Akcea’s rare lipid disorder drug wins PIM status

Akcea Therapeutics’s volanesorsen has been granted a Promising Innovative Medicine (PIM) designation by the Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of familial chylomicronaemia syndrome (FCS), a rare genetic lipid disorder.

This move signifies that the regulator believes the drug is a potential candidate for the UK’s Early Access to Medicines Scheme, which allows patients with life-threatening and seriously debilitating conditions to be treated with innovative therapies before regulatory approval is obtained.

In the second stage of the process, the Agency will issue an EAMS Scientific Opinion if the quality, safety and efficacy data provided in support of the application is sufficient to support a positive benefit/risk balance and added clinical value, paving the way for patient access.

Medicines in the EAMS will typically be commissioned by NHS England through its specialised commissioning arrangements, but funded by pharmaceutical companies, enabling patients to benefit from the latest treatment breakthroughs at no cost to the NHS.

FCS is a severe, rare disorder characterised by extremely elevated levels of triglycerides, symptoms such as abdominal pain and the risk of recurrent, potentially fatal, acute pancreatitis. People with FCS are unable to effectively metabolise large, triglyceride-rich, lipid particles called chylomicrons due to a deficiency in lipoprotein lipase, an enzyme that helps to break down triglycerides.

"We are delighted about the MHRA's decision to designate volanesorsen as a PIM. This designation demonstrates the potential to address clear unmet medical needs in the treatment of this debilitating condition," said Luke Robinson, general manager of Akcea Therapeutics, UK, Ireland & Nordics.

"FCS is a significant burden on people affected by the condition," added Louis O'Dea, chief medical officer of the firm. "FCS patients have a high unmet need with potentially life-threatening consequences, and we seek to bring this new treatment to these patients as expeditiously as possible."