The British global leader in rare diseases said lyophilised ONCASPAR had been approved as a component of antineoplastic combination therapy in acute lymphoblastic leukaemia (ALL) in paediatric patients from birth to 18 years, and in adult patients.
The move permits Shire to commercialise lyophilised ONCASPAR in the 28 member states of the European Union, as well as Iceland, Liechtenstein and Norway.
With this lyophilised formulation, we aim to make pegylated asparaginase, part of the paediatric standard therapy in acute lymphoblastic leukaemia, available to patients in countries where liquid ONCASPAR is not currently offered.
The positive opinion recommends the marketing authorisation of lyophilised ONCASPAR as a component in antineoplastic combination therapy for acute lymphoblastic leukemia (ALL) in paediatric patients from birth to 18 years, and in adult patients.
Lyophilised ONCASPAR is a freeze-dried formulation of ONCASPAR.
The company enrolled a total of 24 healthy male subjects in the single-centre, open-label, randomised, single dose cross-over Phase I trial that assessed the bioequivalence and tolerability of liquid and reconstituted lyophilised
formulations of caplacizumab administered subcutaneously.