Journal article
Turoctocog alfa and drug development for hemophilia A
Expert opinion on orphan drugs, Vol.2(4), pp.419-431
04/01/2014
DOI: 10.1517/21678707.2014.891458
Abstract
Introduction:
Prophylactic replacement therapy with factor VIII (FVIII) is recommended as the foundation of treatment for severe hemophilia A; however, access to such therapy remains limited in many parts of the world. Introducing new products into the market may increase treatment accessibility across the global hemophilia community.
Turoctocog alfa
, a new and recently developed B-domain-truncated recombinant FVIII concentrate, further expands the therapeutic armamentarium for the prevention and treatment of bleeding episodes in hemophilia A. The lessons learned and insights gained throughout the nonclinical and clinical development of turoctocog alfa may offer guidance for future research in hemophilia A.
Areas covered:
This review summarizes the development of turoctocog alfa from production methods through to Phase III clinical development and the outcomes achieved in the pivotal guardian
™
trial program. Key references relating to each step of the development process have been considered.
Expert opinion:
The turoctocog alfa clinical development program has led not only to the availability of a new, effective FVIII product for the treatment of hemophilia A but it has also demonstrated that large multinational trials can be successfully and quickly completed in this rare patient population.
Details
- Title: Subtitle
- Turoctocog alfa and drug development for hemophilia A
- Creators
- Steven R Lentz - University of Iowa Carver College of Medicine, Department of Internal Medicine, C32 GHStephanie Seremetis - Novo Nordisk, A/SJanice Staber - University of Iowa Carver College of Medicine, Department of PediatricsRoshni Kulkarni - Michigan State University, Department of Pediatrics and Human Development
- Resource Type
- Journal article
- Publication Details
- Expert opinion on orphan drugs, Vol.2(4), pp.419-431
- Publisher
- Taylor & Francis
- DOI
- 10.1517/21678707.2014.891458
- ISSN
- 2167-8707
- eISSN
- 2167-8707
- Language
- English
- Date published
- 04/01/2014
- Academic Unit
- Internal Medicine; Stead Family Department of Pediatrics; Hematology, Oncology, and Blood & Marrow Transplantation; Iowa Neuroscience Institute; Hematology/Oncology
- Record Identifier
- 9984065477802771
Metrics
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