(19)

(11)

EP 2 804 600 A2

(12)

(88) Date of publication A3: 25.07.2013 (43) Date of publication: 26.11.2014 Bulletin 2014/48 (21) Application number: 13739040.7 (22) Date of filing: 18.01.2013

(51) International Patent Classification (IPC):  A61K 31/196(2006.01) A61K 31/27(2006.01) A61K 9/22(2006.01) A61K 31/195(2006.01) A61K 31/16(2006.01) A61K 9/20(2006.01) (86) International application number: PCT/US2013/022167 (87) International publication number: WO 2013/109906 (25.07.2013 Gazette 2013/30)

(84) Designated Contracting States: AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR (30) Priority: 18.01.2012 US 201261588069 P 04.10.2012 US 201261709549 P (71) Applicant: Ultragenyx Pharmaceutical Inc. Novato, CA 94949 (US)	(72) Inventor: KAKKIS, Emil D. Novato, California 94945 (US) (74) Representative: Wills, Andrew Jonathan et al Mewburn Ellis LLP 33 Gutter Lane London EC2V 8AS London EC2V 8AS (GB)