Nitisinone - Acorda Therapeutics

Drug Profile

Nitisinone - Acorda Therapeutics

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

Latest Information Update: 12 Sep 2017

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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

  • 05 Sep 2017 Safety and efficacy data from a clinical trial in Tyrosinaemia type I released by Sobi
  • 05 Sep 2017 The US FDA approves nitisinone for reduced dosing frequency for the treatment of Tyrosinaemia type I from twice daily to once daily in patients 5 years of age and older
  • 24 Jul 2017 Swedish Orphan Biovitrum completes a phase I drug-drug interaction trial in Healthy volunteers in Germany (PO) (NCT03103568)
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