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: 09 Aug 2017

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At a glance

  • Originator Pfizer
  • Developer 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
  • Phase I Haematological malignancies
  • No development reported Solid tumours

Most Recent Events

  • 21 Jul 2017 Pfizer completes a phase I bioequivalence trial in Healthy volunteers in USA (PO) (NCT03130556)
  • 09 Jun 2017 Pfizer initiates enrolment in a phase I trial in Healthy volunteers in Belgium (PO) (NCT03162900)
  • 02 Jun 2017 Interim adverse events and efficacy data from a phase I/II trial in Myelofibrosis presented at the 53rd Annual Meeting of the American Society of Clinical Oncology (ASCO-2017)
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