Drug Profile


Alternative Names: Oral Hedgehog Inhibitor - Pfizer; PF 04; PF-04449913; PF-4449913; Smo Inhibitor - Pfizer; Sonic Hedgehog pathway inhibitor (PF 4449913) - Pfizer

Latest Information Update: 16 Oct 2017

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At a glance

  • Originator Pfizer
  • Developer Grupo Espanol de Trasplante Hematopoyetico y Terapia Celular; H. Lee Moffitt Cancer Center and Research Institute; Netherlands Cancer Institute; Pfizer
  • Class Antineoplastics; Benzimidazoles; Phenylurea compounds; Piperidines; Small molecules
  • Mechanism of Action SMO protein inhibitors
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

  • New Molecular Entity Yes

Highest Development Phases

  • Phase II Acute myeloid leukaemia; Chronic myeloid leukaemia; Colorectal cancer; Myelodysplastic syndromes; Myelofibrosis; Non-small cell lung cancer
  • Phase I/II Chronic myelomonocytic leukaemia; Graft-versus-host disease
  • Phase I Cancer; Haematological malignancies
  • No development reported Solid tumours

Most Recent Events

  • 28 Sep 2017 Phase-I/II clinical trials in Graft-versus-host disease (Treatment-experienced) in Spain (PO, Tablet) (EudraCT2017-000862-31)
  • 07 Sep 2017 Phase-I clinical trials in Cancer (In volunteers) in USA (PO) (IV) (NCT03270878)
  • 04 Sep 2017 Pfizer plans a phase I trial in Healthy volunteers in USA (IV) (PO) (NCT03270878)
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