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Program broadens pre-approval access to volanesorsen
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Akcea Therapeutics announced its global early access program for volanesorsen, an investigational drug for treatment of patients with familial chylomicronemia syndrome, has been expanded.
Volanesorsen, which is in development for treatment of the rare lipid disorder familial chylomicronemia syndrome (FCS) by reducing the production of apolipoprotein C-III, is under regulatory review in the U.S, Europe and Canada, and would be the first and only therapy indicated for people with FCS if approved, according to the release.
Early access programs, also called compassionate use, make available outside of clinical trials an investigational medicinal product that is intended to treat a serious or life-threatening condition. Patients with FCS are at risk for fatal acute pancreatitis, according to the release.
“Our work is driven by a steadfast commitment to the FCS community, and we are pleased to make volanesorsen available through our global early access program to people who may benefit while our regulatory applications are being reviewed,” Paula Soteropoulos, CEO of Akcea Therapeutics, said in the release. “FCS is a serious condition with a variety of symptoms, including potentially fatal acute pancreatitis, that can severely impact quality of life. As there are no currently approved treatments for FCS, we are working closely with lipid specialists to responsibly enable patient access.”
Physicians can email volanesorsenEAP@akceatx.com to learn more about the program.
Disclosure: Soteropoulos is an employee of Akcea Therapeutics.