Rare Diseases

Orphan drug development for conditions affecting small patient populations, often with significant unmet medical need.

Companies

Pipeline Drugs

829

Key People

1118

Rare Diseases Pipeline (829 drugs)

Preclinical: 121Phase 3: 94Pre-clinical: 74Commercial: 69Approved: 63Phase 2: 61Phase 1: 38Phase 1/2: 34Marketed: 27Discovery: 26Research: 14Development: 12Phase III: 11Phase 2b: 9Phase 2/3: 9Discovery/Preclinical: 5Phase 2A: 4Discovery/Pre-clinical: 4Phase I/II: 4Commercial/Development: 3NDA Submitted: 3Service: 3Development/Validation: 3Phase 3 / Registration: 3Unknown: 3Phase 2B/3: 3Preclinical/Phase 1: 3Clinical-stage: 3Phase II: 3Not Specified: 3Phase 1/2a: 2Pre-clinical / Phase 1: 2Clinical Stage (Phase not specified): 2Research/Development: 2Phase 2-ready: 2Pivotal: 2Phase 1/Phase 2: 2Phase 2b/3: 2Discovery/Optimization: 2Lead Optimization: 2NDA Filed: 2Regulatory Review: 2Phase 1b/2: 1Conditionally Approved: 1Seeking Approval: 1Phase 2a: 1Phase I-III: 1N/A: 1Phase IV: 1Development/Commercial (LDT): 1Discovery/Development: 1N/A (Diagnostic): 1Under Review: 1Marketed (EU): 1Pre-clinical or Undisclosed: 1Commercialization: 1Phase 1/2 (inferred): 1Pre-clinical/Early Clinical: 1Pre-clinical/Research: 1Prototyping: 1Feasibility Study: 1N/A (Technology Development): 1Phase 2a completed: 1Developed (LDT): 1IND enabling: 1AI Discovery: 1Commercial / Line Extension: 1Pre-clinical / Early Clinical: 1Preclinical or Phase 1: 1Registry: 1Clinical-stage (Phase unspecified): 1Early Access: 1Research Collaboration: 1Phase 2B: 1Pre-clinical/Development: 1ANDA Filed/Development: 1Preclinical/Research: 1Research/Preclinical: 1N/A (Early Access Program): 1N/A (Commercial/Expanded Access): 1N/A (Donation/Distribution Program): 1Clinical Trial Initiated: 1Preclinical/IND-enabling: 1IND-enabling: 1Licensed/Orphan Designation: 1Phase 2/Spin-out: 1Development/Commercial: 1Research to Clinical: 1Phase 1/2 (not specified): 1Diagnostic Development: 1Service/Commercial: 1Commercial/Implementation: 1Research Use: 1Phase 3 Preparations: 1In-vivo: 1In-vitro: 1Pre-clinical development: 1Diagnostic: 1Diagnostic/Research: 1Research Service: 1Phase 1/1b: 1Approved (seeking new formulation): 1Clinical Development (phase unspecified): 1Preclinical/Co-development: 1Pre-registration: 1Preclinical to Phase 2: 1Phase III / Marketed: 1Phase I: 1Launched: 1Phase II/III: 1Regulatory (EU): 1Approved (EU): 1Clinical Utility Study: 1Clinical Evidence Generation: 1Commercial Launch: 1Translational Studies: 1Collaborative Development: 1Data Analysis: 1Ongoing Validation: 1Phase 3b / Commercial: 1Marketed/Phase 3: 1Marketed / Late-stage Study: 1Preclinical/Phase I: 1Registration: 1Pilot/Development: 1Approved / Phase 3: 1Filed: 1Phase 2/Phase 3: 1BLA Submission: 1Submitted: 1Phase 2 Ready: 1Phase 1/IIa: 1Preclinical (IND granted per timeline): 1Phase 3 (Planned): 1Phase 1b: 1

