It’s been quite a year for Akcea Therapeutics (AKCA). After going public in July 2017, the Cambridge, Mass., company almost immediately filed for FDA approval on two drugs for rare diseases.
Volanesorsen, also known as Waylivra, is intended to treat familial chylomicronemia syndrome (FCS), a condition that leads to a buildup of fat in organs. Inotersen, which has the brand name Tegsedi, treats hereditary transthyretin amyloidosis, known as hATTR for short. The condition leads to a buildup of proteins that can cause issues in the nerves and heart. European authorities have already approved inotersen, and the FDA is set to decide on both those drugs before the end of the year.