Nitisinone

Drug Profile

Nitisinone

Alternative Names: NTBC; Orfadin; SC 0735; SYN-118

Latest Information Update: 20 May 2017

Price : $50

At a glance

  • Originator Syngenta
  • Developer Biotie Therapies Corp.; Swedish Orphan Biovitrum; University of Liverpool
  • Class Antiparkinsonians; Cyclohexanones; Nitrobenzoates; Small molecules
  • Mechanism of Action 4-hydroxyphenylpyruvate dioxygenase inhibitors; Dopamine release stimulants
  • Orphan Drug Status

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

    Yes - Tyrosinaemia type I
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Tyrosinaemia type I
  • Phase III Inborn error metabolic disorders
  • Discontinued Parkinson's disease

Most Recent Events

  • 17 May 2017 Registered for Tyrosinaemia type I in Saudi Arabia (PO)
  • 28 Mar 2017 Swedish Orphan Biovitrum initiates enrolment in a phase I trial in Healthy volunteers in Germany (NCT03103568)
  • 06 Mar 2017 US FDA approves nitisinone for increased shelf life and in-use storage at room temperature for treatment of Tyrosinaemia type 1 in USA
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