACALABRUTINIB MALEATE	inhibitor	Gene-targeted
ACETAZOLAMIDE	None	Gene-targeted
AFATINIB		Gene-targeted
ALECTINIB	inhibitor; inhibitor	Gene-targeted
ALECTINIB HYDROCHLORIDE	inhibitor	Gene-targeted
AMBENONIUM		Gene-targeted
AMBENONIUM CHLORIDE	inhibitor	Gene-targeted
AMPHETAMINE	None	Gene-targeted
ANTI-THYMOCYTE GLOBULIN		Gene-targeted
APIGENIN	inhibitor	Gene-targeted
ATEZOLIZUMAB		Gene-targeted
AVACINCAPTAD PEGOL	inhibitor	Gene-targeted
AXICABTAGENE CILOLEUCEL		Gene-targeted
BENZGALANTAMINE GLUCONATE	inhibitor	Gene-targeted
BETAMETHASONE		Gene-targeted
BEVACIZUMAB		Gene-targeted
BICALUTAMIDE	None	Gene-targeted
BLEOMYCIN		Gene-targeted
BLINATUMOMAB	antibody	Gene-targeted
BRENTUXIMAB VEDOTIN		Gene-targeted
BREXUCABTAGENE AUTOLEUCEL	binder	Gene-targeted
BRIGATINIB	inhibitor; inhibitor	Gene-targeted
CARBACHOL	None	Gene-targeted
CARBAMAZEPINE		Gene-targeted
CEMIPLIMAB		Gene-targeted
CERITINIB	inhibitor; inhibitor	Gene-targeted
CIPROFLOXACIN	None	Gene-targeted
CISPLATIN		Gene-targeted
CLOMIPRAMINE	None	Gene-targeted
CRIZOTINIB	inhibitor; inhibitor	Gene-targeted
CROVALIMAB	inhibitor	Gene-targeted
CYCLOPHOSPHAMIDE ANHYDROUS		Gene-targeted
CYCLOSPORINE		Gene-targeted
CYTARABINE		Gene-targeted
DABRAFENIB		Gene-targeted
DASATINIB ANHYDROUS		Gene-targeted
DEHYDRATED ALCOHOL		Gene-targeted
DEMECARIUM BROMIDE		Gene-targeted

Therapeutics

Direct disease associations, biomarker-linked drugs, OSMF narrative-review natural agents, and merged ranking. Only agents with registry trials or published clinical literature are shown by default.

