- New England Journal of Medicine Publishes Results from Pivotal Study of WAYLIVRA in Patients with Familial Chylomicronemia Syndrome
Akcea Therapeutics (AKCA), an affiliate of Ionis Pharmaceuticals (IONS), announced that the final study results from the Phase 3 APPROACH study evaluating Waylivra in patients with familial chylomicronemia syndrome
were published in the August 8 issue of The New England Journal of Medicine, or NEJM.
Pending approval, Arrowhead intends to proceed with AROAPOC31001 (NCT03783377), a Phase 1 single and multiple dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of ARO-APOC3 in adult healthy volunteers, hypertriglyceridemic patients, and patients with familial chylomicronemia syndrome
Familial chylomicronemia syndrome
(FCS) is a rare inherited lipid disorder that poses significant clinical and psychosocial burdens on patients.
An advisory committee of the US Food and Drug Administration (FDA) has voted to support approval of Waylivra (volanesorsen) for the treatment of people with familial chylomicronemia syndrome
(FCS), Akcea Therapeutics Inc (NASDAQ: AKCA) and Ionis Pharmaceuticals Inc (NASDAQ: IONS) revealed on Thursday.
a Study of Volanesorsen (formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome
(APPROACH) and the Cardiovascular Outcomes for People Using Anticoagulation Strategies (COMPASS) trial,  have shown a serum TG reduction of up to 50% with the use of volanesorsen, an apo C3 inhibitor, when used as monotherapy in the management of dyslipidaemia.
Drugs currently in Phase 3 development include volanesorsen, a drug Ionis is developing and plans to commercialize through its wholly owned subsidiary, Akcea Therapeutics, to treat patients with either familial chylomicronemia syndrome
or familial partial lipodystrophy; and IONIS-TTRRx, a drug Ionis is developing with GSK to treat patients with TTR amyloidosis.
Or that my daughter would inspire me to become something I never even dreamed of--a patient advocate for others who might be affected by familial chylomicronemia syndrome
(i) Familial Chylomicronemia Syndrome
with either Lipoprotein Lipase (LPL) Deficiency or Apolipoprotein C II deficiency or
The familial chylomicronemia syndrome
. Endocrinol Metab Clin North Am 1998;27(3):551-567.
Received orphan drug designation from FDA for ARO-APOC3 for the treatment of familial chylomicronemia syndrome
Akcea Theraputics (AKCA), up 3% after a report that a group for patients suffering from the genetic condition familial chylomicronemia syndrome
, or FCS, is circulating a petition asking the FDA to reconsider its decision to reject Akcea 's volanesorsen.