Intelligence · Clinical pipeline
Rare disease · Phase III
Rare disease programs at Phase III — ClinicalTrials.gov pipeline intelligence.
Quick answer
Rare disease Phase III programs: NovaPharmaNews indexes 8 matching clinical development programs from ClinicalTrials.gov with sponsor, modality, and NCT cross-links.
Top sponsors
Pipeline programs
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2021/ABM/01/00010
Bial Portela & Companhia S.A.
Rare and ultra-rare glomerulonephritis
1 trial -
2025-521093-34-00
Pari Pharma GmbH
rare disease
1 trial -
AXS-05 (dextromethorphan and bupropion) oral tablets
Axsome Therapeutics
Major Depressive Disorder
1 trial -
AXS-05 (dextromethorphan-bupropion)
Axsome Therapeutics
Agitation in Patients With Dementia of the Alzheimer's Type
2 trials -
CROSSING
AstraZeneca AB
Eosinophilic esophagitis (EoE) is a rare, chronic inflammatory disorder triggered by an immune response to foods and aeroantigens and characterized by a combination of esophageal dysfunction and eosinophilic infiltration of the esophagus
1 trial -
Infrared ray heat treatment
The First People's Hospital of Lianyungang
Liver Cirrhosis
1 trial -
Nomlabofusp, Placebo to Nomlabofusp
Larimar Therapeutics
Friedreich’s ataxia is the most common inherited ataxia in humans and results from a deficiency of the mitochondrial protein, FXN. Friedreich’s ataxia is a rare, progressive, multisystem disease with an incidence that is estimated to be 1:29,000, and a carrier frequency of ~1:85.
1 trial -
RCAPHM18_0018
ISTA Pharma Limited
Hirschsprung's disease
1 trial
Frequently asked questions
How many rare disease Phase III programs exist?
8 programs match both the therapeutic area and stage filters in our published index.