Drug	Company	Indication	Phase	Status
Lectin-Fusion for LSDs (e.g., MPS I, Pompe)	SylamoreBio	Lysosomal Storage Disorders with CNS involvement	Pre-clinical	Active
Lectin-Fusion for Bone/Muscle Disorders	SylamoreBio	Musculoskeletal pathologies, abnormal bone growth	Pre-clinical	Active
GS1191	Gritgen Therapeutics	Hemophilia A	Phase 1	Active
PCS6422	Processa Pharmaceuticals	Metastatic Colorectal Cancer & Metastatic Breast Cancer	Phase 1b/2	Active
PCS499	Processa Pharmaceuticals	Ulcerative Necrobiosis Lipoidica	Phase 2b	Active (Seeking Partnership)
PCS12852	Processa Pharmaceuticals	Gastroparesis and GI Motility Disorders	Phase 2	Active (Seeking Partnership)
PCS11T	Processa Pharmaceuticals	Oncology (Not Specified)	Preclinical	Active
Nomacopan	Akari Therapeutics	Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy (HSCT-TMA)	Phase 3	Active
SYNB1934	Synlogic	Phenylketonuria (PKU)	Phase 3	Completed; Did Not Meet Primary Endpoint
SYNB1353	Synlogic	Homocystinuria (HCU)	Phase 1/2a	Active
CGC729	CureGenetics	Relapsed/Refractory Metastatic Clear Cell Renal Cell Carcinoma	Phase 1	Active
Terlipressin (TERLIVAZ®)	Mallinckrodt Pharmaceuticals	Hepatorenal Syndrome Type 1 (HRS-1)	Approved	Commercial
Stannsoporfin (INH-001)	Mallinckrodt Pharmaceuticals	Prevention of severe hyperbilirubinemia in newborns	Phase 3	Development
StrataGraft	Mallinckrodt Pharmaceuticals	Thermal Burns	Approved	Commercial
H.P. Acthar® Gel	Mallinckrodt Pharmaceuticals	Multiple autoimmune/inflammatory conditions	Approved	Commercial
eDSP (EryDex)	Quince Therapeutics	Ataxia-Telangiectasia (A-T)	Phase 3	Pivotal NEAT trial completed Jan 2026; primary endpoint not met. Under strategic review.
ATR-12	Azitra	Netherton Syndrome	Phase 1	Active
ATR-04	Azitra	EGFR Inhibitor-Associated Rash	Preclinical	Active
ATR-01	Azitra	Ichthyosis Vulgaris	Preclinical	Active
Mytesi (crofelemer)	Jaguar Health	Non-infectious diarrhea in adults with HIV/AIDS on ART	Commercial	Approved
Crofelemer	Jaguar Health	Cancer Therapy-Related Diarrhea (CTD)	Phase 3	Ongoing - OnTarget trial
Crofelemer (liquid)	Jaguar Health	SBS-IF & Congenital Diarrheal Disorders (CDD)	Development	Formulation development
NP-300	Jaguar Health	Diarrhea from cholera	Preclinical	NIAID-funded toxicity studies
Canalevia-CA1 (crofelemer)	Jaguar Health	Chemotherapy-Induced Diarrhea in dogs	Conditionally Approved	Conditional market approval (US)
Canalevia-CA2	Jaguar Health	Exercise-Induced Diarrhea in dogs	Seeking Approval	Pursuing conditional market approval
eRapa	Biodexa Pharmaceuticals	Familial Adenomatous Polyposis (FAP)	Phase 3	Active - Enrollment
MTX240 (OPB-171775)	Biodexa Pharmaceuticals	TKI-Resistant Gastrointestinal Stromal Tumors (GIST)	Phase 1/2	Licensed Asset
Tolimidone	Biodexa Pharmaceuticals	Type 2 Diabetes	Phase 2	Acquired Asset
FP-045	Foresee Pharmaceuticals	Fanconi Anemia	Phase 2	Active
Leuprolide (SIF Formulation)	Foresee Pharmaceuticals	Central Precocious Puberty (CPP)	Phase 3	Active (Caspian Study)
FP-020 (BMS-986235)	Foresee Pharmaceuticals	Idiopathic Pulmonary Fibrosis (IPF)	Phase 2	Active
FP-025	Foresee Pharmaceuticals	Asthma / Inflammatory Diseases	Phase 2	Completed (Out-licensed)
FP-004 (SIF Formulation)	Foresee Pharmaceuticals	Opioid Use Disorder	Preclinical	Active
IMX101	Imunexus Therapeutics	Small Cell Lung Cancer	Preclinical	Active
IMX113	Imunexus Therapeutics	Rare Pediatric Disease	Preclinical	Active
IMX39	Imunexus Therapeutics	Multiple Sclerosis	Preclinical	Active
Dupixent (dupilumab)	Regeneron	Chronic Obstructive Pulmonary Disease (COPD)	Phase 3	Active
Odronextamab	Regeneron	Relapsed/Refractory Follicular Lymphoma & DLBCL	Phase 3	Active (BLA under review)
Pozelimab	Regeneron	Complement 3 Glomerulopathy (C3G)	Phase 3	Active
Garetosmab	Regeneron	Fibrodysplasia Ossificans Progressiva (FOP)	Phase 3	Active
Linvoseltamab	Regeneron	Relapsed/Refractory Multiple Myeloma	Phase 3	Active
Fianlimab + Cemiplimab	Regeneron	Advanced Melanoma (1L adjuvant)	Phase 3	Active
REGN1908-1909 (Fel d 1 mAb)	Regeneron	Cat Allergy	Phase 3	Active
VEGF C-D	Regeneron	Neovascular Age-Related Macular Degeneration (nAMD)	Phase 2/3	Active
TAK-755 (rADAMTS13)	Takeda	Congenital Thrombotic Thrombocytopenic Purpura (cTTP)	Phase 3	Active
TAK-861	Takeda	Narcolepsy Type 1 (NT1)	Phase 3	Active
TAK-279 (NDI-034858)	Takeda	Moderate-to-Severe Plaque Psoriasis	Phase 3	Active
Soticlestat (TAK-935)	Takeda	Dravet Syndrome, Lennox-Gastaut Syndrome	Phase 3	Active
TAK-676 + pembrolizumab	Takeda	Solid Tumors	Phase 1/2	Active
TAK-500	Takeda	Solid Tumors	Phase 1/2	Active