Sebelipase alfa

Drug Profile

Sebelipase alfa

Alternative Names: Kanuma; Lysosomal acid lipase - Synageva BioPharma; Recombinant human lysosomal acid lipase - Synageva BioPharma; Recombinant lysosomal acid lipase - Synageva BioPharma; rhLAL - Synageva BioPharma; rLAL; SBC-102

Latest Information Update: 22 Nov 2016

Price : $50

At a glance

  • Originator Synageva BioPharma
  • Developer Alexion Pharmaceuticals
  • Class Carboxylic ester hydrolases; Proteins
  • Mechanism of Action Sterol esterase replacements; Sterol esterase stimulants
  • Orphan Drug Status

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

    Yes - Wolman disease; Cholesterol ester storage disease
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

    Wolman disease
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Cholesterol ester storage disease; Wolman disease

Most Recent Events

  • 11 Nov 2016 Additional long term safety and efficacy data from the phase III ARISE trial in Cholesterol ester storage disease presented at the Liver Meeting 2016, the annual meeting of the American Association for the Study of Liver Diseases (AASLD-2016)
  • 06 Oct 2016 Positive long-term efficacy data from the phase III ARISE trial in Cholesterol ester storage disease released by Alexion pharmaceuticals
  • 28 Mar 2016 Registered for Cholesterol ester storage disease (In children, In adults) in Japan (IV)
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