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Intelligence · Clinical Pipeline

R&D Pipeline Programs

ClinicalTrials.gov programs linked to pharma sponsors — phase, modality, and trial IDs for BD and competitive intelligence.

Programs indexed
18,156
Companies
1,062
Drugs tracked
3,385

Quick answer

NovaPharmaNews tracks 18,156 pharmaceutical R&D pipeline programs sourced from ClinicalTrials.gov, linked to sponsor companies. Each profile includes development phase, modality, trial status, NCT identifiers, and indication text — searchable for business development and competitive intelligence.

201 results for “Ultragenyx” Clear

  1. ,4-diaminopyridine

    Ultragenyx UK Limited

    Polyradiculoneuropathy, Chronic Inflammatory Demyelinating

    phase 3 small molecule completed

    1 trial

  2. 5 mg prednisone

    Ultragenyx UK Limited

    Granulomatosis With Polyangiitis

    phase 3 small molecule active

    2 trials

  3. Aceneuramic Acid Extended-Release Tablets

    Ultragenyx UK Limited

    Hereditary Inclusion Body Myopathy

    phase 3 small molecule terminated

    1 trial

  4. Adjuvant Immunomodulatory (IM) Therapy

    Ultragenyx UK Limited

    Mucopolysaccharidosis IIIA

    phase 3 small molecule active

    1 trial

  5. Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day

    Ultragenyx UK Limited

    Sanfilippo Syndrome

    phase 3 small molecule active

    1 trial

  6. Analatro

    Ultragenyx UK Limited

    Latrodectism

    phase 3 small molecule completed

    1 trial

  7. Anti-thymocyte globulin

    Ultragenyx UK Limited

    Aplastic Anemia

    phase 3 small molecule completed

    1 trial

  8. Bisphosphonate

    Ultragenyx UK Limited

    Osteogenesis Imperfecta

    phase 3 small molecule active

    1 trial

  9. Burosumab

    Ultragenyx UK Limited

    Hypophosphatemia

    phase 3 mab completed

    1 trial

  10. DTX301

    Ultragenyx UK Limited

    OTC Deficiency

    phase 3 gene therapy active

    1 trial

  11. DTX401

    Ultragenyx UK Limited

    Glycogen Storage Disease Type IA

    phase 3 gene therapy completed

    1 trial

  12. Evinacumab

    Ultragenyx UK Limited

    Homozygous Familial Hypercholesterolemia

    phase 3 small molecule completed

    1 trial

  13. Fluticasone Propionate, 880 mcg twice daily (after 6FED failure)

    Ultragenyx UK Limited

    Eosinophilic Gastrointestinal Disorders (EGIDs)

    phase 3 small molecule completed

    1 trial

  14. GTX-102

    Ultragenyx UK Limited

    Angelman Syndrome

    phase 3 small molecule active

    4 trials

  15. Plasma exchange

    Ultragenyx UK Limited

    Acute Disseminated Encephalomyelitis

    phase 3 other active

    1 trial

  16. Rifampicin

    Ultragenyx UK Limited

    Multiple System Atrophy

    phase 3 small molecule terminated

    1 trial

  17. Sirolimus

    Ultragenyx UK Limited

    Lymphangioleiomyomatosis

    phase 3 small molecule completed

    1 trial

  18. Tiratricol

    Ultragenyx UK Limited

    Monocarboxylate Transporter 8 Deficiency

    phase 3 small molecule completed

    1 trial

  19. Triheptanoin

    Ultragenyx UK Limited

    Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

    phase 3 small molecule active

    1 trial

  20. UX003

    Ultragenyx UK Limited

    Sly Syndrome

    phase 3 small molecule completed

    2 trials

  21. UX003

    Ultragenyx UK Limited

    MPS 7

    phase 3 small molecule completed

    1 trial

  22. UX007

    Ultragenyx UK Limited

    Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

    phase 3 small molecule terminated

    2 trials

  23. aceneuramic acid extended-release (Ace-ER)

    Ultragenyx UK Limited

    Hereditary Inclusion Body Myopathy

    phase 3 small molecule completed

    1 trial

  24. acyclovir

    Ultragenyx UK Limited

    Herpes Simplex

    phase 3 small molecule completed

    2 trials

Frequently asked questions

What is the NovaPharmaNews pipeline directory?

It is a searchable index of 18,156 clinical development programs grouped by sponsor company, with phase (Phase 1–3), modality, trial status, and linked NCT IDs from ClinicalTrials.gov.

Where does pipeline data come from?

Program records are derived from ClinicalTrials.gov and linked to NovaPharmaNews company profiles. Trial status and phase reflect registry data at last sync — verify on ClinicalTrials.gov before investment or partnering decisions. See our data sources page for attribution.

How do I find pipeline programs by company or phase?

Use the search box to match program name, indication, company name, or slug. Results are ordered with late-stage (Phase 3) programs first. Open any row to view the full program profile under its sponsor company.

Data attribution and licensing: Data sources