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PTC THERAPEUTICS Pipeline

All ClinicalTrials.gov programs at PTC THERAPEUTICS — phase, modality, and trial IDs.

Programs
52

Quick answer

PTC THERAPEUTICS has 52 clinical development programs indexed from ClinicalTrials.gov, including phase, modality, indication text, and linked NCT identifiers.

  1. Ataluren

    PTC THERAPEUTICS, INC.

    Cystic Fibrosis

    phase 3 small molecule completed

    6 trials

  2. Ataluren

    PTC THERAPEUTICS, INC.

    Muscular Dystrophy, Duchenne

    phase 3 small molecule terminated

    3 trials

  3. Ataluren

    PTC THERAPEUTICS, INC.

    Duchenne Muscular Dystrophy

    phase 3 small molecule terminated

    9 trials

  4. Ataluren (PTC124®)

    PTC THERAPEUTICS, INC.

    Cystic Fibrosis

    phase 3 small molecule completed

    1 trial

  5. Deflazacort

    PTC THERAPEUTICS, INC.

    Duchenne Muscular Dystrophy

    phase 3 small molecule completed

    3 trials

  6. Deflazacort

    PTC THERAPEUTICS, INC.

    Limb-Girdle Muscular Dystrophy

    phase 3 small molecule terminated

    1 trial

  7. PTC299

    PTC THERAPEUTICS, INC.

    Pneumonia

    phase 3 small molecule terminated

    1 trial

  8. PTC923

    PTC THERAPEUTICS, INC.

    Phenylketonuria

    phase 3 small molecule active

    2 trials

  9. PTC923, Kuvan 100 mg soluble tablets, PTC923

    PTC THERAPEUTICS, INC.

    Phenylketonuria

    phase 3 small molecule active

    1 trial

  10. PTC923, PTC923

    PTC THERAPEUTICS, INC.

    Metabolic Disorders - Phenylketonuria

    phase 3 small molecule active

    1 trial

  11. Sephience 250 mg oral powder in sachet, Sephience 1 000 mg oral powder in sachet

    PTC THERAPEUTICS, INC.

    Phenylketonuria

    phase 3 small molecule active

    1 trial

  12. Sepiapterin

    PTC THERAPEUTICS, INC.

    Phenylketonuria

    phase 3 small molecule active

    1 trial

  13. Unesbulin

    PTC THERAPEUTICS, INC.

    Leiomyosarcoma

    phase 3 small molecule terminated

    2 trials

  14. Vatiquinone

    PTC THERAPEUTICS, INC.

    Friedreich ataxia

    phase 3 small molecule active

    1 trial

  15. Vatiquinone

    PTC THERAPEUTICS, INC.

    Mitochondrial Diseases

    phase 3 small molecule terminated

    1 trial

  16. Vatiquinone

    PTC THERAPEUTICS, INC.

    Friedreich Ataxia

    phase 3 small molecule active

    3 trials

  17. Vatiquinone

    PTC THERAPEUTICS, INC.

    Inherited Mitochondrial Disease

    phase 3 small molecule completed

    1 trial

  18. AAV2-hAADC

    PTC THERAPEUTICS, INC.

    Aromatic Amino Acid Decarboxylase Deficiency

    phase 2 small molecule completed

    1 trial

  19. Ataluren

    PTC THERAPEUTICS, INC.

    Aniridia

    phase 2 small molecule terminated

    2 trials

  20. Ataluren

    PTC THERAPEUTICS, INC.

    Nonsene Mutation Duchenne Muscular Dystrophy

    phase 2 small molecule completed

    1 trial

  21. Ataluren

    PTC THERAPEUTICS, INC.

    Amino Acid Metabolism, Inborn Errors

    phase 2 small molecule terminated

    1 trial

  22. Ataluren

    PTC THERAPEUTICS, INC.

    Hemophilia A

    phase 2 small molecule terminated

    1 trial

  23. Ataluren + Pembrolizumab

    PTC THERAPEUTICS, INC.

    Colorectal Cancer

    phase 2 small molecule active

    1 trial

  24. CNSA-001

    PTC THERAPEUTICS, INC.