Show agents without direct trial evidence

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
TACROLIMUS ANHYDROUS	Small molecule	Approved	Strong	105	6 trials View evidence Registry trials NCT04768465 — Tacrolimus Combined With Low-dose Prednisone for Treatment of Myasthenia Gravis (UNKNOWN) NCT04182984 — TheRapeutic Effect of Different immunosuppressAnts on Non-Thymoma Ocular Myasthenia Gravis: a Real-world Study (RECRUITING) NCT00309088 — FK506 Phase 3 Study: a Study for Steroid Non-resistant Myasthenia Gravis (MG) Patients (COMPLETED) PHASE3 NCT00309101 — An Open Study for Steroid Resistant, Non-Thymectomized MG Patients (COMPLETED) PHASE3 NCT01325571 — A Study to Compare the Efficacy and Safety of Tacrolimus Capsules in Patient With Myasthenia Gravis (COMPLETED) PHASE3 NCT04101578 — Clinical Prognosis and Progression of Myasthenia Gravis Patients (UNKNOWN) CT.gov search · Cochrane · PubMed	Open Targets, ChEMBL	ChEMBL Open Targets
ABATACEPT Repurposing	Small molecule	Approved	Strong	95	1 trials 1 Cochrane View evidence Registry trials NCT03059888 — Trial of Orencia in Patients With Myasthenia Gravis (TERMINATED) EARLY_PHASE1 CT.gov search · Cochrane · PubMed	DGIdb	—
ACALABRUTINIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ACALABRUTINIB MALEATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ACETAZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
AFATINIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ALECTINIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ALECTINIB HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
AMBENONIUM Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AMBENONIUM CHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
AMPHETAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ANTI-THYMOCYTE GLOBULIN Repurposing	Small molecule	Approved	Strong	95	1 trials View evidence Registry trials NCT00716066 — Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases (ACTIVE_NOT_RECRUITING) PHASE2 CT.gov search · Cochrane · PubMed	DGIdb	—
APIGENIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ATEZOLIZUMAB Repurposing	Small molecule	Approved	Strong	95	23 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
AVACINCAPTAD PEGOL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AXICABTAGENE CILOLEUCEL Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
BENZGALANTAMINE GLUCONATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BEVACIZUMAB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BICALUTAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
BLEOMYCIN Repurposing	Small molecule	Approved	Strong	95	2 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
BLINATUMOMAB Repurposing	Small molecule	Approved	Strong	95	2 trials View evidence Registry trials NCT06684184 — Blinatumomab in Refractory Myasthenia Gravis (BLINA-MG) (NOT_YET_RECRUITING) PHASE2, PHASE3 NCT06836973 — Blinatumomab for Treatment of Refractory Myasthenia Gravis (NOT_YET_RECRUITING) PHASE2, PHASE3 CT.gov search · Cochrane · PubMed	DGIdb	—
BRENTUXIMAB VEDOTIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BREXUCABTAGENE AUTOLEUCEL Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
BRIGATINIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CARBACHOL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CARBAMAZEPINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CEMIPLIMAB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CERITINIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CIPROFLOXACIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CISPLATIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CLOMIPRAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CRIZOTINIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CROVALIMAB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
CYCLOPHOSPHAMIDE ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CYCLOSPORINE Repurposing	Small molecule	Approved	Strong	95	1 trials 1 Cochrane 2 Meta-analysis 14 Trial pubs 3 RCT View evidence Registry trials NCT04101578 — Clinical Prognosis and Progression of Myasthenia Gravis Patients (UNKNOWN) Published literature Cochrane review Quantitative evaluation of drug efficacy in the treatment of myasthenia gravis (Expert opinion on investigational drugs, 2021) Meta-analysis Immunosuppressive and monoclonal antibody treatment for myasthenia gravis: A network meta-analysis (CNS neuroscience & therapeutics, 2019) Systematic review Clinical characteristics and treatment outcomes in thymoma- related aplastic anemia: a case report and literature review (Journal of cardiothoracic surgery, 2026) Clinical trial publication Pharmacological Management of Myasthenia Gravis: A Century of Expert Opinions in Cecil Textbook of Medicine (American journal of therapeutics, 2021) Clinical trial publication Two-year treatment with cyclosporine microemulsion for responder myasthenia gravis patients (European neurology, 2010) Clinical trial publication Cyclosporine A: good response for patients affected by autoimmune disorders and HCV infection? (European review for medical and pharmacological sciences, 2009) Clinical trial publication Response to therapy in myasthenia gravis with anti-MuSK antibodies (Annals of the New York Academy of Sciences, 2008) Clinical trial publication P-glycoprotein function in peripheral blood mononuclear cells of myasthenia gravis patients treated with tacrolimus (Biological & pharmaceutical bulletin, 2007) Randomized controlled trial A clinical therapeutic trial of cyclosporine in myasthenia gravis (Annals of the New York Academy of Sciences, 1993) Randomized controlled trial Preliminary results of a double-blind, randomized, placebo-controlled trial of cyclosporine in myasthenia gravis (The New England journal of medicine, 1987) CT.gov search · Cochrane · PubMed	DGIdb	—
CYTARABINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DABRAFENIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DASATINIB ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DEHYDRATED ALCOHOL Repurposing	Small molecule	Approved	Strong	95	1 trials View evidence Registry trials NCT02761707 — Biomarkers in Neural Disorders (COMPLETED) CT.gov search · Cochrane · PubMed	DGIdb	—
DEMECARIUM BROMIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DEXAMETHASONE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DIAZEPAM Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DICHLORPHENAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DIETHYLSTILBESTROL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DISTIGMINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
DONEPEZIL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DONEPEZIL HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
DOXORUBICIN HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DURVALUMAB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
AZATHIOPRINE	Small molecule	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
BATOCLIMAB	Biologic	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
BQ 788	Small molecule	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
CEMDISIRAN	Small molecule	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
CLADRIBINE	Small molecule	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
ECULIZUMAB	Biologic	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
GEFURULIMAB	Biologic	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
HUMAN IMMUNOGLOBULIN G	Small molecule	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
INEBILIZUMAB	Biologic	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
IPTACOPAN	Small molecule	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
LEFLUNOMIDE	Small molecule	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
MYCOPHENOLATE MOFETIL	Small molecule	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
POZELIMAB	Biologic	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
PREDNISONE	Small molecule	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
RAVULIZUMAB	Biologic	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
REMIBRUTINIB	Small molecule	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
RITUXIMAB	Biologic	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
SATRALIZUMAB	Biologic	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
SODIUM CHLORIDE	Small molecule	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
TACROLIMUS	Small molecule	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
TACROLIMUS ANHYDROUS	Small molecule	Phase III	Moderate	80	6 trials View evidence Registry trials NCT04768465 — Tacrolimus Combined With Low-dose Prednisone for Treatment of Myasthenia Gravis (UNKNOWN) NCT04182984 — TheRapeutic Effect of Different immunosuppressAnts on Non-Thymoma Ocular Myasthenia Gravis: a Real-world Study (RECRUITING) NCT00309088 — FK506 Phase 3 Study: a Study for Steroid Non-resistant Myasthenia Gravis (MG) Patients (COMPLETED) PHASE3 NCT00309101 — An Open Study for Steroid Resistant, Non-Thymectomized MG Patients (COMPLETED) PHASE3 NCT01325571 — A Study to Compare the Efficacy and Safety of Tacrolimus Capsules in Patient With Myasthenia Gravis (COMPLETED) PHASE3 NCT04101578 — Clinical Prognosis and Progression of Myasthenia Gravis Patients (UNKNOWN) CT.gov search · Cochrane · PubMed	Open Targets	ChEMBL Open Targets
TELITACICEPT	Biologic	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
TOLEBRUTINIB	Small molecule	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
ZILUCOPLAN	Biologic	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
ALBUTEROL	Small molecule	Phase II	Emerging	60	—	Open Targets	ChEMBL Open Targets
BELIMUMAB	Biologic	Phase II	Emerging	60	—	Open Targets	ChEMBL Open Targets
BLINATUMOMAB	Biologic	Phase II	Emerging	60	—	Open Targets	ChEMBL Open Targets
BORTEZOMIB	Small molecule	Phase II	Emerging	60	—	Open Targets	ChEMBL Open Targets
ISCALIMAB	Biologic	Phase II	Emerging	60	—	Open Targets	ChEMBL Open Targets
METHOTREXATE	Small molecule	Phase II	Emerging	60	—	Open Targets	ChEMBL Open Targets
MEZAGITAMAB	Biologic	Phase II	Emerging	60	—	Open Targets	ChEMBL Open Targets
ONDANSETRON	Small molecule	Phase II	Emerging	60	—	Open Targets	ChEMBL Open Targets
ORILANOLIMAB	Biologic	Phase II	Emerging	60	—	Open Targets	ChEMBL Open Targets
TIRASEMTIV	Small molecule	Phase II	Emerging	60	—	Open Targets	ChEMBL Open Targets
TOCILIZUMAB	Biologic	Phase II	Emerging	60	—	Open Targets	ChEMBL Open Targets
VEMIRCOPAN	Small molecule	Phase II	Emerging	60	—	Open Targets	ChEMBL Open Targets
ABATACEPT	Biologic	Phase I	Emerging	35	—	Open Targets	ChEMBL Open Targets
ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE)	Small molecule	Phase I	Emerging	35	—	Open Targets	ChEMBL Open Targets
CIZUTAMIG	Biologic	Phase I	Emerging	35	—	Open Targets	ChEMBL Open Targets
CYCLOPHOSPHAMIDE	Small molecule	Phase I	Emerging	35	—	Open Targets	ChEMBL Open Targets

