PTC in-licenses rights to two rare disease drug for Latin America

PTC Therapeutics (Nasdaq: PTC) and Akcea Therapeutics (Nasdaq: AKCA), an affiliate of Ionis Pharmaceuticals (Nasdaq: IONS), today announced a collaboration under which PTC will commercialize two of Akcea's rare disease drugs in Latin America: Tegsedi (inotersen) and Waylivra (volanesorsen).

Tegsedi has received marketing authorization approval from the European Commission for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR amyloidosis). Tegsedi is also the subject of a pending new drug application in the US and Canada. Tegsedi has a Prescription Drug User Fee Act (PDUFA) date of October 6, 2018.

Waylivra is under regulatory review in the USA, Europe and Canada for the treatment of people with familial chylomicronemia syndrome (FCS). Waylivra recently received a positive vote from the Food and Drug Administration's Division of Metabolism and Endocrinology Products Advisory Committee and has a PDUFA date of August 30, 2018. Waylivra is also in clinical development for familial partial lipodystrophy, or FPL.