A multi-national, open-label phase II study of 26 patients with Gaucher disease type 1 to evaluate the efficacy, pharmacokinetics and safety of eliglustat tartrate as an oral therapy has yielded positive results.
The drug decreases the production of fatty substances from the body to avoid their accumulation in cells, according to a study published in Blood, the official journal of The American Society of Hematology.
The study endpoint had been met by 77% of all patients and 91% of the 22 patients who completed the full 52-week trial, with overall improvements seen in haemoglobin levels and spleen volume.
The research team found that patients’ glucosylceramide plasma levels normalised, disease symptoms responded rapidly, and saw improvements in bone mineral density.
A controlled Phase III study in untreated patients and in-patients previously stabilised with imiglucerase– an intravenously administered medication currently used to treat Gaucher disease – is in progress.
Russian Academy of Medical Sciences’ Hematology Research Center MD and lead study author Elena Lukina said an oral alternative expected to treat the disease would be viable.