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
ABATACEPT Repurposing via CTLA4	Small molecule	Approved	Strong	95	1 trials 1 Cochrane View evidence Registry trials NCT03059888 — Trial of Orencia in Patients With Myasthenia Gravis (TERMINATED) EARLY_PHASE1 CT.gov search · Cochrane · PubMed	DGIdb	—
ACALABRUTINIB Repurposing via BTK	Small molecule	Approved	Strong	95	—	DGIdb	—
ACALABRUTINIB MALEATE Repurposing via BTK	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ACETAZOLAMIDE Repurposing via ACHE	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
AFATINIB Repurposing via ALK	Small molecule	Approved	Strong	95	—	DGIdb	—
ALECTINIB Repurposing via ALK	Small molecule	Approved	Strong	95	—	DGIdb	—
ALECTINIB HYDROCHLORIDE Repurposing via ALK	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
AMBENONIUM Repurposing via ACHE	Small molecule	Approved	Strong	95	—	DGIdb	—
AMBENONIUM CHLORIDE Repurposing via ACHE	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
AMPHETAMINE Repurposing via ACHE	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ANTI-THYMOCYTE GLOBULIN Repurposing via CTLA4	Small molecule	Approved	Strong	95	1 trials View evidence Registry trials NCT00716066 — Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases (ACTIVE_NOT_RECRUITING) PHASE2 CT.gov search · Cochrane · PubMed	DGIdb	—
APIGENIN Repurposing via ACHE	Small molecule	Approved	Strong	95	—	DGIdb	—
ATEZOLIZUMAB Repurposing via CTLA4	Small molecule	Approved	Strong	95	23 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
AVACINCAPTAD PEGOL Repurposing via C5	Small molecule	Approved	Strong	95	—	DGIdb	—
AXICABTAGENE CILOLEUCEL Repurposing via CD19	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
BENZGALANTAMINE GLUCONATE Repurposing via ACHE	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE Repurposing via ACHE	Small molecule	Approved	Strong	95	—	DGIdb	—
BEVACIZUMAB Repurposing via ALK	Small molecule	Approved	Strong	95	—	DGIdb	—
BICALUTAMIDE Repurposing via ACHE	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
BLEOMYCIN Repurposing via CTLA4	Small molecule	Approved	Strong	95	2 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
BLINATUMOMAB Repurposing via CD19	Small molecule	Approved	Strong	95	2 trials View evidence Registry trials NCT06684184 — Blinatumomab in Refractory Myasthenia Gravis (BLINA-MG) (NOT_YET_RECRUITING) PHASE2, PHASE3 NCT06836973 — Blinatumomab for Treatment of Refractory Myasthenia Gravis (NOT_YET_RECRUITING) PHASE2, PHASE3 CT.gov search · Cochrane · PubMed	DGIdb	—
BRENTUXIMAB VEDOTIN Repurposing via ALK	Small molecule	Approved	Strong	95	—	DGIdb	—
BREXUCABTAGENE AUTOLEUCEL Repurposing via CD19	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
BRIGATINIB Repurposing via ALK	Small molecule	Approved	Strong	95	—	DGIdb	—
CARBACHOL Repurposing via ACHE	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CARBAMAZEPINE Repurposing via ALK	Small molecule	Approved	Strong	95	—	DGIdb	—
CEMIPLIMAB Repurposing via ALK	Small molecule	Approved	Strong	95	—	DGIdb	—