    Gastroparesis

    phase 2 small molecule completed

    1 trial

  25. EPI-589

    PTC THERAPEUTICS, INC.

    Amyotrophic Lateral Sclerosis

    phase 2 small molecule completed

    1 trial

  26. EPI-743

    PTC THERAPEUTICS, INC.

    Leigh Syndrome

    phase 2 small molecule completed

    1 trial

  27. Eladocagene Exuparvovec

    PTC THERAPEUTICS, INC.

    AADC Deficiency

    phase 2 gene therapy active

    1 trial

  28. PTC124

    PTC THERAPEUTICS, INC.

    Cystic Fibrosis

    phase 2 small molecule completed

    2 trials

  29. PTC124

    PTC THERAPEUTICS, INC.

    Duchenne Muscular Dystrophy

    phase 2 small molecule completed

    1 trial

  30. PTC299

    PTC THERAPEUTICS, INC.

    Neurofibromatosis 2

    phase 2 small molecule terminated

    1 trial

  31. PTC518

    PTC THERAPEUTICS, INC.

    Huntington Disease

    phase 2 small molecule completed

    1 trial

  32. PTC857

    PTC THERAPEUTICS, INC.

    Amyotrophic Lateral Sclerosis

    phase 2 small molecule terminated

    1 trial

  33. PTC857, Placebo to match PTC857 60 mg/mL oral solution, Utreloxastat 62.5

    PTC THERAPEUTICS, INC.

    Amyotrophic Lateral Sclerosis

    phase 2 small molecule active

    1 trial

  34. PTC923

    PTC THERAPEUTICS, INC.

    BH4 Deficiency

    phase 2 small molecule completed

    1 trial

  35. Placebo

    PTC THERAPEUTICS, INC.

    Leigh Syndrome

    phase 2 small molecule completed

    1 trial

  36. Placebo

    PTC THERAPEUTICS, INC.

    Friedreich's Ataxia

    phase 2 small molecule completed

    1 trial

  37. Placebo to match 5-mg PTC518 tablets, Placebo to match 20-mg PTC518 tablets, PTC518, PTC518

    PTC THERAPEUTICS, INC.

    Huntington’s Disease

    phase 2 small molecule active

    1 trial

  38. VEGF inhibitor PTC299

    PTC THERAPEUTICS, INC.

    Kaposi's Sarcoma

    phase 2 small molecule terminated

    1 trial

  39. ataluren

    PTC THERAPEUTICS, INC.

    Epilepsy

    phase 2 small molecule completed

    1 trial

  40. gene therapy

    PTC THERAPEUTICS, INC.

    Aromatic L-amino Acid Decarboxylase (AADC) Deficiency

    phase 2 small molecule completed

    1 trial

  41. Ataluren

    PTC THERAPEUTICS, INC.

    Healthy

    phase 1 small molecule completed

    1 trial

  42. Deflazacort

    PTC THERAPEUTICS, INC.

    Renal Impairment

    phase 1 small molecule completed

    1 trial

  43. Deflazacort

    PTC THERAPEUTICS, INC.

    Hepatic Impairment

    phase 1 small molecule completed

    1 trial

  44. Deflazacort

    PTC THERAPEUTICS, INC.

    Healthy Volunteers

    phase 1 small molecule completed

    1 trial

  45. Deflazacort and rifampin

    PTC THERAPEUTICS, INC.

    Healthy Volunteers

    phase 1 small molecule completed

    1 trial

  46. PTC299

    PTC THERAPEUTICS, INC.

    Leukemia, Myeloid, Acute

    phase 1 small molecule terminated

    1 trial

  47. PTC299

    PTC THERAPEUTICS, INC.

    Advanced Cancer

    phase 1 small molecule terminated

    1 trial

  48. PTC299

    PTC THERAPEUTICS, INC.

    Metastatic Breast Cancer

    phase 1 small molecule completed

    1 trial

Frequently asked questions

How many pipeline programs does PTC THERAPEUTICS have?

52 published programs are linked to this sponsor in our ClinicalTrials.gov index.

Is this the complete company pipeline?

This hub lists all published programs for the sponsor in our database. Partnered or out-licensed assets may appear under multiple companies.

Data attribution: Data sources · All pipeline programs

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