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Rare Lipid Disorder Drug To Reach Patients Faster Under UK Early Access Scheme

 
• By Neena Brizmohun

The UK drug regulator has granted volanesorsen a positive scientific opinion under its early access to medicines scheme for use in patients with familial chylomicronemia syndrome. The drug’s sponsor, Akcea Therapeutics, is hoping to get the drug approved in the EU, the US and Canada this year.

Vaccination
Akcea's volanesorsen is injected subcutaneously to treat the lipid disorder FCS

Akcea Therapeutics has received permission to make its unlicensed treatment for familial chylomicronemia syndrome (FCS) available to patients in the UK under the government’s early access to medicines scheme (EAMS) while the product undergoes evaluation for marketing throughout the EU.

Volanesorsen, an EU orphan designated drug for treating patients with the rare lipid disorder, was granted a positive scientific opinion under EAMS by the Medicines and Healthcare products Regulatory...

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