CERITINIB Repurposing via ALK	Small molecule	Approved	Strong	95	—	DGIdb	—
CIPROFLOXACIN Repurposing via ACHE	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CISPLATIN Repurposing via ALK	Small molecule	Approved	Strong	95	—	DGIdb	—
CLOMIPRAMINE Repurposing via ACHE	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CRIZOTINIB Repurposing via ALK	Small molecule	Approved	Strong	95	—	DGIdb	—
CROVALIMAB Repurposing via C5	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
CYCLOPHOSPHAMIDE ANHYDROUS Repurposing via ALK	Small molecule	Approved	Strong	95	—	DGIdb	—
CYCLOSPORINE Repurposing via CTLA4	Small molecule	Approved	Strong	95	1 trials 1 Cochrane 2 Meta-analysis 14 Trial pubs 3 RCT View evidence Registry trials NCT04101578 — Clinical Prognosis and Progression of Myasthenia Gravis Patients (UNKNOWN) Published literature Cochrane review Quantitative evaluation of drug efficacy in the treatment of myasthenia gravis (Expert opinion on investigational drugs, 2021) Meta-analysis Immunosuppressive and monoclonal antibody treatment for myasthenia gravis: A network meta-analysis (CNS neuroscience & therapeutics, 2019) Systematic review Clinical characteristics and treatment outcomes in thymoma- related aplastic anemia: a case report and literature review (Journal of cardiothoracic surgery, 2026) Clinical trial publication Pharmacological Management of Myasthenia Gravis: A Century of Expert Opinions in Cecil Textbook of Medicine (American journal of therapeutics, 2021) Clinical trial publication Two-year treatment with cyclosporine microemulsion for responder myasthenia gravis patients (European neurology, 2010) Clinical trial publication Cyclosporine A: good response for patients affected by autoimmune disorders and HCV infection? (European review for medical and pharmacological sciences, 2009) Clinical trial publication Response to therapy in myasthenia gravis with anti-MuSK antibodies (Annals of the New York Academy of Sciences, 2008) Clinical trial publication P-glycoprotein function in peripheral blood mononuclear cells of myasthenia gravis patients treated with tacrolimus (Biological & pharmaceutical bulletin, 2007) Randomized controlled trial A clinical therapeutic trial of cyclosporine in myasthenia gravis (Annals of the New York Academy of Sciences, 1993) Randomized controlled trial Preliminary results of a double-blind, randomized, placebo-controlled trial of cyclosporine in myasthenia gravis (The New England journal of medicine, 1987) CT.gov search · Cochrane · PubMed	DGIdb	—
CYTARABINE Repurposing via ALK	Small molecule	Approved	Strong	95	—	DGIdb	—
DABRAFENIB Repurposing via ALK	Small molecule	Approved	Strong	95	—	DGIdb	—
DASATINIB ANHYDROUS Repurposing via BTK	Small molecule	Approved	Strong	95	—	DGIdb	—
DEHYDRATED ALCOHOL Repurposing via CTLA4	Small molecule	Approved	Strong	95	1 trials View evidence Registry trials NCT02761707 — Biomarkers in Neural Disorders (COMPLETED) CT.gov search · Cochrane · PubMed	DGIdb	—
DEMECARIUM BROMIDE Repurposing via ACHE	Small molecule	Approved	Strong	95	—	DGIdb	—

