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Intelligence · Clinical Pipeline

Rare disease R&D Pipeline

Orphan and rare disease clinical programs including gene and enzyme therapies.

Programs
21

Quick answer

Rare disease: NovaPharmaNews indexes 21 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

  1. -, Sodium butyrate 1,1 g/L sol prep

    ISTA Pharma Limited

    Hirschsprung's disease

    phase 3 small molecule active

    1 trial

  2. AXS-05 (dextromethorphan and bupropion) oral tablets

    Axsome Therapeutics

    Major Depressive Disorder

    phase 3 small molecule completed

    1 trial

  3. AXS-05 (dextromethorphan-bupropion)

    Axsome Therapeutics

    Agitation in Patients With Dementia of the Alzheimer's Type

    phase 3 small molecule completed

    2 trials

  4. Infrared ray heat treatment

    The First People's Hospital of Lianyungang

    Liver Cirrhosis

    phase 3 other active

    1 trial

  5. 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.

    phase 3 small molecule active

    1 trial

  6. Placebo ADCETRIS ( NaCl 0.9% 100 mL - Injection IV ), ADCETRIS 50 mg powder for concentrate for solution for infusion.

    Pari Pharma GmbH

    rare disease

    phase 3 small molecule active

    1 trial

  7. Placebo, Minirin, 4 mikrogramy/ml, roztwór do wstrzykiwań

    Bial Portela & Companhia S.A.

    Rare and ultra-rare glomerulonephritis

    phase 3 small molecule active

    1 trial

  8. Tezspire 210 mg solution for injection in pre-filled syringe, Tezepelumab-placebo

    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

    phase 3 small molecule active

    1 trial

  9. AXS-05 (dextromethorphan-bupropion)

    Axsome Therapeutics

    Treatment Resistant Depression

    phase 2 small molecule terminated

    1 trial

  10. Almonertinib 110 MG

    Chinese University of Hong Kong

    Rare Tumor

    phase 2 small molecule active

    1 trial

  11. Avastin 25 mg/ml concentrate for solution for infusion., Lynparza 150 mg film-coated tablets, Cotellic 20 mg film-coated tablets, Ipatasertib, Cotellic, Cotellic, INAVOLISIB, Sindaxel 6 mg/ml concentrat pentru soluţie perfuzabilă, Verzenios 50 mg film-coated tablets, Cotellic, Lynparza 100 mg film-coated tablets, Kadcyla 160 mg powder for concentrate for solution for infusion., LETRO-cell® 2,5 mg Filmtabletten, INAVOLISIB, Verzenios 100 mg film-coated tablets, INAVOLISIB, Lynparza 150 mg film-coated tablets

    Hoffmann-La Roche

    Persistent or Recurrent Rare Epithelial Ovarian Tumors

    phase 2 small molecule active

    1 trial

  12. Dextromethorphan-Bupropion

    Axsome Therapeutics

    Major Depressive Disorder (MDD)

    phase 2 small molecule active

    1 trial

  13. Nintedanib,Dextromethorphan

    The First People's Hospital of Lianyungang

    Idiopathic Pulmonary Fibrosis (IPF)

    phase 2 small molecule active

    1 trial

  14. 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

    phase 2 small molecule active

    1 trial

  15. A study to investigate the effect of capivasertib on the pharmacokinetics of oral dextromethorphan (CYP2D6) in healthy participants

    AstraZeneca AB

    Healthy participants

    phase 1 other active

    1 trial

  16. DEXTROMETHORPHANE ELERTE 1,5 mg/ml, sirop, AZD3470, AZD3470, Midazolam-ratiopharm® 2 mg/ml orale Lösung

    AstraZeneca AB

    Advanced/metastatic solid tumors that are MTAP deficient.

    phase 1 small molecule active

    1 trial

  17. Dextromethorphan HBr

    Galera Therapeutics

    Healthy

    phase 1 small molecule completed

    1 trial

  18. STRATIFY; Near-infrared fluorescence molecular imaging of adalimumab-800CW to elucidate the drug distribution throughout inflamed tissue in inflammatory bowel disease and rheumatoid arthritis

    Disc Medicine

    Crohn's disease, Spondyloarthritis, Rheumatoid arthritis, Ulcerative colitis

    phase 1 other active

    1 trial

  19. Butorphanol

    Xiyuan Hospital of China Academy of Chinese Medical Sciences

    Visceral Pain

    small molecule completed

    1 trial

  20. Infrared thermography

    The First People's Hospital of Lianyungang

    Chronic Obstructive Pulmonary Disease

    other active

    1 trial

  21. dextromethorphan

    Ultragenyx UK Limited

    Rett Syndrome

    small molecule completed

    1 trial

Frequently asked questions

How many rare disease pipeline programs are tracked?

21 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.

Data attribution: Data sources · All pipeline programs

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