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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.

91 results for “BioMarin” Clear

  1. Phenoptin

    BioMarin International Limited

    Tetrahydrobiopterin Deficiencies

    phase 2 small molecule completed

    1 trial

  2. Regimen Selection Phase Group 2

    BioMarin International Limited

    Duchenne Muscular Dystrophy

    phase 2 small molecule terminated

    1 trial

  3. Sapropterin

    BioMarin International Limited

    Phenylketonuria

    phase 2 small molecule terminated

    1 trial

  4. Sapropterin

    BioMarin International Limited

    GTP Cyclohydrolase Deficiency

    phase 2 small molecule completed

    1 trial

  5. Sapropterin Dihydrochloride

    BioMarin International Limited

    Endothelial Dysfunction

    phase 2 small molecule completed

    1 trial

  6. Sapropterin Dihydrochloride

    BioMarin International Limited

    Sickle Cell Disease

    phase 2 small molecule completed

    1 trial

  7. Sapropterin Dihydrochloride

    BioMarin International Limited

    Intermittent Claudication

    phase 2 small molecule completed

    1 trial

  8. Talazoparib Tosylate

    BioMarin International Limited

    Advanced Breast Cancer

    phase 2 small molecule completed

    1 trial

  9. Valoctocogene roxaparvovec

    BioMarin International Limited

    Hemophilia A With Inhibitor

    phase 2 mab active

    1 trial

  10. Vosoritide Injection

    BioMarin International Limited

    Idiopathic Short Stature

    phase 2 small molecule active

    1 trial

  11. Vosoritide Injection

    BioMarin International Limited

    Noonan Syndrome

    phase 2 small molecule active

    1 trial

  12. Voxzogo 0.56 mg powder and solvent for solution for injection, Voxzogo 1.2 mg powder and solvent for solution for injection

    BioMarin International Limited

    Achondroplasia

    phase 2 small molecule active

    1 trial

  13. Voxzogo 0.56 mg powder and solvent for solution for injection, modified recombinant human C-type natriuretic peptide (rhCNP), Lyophilized powder for reconstitution, Voxzogo 1.2 mg powder and solvent for solution for injection

    BioMarin International Limited

    Idiopathic Short Stature (ISS)

    phase 2 small molecule active

    1 trial

  14. Voxzogo 0.56 mg powder and solvent for solution for injection, powder and solvent for solution for injection

    BioMarin International Limited

    Hypochondroplasia

    phase 2 small molecule active

    1 trial

  15. Voxzogo 1.2 mg powder and solvent for solution for injection, Voxzogo 0.56 mg powder and solvent for solution for injection, modified recombinant human C-type natriuretic peptide (rhCNP)

    BioMarin International Limited

    Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome

    phase 2 small molecule active

    1 trial

  16. rAvPAL-PEG

    BioMarin International Limited

    Phenylketonuria

    phase 2 small molecule completed

    3 trials

  17. rAvPAL-PEG 0.001 mg/kg

    BioMarin International Limited

    Phenylketonuria

    phase 2 small molecule completed

    1 trial

  18. sapropterin

    BioMarin International Limited

    Autistic Disorder

    phase 2 small molecule completed

    1 trial

  19. valoctocogene roxaparvovec

    BioMarin International Limited

    Severe Haemophilia A

    phase 2 mab completed

    1 trial

  20. vosoritide

    BioMarin International Limited

    Achondroplasia

    phase 2 mab active

    1 trial

  21. BMN 111 administration via Injector Pen

    BioMarin International Limited

    Achondroplasia

    phase 1 other terminated

    1 trial

  22. BMN 255

    BioMarin International Limited

    Hyperoxaluria

    phase 1 small molecule terminated

    1 trial

  23. BMN 349

    BioMarin International Limited

    Alpha 1-Antitrypsin Deficiency

    phase 1 small molecule active

    1 trial

  24. Cisplatin

    BioMarin International Limited

    Solid Tumors

    phase 1 small molecule terminated

    1 trial

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