Natural Products

12 natural products with the same evidence schema as repurposed drugs — registry trials, PubMed literature, and reference links (pipeline-ranked). Only agents with direct clinical evidence are shown by default.

Reference lookups: Examine.com

Show agents without direct trial evidence

Drug	Type	Phase	Evidence	Score	Trials & literature	Links
Astragalus Natural product Clinical trial publication [Effect of compound astragalus recipe on lymphocyte subset, immunoglobu… In Vitro Study Astragalus significantlyy reduces autoantibodies in cell cultures from…	TCM herb	Preclinical	Emerging	7	1 Trial pubs 1 RCT View evidence Published literature Clinical trial publication [Effect of compound astragalus recipe on lymphocyte subset, immunoglobulin and complements in patients with myasthenia gravia] (Zhongguo Zhong xi yi jie he za zhi Zhongguo Zhongxiyi jiehe zazhi = Chinese journal of integrated traditional and Western medicine, 2009) CT.gov search · Cochrane · PubMed	—
Creatine Natural product Systematic review Characterization of the neuromuscular manifestations in patients with I… Systematic review Immune Checkpoint Inhibitor-Induced Myocarditis with Myositis/Myastheni… Human Study Resistance exercise and creatine supplementation have a therapeutic eff…	Amino acid	Preclinical	Emerging	7	136 pubs View evidence Published literature Systematic review Characterization of the neuromuscular manifestations in patients with IgG4-related disease: a systematic review (Immunologic research, 2025) Systematic review Immune Checkpoint Inhibitor-Induced Myocarditis with Myositis/Myasthenia Gravis Overlap Syndrome: A Systematic Review of Cases (The oncologist, 2021) CT.gov search · Cochrane · PubMed	PubChem
Berberine Natural product In Vitro Study Berberine attenuates experimental autoimmune myasthenia gravis via reba…	Botanical	Preclinical	Emerging	7	1 pubs View evidence CT.gov search · Cochrane · PubMed	PubChem
Curcumin Natural product Commentary Curcumin (from Turmeric) inhibits autoimmune diseases.	Food compound	Preclinical	Emerging	7	4 pubs View evidence CT.gov search · Cochrane · PubMed	PubChem
Melatonin Natural product In Vitro Study Melatonin exerts immunoregulatory effects by balancing peripheral effec… Human Study Glyconutrient supplementation improves the condition of patients with m… Human Study Resistance exercise and creatine supplementation have a therapeutic eff…	Nutraceutical	Preclinical	Emerging	7	4 pubs View evidence CT.gov search · Cochrane · PubMed	PubChem
Thunder God Vine Natural product Human Study Tripterygium wilfordii has a synergistic effect on the efficacy of pred… Animal Study Tripterygium wilfordii attenuates experimentally induced myasthenia gra…	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
Astragaloside Natural product Animal Study Astragaloside IV ameliorates experimental autoimmune myasthenia gravis. Animal Study Astragaloside IV protects against autoimmune myasthenia gravis in rats…	Nutraceutical	Preclinical	Emerging	0	4 pubs View evidence CT.gov search · Cochrane · PubMed	—
Glyconutrient Supplementation Natural product Human Study Glyconutrient supplementation improves the condition of patients with m… Human Study Resistance exercise and creatine supplementation have a therapeutic eff…	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
Tripterygium Wilfordii Natural product Human Study Tripterygium wilfordii has a synergistic effect on the efficacy of pred… Animal Study Tripterygium wilfordii attenuates experimentally induced myasthenia gra…	Nutraceutical	Preclinical	Emerging	0	1 pubs View evidence CT.gov search · Cochrane · PubMed	—
Beta-Glucan Natural product Human Study β-glucans reduce LDL cholesterol in patients with myasthenia gravis.	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
Flavonoids Natural product Animal Study Astilbin ameliorates experimental autoimmune myasthenia gravis.	Nutraceutical	Preclinical	Emerging	0	1 pubs View evidence CT.gov search · Cochrane · PubMed	—
Ligustrazine Natural product Animal Study Tetramethylpyrazine attenuates experimental autoimmune myasthenia gravi…	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—

Alterations and therapeutics are aggregated from public databases (Open Targets, HPO, ChEMBL, DGIdb, and others). Natural agents are candidates from OSMF narrative reviews on opensourcemed.info/chronic-disease.html — not approved treatments. This page is for research orientation only — not medical advice.