UK grants rare disease patients early access to Akcea candidate

The UK business of Akcea Therapeutics (Nasdaq: AKCA) has received a positive scientific opinion for volanesorsen, from the country’s Early Access to Medicines Scheme (EAMS).

The judgment from the UK’s medicines regulator, the Medicines and Healthcare Products Regulatory Agency (MHRA), means eligible patients will be able to access volanesorsen before the European Medicines Agency formally rules on its regulatory status in Europe.

Akcea is developing volanesorsen for the treatment of familial chylomicronemia syndrome (FCS), a rare genetic lipid disorder.