Rare Diseases

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

Companies

Pipeline Drugs

588

Key People

1087

Rare Diseases Pipeline (588 drugs)

Commercial: 81Approved: 77Preclinical: 69Phase 3: 66Phase 1/2: 53Phase 1: 44Phase 2: 44Pre-clinical: 33Approved/Commercial: 27Phase 2/3: 10N/A: 8Discovery: 6Research: 5Preclinical/Phase 1: 5Clinical: 3Phase 2b: 3Development: 3Clinical Development: 3Not Specified: 3Phase 2a: 2Registrational: 2Proof of Concept: 2Regulatory: 2Phase 1b/2: 2Phase 1b: 2Clinical Research: 2Multiple Phases: 1Pivotal Phase 2: 1NDA Review: 1Pre-clinical/Clinical: 1NDA Submitted: 1Phase (Partnered): 1Approved (Tentative): 1Preclinical/Research: 1Pre-Clinical/Phase 1: 1Pipeline: 1Research/Preclinical: 1Research/Commercial: 1Filed (EU): 1Phase 1 / Phase 2: 1Not Specified (Preclinical/Discovery): 1Phase 1b/2a: 1Clinical Evaluation: 1Marketed: 1Phase 2 (IND Active): 1Phase 2 Ready: 1Pre-clinical / Clinical: 1Discovery/Research: 1Phase 3 (likely): 1Phase 1/Phase 2 (implied): 1NDA/BLA Filed: 1Pivotal Trial: 1Preclinical / Phase 1: 1Approved/Procedure: 1Not Disclosed: 1N/A (Diagnostic): 1Under Review: 1

Drug	Company	Indication	Phase	Status
Patisiran (ONPATTRO)	Alnylam Pharmaceuticals	Hereditary Transthyretin Amyloidosis	Approved	Commercial
Givosiran (GIVLAARI)	Alnylam Pharmaceuticals	Acute Hepatic Porphyria	Approved	Commercial
Lumasiran (OXLUMO)	Alnylam Pharmaceuticals	Primary Hyperoxaluria Type 1	Approved	Commercial
Inclisiran (LEQVIO)	Alnylam Pharmaceuticals	Hypercholesterolemia	Approved	Commercial - Partnered
Vutrisiran	Alnylam Pharmaceuticals	Hereditary Transthyretin Amyloidosis	Phase 3	Active
ALN-AGT01	Alnylam Pharmaceuticals	Hypertension	Phase 1	Active
ALN-KHK	Alnylam Pharmaceuticals	Primary Hyperoxaluria	Phase 3	Active
ALN-APP	Alnylam Pharmaceuticals	Alzheimer's Disease	Phase 1	Active
Cemdisiran	Alnylam Pharmaceuticals	C5 Complement-mediated Diseases	Phase 2	Active
Tagrisso (osimertinib)	AstraZeneca	EGFR-mutated lung cancer	Approved	Commercial
Imfinzi (durvalumab)	AstraZeneca	Various cancers	Approved	Commercial
Lynparza (olaparib)	AstraZeneca	BRCA-mutated cancers	Approved	Commercial
Calquence (acalabrutinib)	AstraZeneca	Blood cancers	Approved	Commercial
Farxiga (dapagliflozin)	AstraZeneca	Diabetes/Heart failure/CKD	Approved	Commercial
Brilinta (ticagrelor)	AstraZeneca	Cardiovascular disease	Approved	Commercial
Symbicort	AstraZeneca	Asthma/COPD	Approved	Commercial
Breztri	AstraZeneca	COPD	Approved	Commercial
Forxiga	AstraZeneca	Type 2 diabetes	Approved	Commercial
Crestor (rosuvastatin)	AstraZeneca	High cholesterol	Approved	Commercial
Venglustat	Sanofi	Type 3 Gaucher disease	Phase 3	Breakthrough Therapy designation
Rilzabrutinib	Sanofi	IgG4-related disease	Clinical	Orphan drug designation
Dupixent	Sanofi	Atopic dermatitis	Commercial	Marketed
Toujeo	Sanofi	Diabetes	Commercial	Marketed
Lantus	Sanofi	Diabetes	Commercial	Marketed
Entyvio (vedolizumab)	Takeda Pharmaceutical	Inflammatory Bowel Disease	Commercial	Approved
Ninlaro (ixazomib)	Takeda Pharmaceutical	Multiple Myeloma	Commercial	Approved
Vyvanse (lisdexamfetamine)	Takeda Pharmaceutical	ADHD	Commercial	Approved
Adcetris (brentuximab vedotin)	Takeda Pharmaceutical	Lymphomas	Commercial	Approved
Takecab (vonoprazan)	Takeda Pharmaceutical	Acid-related disorders	Commercial	Approved
TAK-981	Takeda Pharmaceutical	Solid tumors	Phase 1/2	Active
TAK-676	Takeda Pharmaceutical	Solid tumors	Phase 1	Active
TAK-573	Takeda Pharmaceutical	Solid tumors	Phase 1	Active
TAK-164	Takeda Pharmaceutical	Solid tumors	Phase 1	Active
Soticlestat (TAK-935)	Takeda Pharmaceutical	Epilepsy	Phase 3	Active
TAK-721	Takeda Pharmaceutical	Narcolepsy	Phase 3	Active
Hemlibra (emicizumab)	Chugai Pharmaceutical	Hemophilia A	Approved	Commercial
Tecentriq (atezolizumab)	Chugai Pharmaceutical	Multiple Cancers	Approved	Commercial
Kadcyla (T-DM1)	Chugai Pharmaceutical	HER2+ Breast Cancer	Approved	Commercial
Phesgo	Chugai Pharmaceutical	HER2+ Breast Cancer	Approved	Commercial
Herceptin (trastuzumab)	Chugai Pharmaceutical	HER2+ Cancers	Approved	Commercial
Avastin (bevacizumab)	Chugai Pharmaceutical	Multiple Cancers	Approved	Commercial
Actemra (tocilizumab)	Chugai Pharmaceutical	Rheumatoid Arthritis	Approved	Commercial
Satralizumab	Chugai Pharmaceutical	Neuromyelitis Optica	Approved	Commercial
Crovalimab	Chugai Pharmaceutical	Paroxysmal Nocturnal Hemoglobinuria	Phase 3	Active
GYM329	Chugai Pharmaceutical	Spinal Muscular Atrophy	Phase 2	Active
Tiragolumab	Chugai Pharmaceutical	Cancer combination therapy	Phase 3	Active
CSL112	CSL	Acute Coronary Syndrome	Phase 3	Active
CSL312	CSL	Hereditary Angioedema	Phase 3	Active
Eteplirsen	CSL	Duchenne Muscular Dystrophy	Commercial	Marketed
Hemgenix (CSL222)	CSL	Hemophilia B	Commercial	FDA Approved