Intelligence · Clinical pipeline
Rare disease R&D Pipeline
Orphan and rare disease clinical programs including gene and enzyme therapies.
Quick answer
Rare disease: NovaPharmaNews indexes 19 rare disease clinical pipeline programs from ClinicalTrials.gov with phase, modality, NCT identifiers, and sponsor company links. Orphan and rare disease clinical programs including gene and enzyme therapies.
Top sponsors
- Axsome Therapeutics 4 programs
- First People's Hospital 3 programs
- AstraZeneca 1 programs
- Bial Portela & 1 programs
- Chinese Academy of 1 programs
- Chinese University of 1 programs
- Disc Medicine 1 programs
- HOFFMANN-LA ROCHE 1 programs
- Ishita Pharma - 1 programs
- Kailera Therapeutics 1 programs
- Larimar Therapeutics 1 programs
- PARItec 1 programs
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 -
AXS-05 (dextromethorphan-bupropion)
Axsome Therapeutics
Treatment Resistant Depression
1 trial -
Almonertinib 110 MG
Chinese University of Hong Kong
Rare Tumor
1 trial -
Dextromethorphan-Bupropion
Axsome Therapeutics
Major Depressive Disorder (MDD)
1 trial -
Nintedanib,Dextromethorphan
The First People's Hospital of Lianyungang
Idiopathic Pulmonary Fibrosis (IPF)
1 trial -
Rapamune 1 mg/mL oral solution
Tour Hekla
Lingual microcystic lymphatic malformations (LMLMs) are rare congenital vascular malformations, presenting as clusters of cysts filled with lymph fluid or blood. They are responsible for a heavy burden even with small well-limited lesions because of oozing, bleeding, infections, or even speech, chewing or breathing impairment. Pain and aesthetic prejudice are also frequently reported
1 trial -
WO42178
Hoffmann-La Roche
Persistent or Recurrent Rare Epithelial Ovarian Tumors
1 trial -
Dextromethorphan HBr
Galera Therapeutics
Healthy
1 trial -
STRATIFY; Near-infrared fluorescence molecula...
Disc Medicine
Crohn's disease, Spondyloarthritis, Rheumatoid arthritis, Ulcerative colitis
1 trial -
Butorphanol
Xiyuan Hospital of China Academy of Chinese Medical Sciences
Visceral Pain
1 trial -
Infrared thermography
The First People's Hospital of Lianyungang
Chronic Obstructive Pulmonary Disease
1 trial -
dextromethorphan
Ultragenyx UK Limited
Rett Syndrome
1 trial
Frequently asked questions
How many rare disease pipeline programs are tracked?
19 programs match this therapeutic area based on indication text, program name, and registry metadata.
Where does pipeline data come from?
ClinicalTrials.gov records linked to NovaPharmaNews company profiles. Verify on ClinicalTrials.gov before investment decisions.