Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for rare genetic diseases has announced that its Phase 3 study of a potential therapy for Glucose transporter 1 deficiency syndrome (Glut1 DS) did not produce a positive result. Glut1 DS is a condition which results in seizures, developmental delay as well as ataxia. See Ataxia UK’s Medical Guidelines here

“We had previously observed significant improvements in individual cases of Glut1 DS with UX007 and so we are particularly disappointed by the results of the Glut1 DS study in a larger group of patients that did not demonstrate this same effect” said Emil Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx.

Development of UX007 in Glut 1 DS will therefore unfortunately be discontinued, however it will continue to be investigated for other, similar disorders. For the full press release see here

Posted on 27/11/2018