Rare Daily Staff

The U.K.’s Medicines & Healthcare products Regulatory Agency said it would allow patients with the rare genetic lipid disorder familial chylomicronaemia syndrome access to Akcea Therapeutics’ volanesorsen ahead of the European Commission’s issuing a formal decision for the drug’s use in Europe.

FCS is a rare metabolic disorder where patients have an enzyme deficiency that results in high levels of triglycerides in their blood. The condition causes symptoms including fatigue, abdominal pain, and the risk of recurrent, potentially fatal attacks of pancreatitis.

Volanesorsen is was developed on Ionis Pharmaceutical’s antisense platform and is designed to reduce the production of ApoC-III, a protein produced in the liver that plays a central role in the regulation of plasma triglycerides and may also affect other metabolic parameters.

The MHRA decision came through its Early Access to Medicines Scheme, a program that provides early availability to innovative and new unlicensed medicines to UK patients with a high unmet clinical need. The medicines approved for the scheme are intended to treat, diagnose or prevent seriously debilitating or life-threatening conditions where no adequate treatment options exist.

“With no current treatment options for people living with this debilitating disease, this will enable them to access and benefit from a new innovative therapy to help address a clear unmet need,” said Luke Robinson, General Manager, Akcea Therapeutics, UK, Ireland & Nordics.

March 21, 2018
Photo: Luke Robinson, General Manager, Akcea Therapeutics, UK, Ireland & Nordics