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Diamond Pharma Services Supports Akcea Therapeutics in Gaining EU Regulatory Approval for WAYLIVRA Treatment for Familial Chylomicronaemia Syndrome (FCS)

March 25, 2019

HARLOW, England--(BUSINESS WIRE)--Mar 25, 2019--Diamond Pharma Services (“Diamond”), a leading technical services and regulatory affairs consulting group, has announced that it provided EU regulatory support to Akcea Therapeutics Ltd (Akcea), resulting in a positive Committee for Medicinal Products Human Use (CHMP) opinion for the grant of a Conditional Marketing Authorisation for WAYLIVRA Solution for Injection, the first authorised medicine for the treatment of FCS.

Headquartered in Boston, MA, Akcea is a development and commercialisation company focused on bringing transformative medicines to patients with serious and rare diseases. Since 2016, Diamond provided full and comprehensive EU Regulatory support for WAYLIVRA to Akcea, leading up to and through the Conditional Approval.

Akcea Therapeutics Ltd obtained a positive CHMP opinion for WAYLIVRA on 28 th February 2019, which was also Rare Disease Day.

FCS is a rare genetic disease that prevents the body from breaking down fats, causing extremely high levels of triglycerides in the blood. Prior to WAYLIVRA approval there was no authorised medicine available to treat FCS. Patients rely on limiting their fat intake through diet and triglyceride lowering therapy, but this is not always feasible and sufficiently effective to prevent pancreatitis and other symptoms of FCS, which include severe abdominal pain, hepatosplenomegaly, diabetes, lack of concentration, memory loss and fat-filled spots on the skin (called xanthomas).

WAYLIVRA is indicated as an adjunct to diet in adult patients with genetically confirmed FCS and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate. It will be available as a 285mg solution for injection and offers the ability to reduce levels of fasting triglycerides by inhibiting the formation of apolipoprotein C-III (apoC-III), a protein that regulates both triglyceride metabolism and hepatic clearance of chylomicrons and other triglyceride-rich lipoproteins.

Maureen Graham, Managing Director, Regulatory, Diamond Pharma Services, said: “The Conditional Marketing Authorisation for WAYLIVRA is a major achievement for Akcea, and Diamond were grateful for the opportunity to assist with the European Regulatory support. To help Akcea to bring a product to the market which will help patients who suffer from this condition, and currently have no therapeutic options is tremendous. For Diamond, as well, it has continued our trend of supporting products that are ‘firsts’. WAYLIVRA is the first and only therapy approved for FCS.”

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CONTACT: Lorna Cuddon

Zyme Communications

Tel: +44 7811 996 942

E-mail:lorna.cuddon@zymecommunications.com

KEYWORD: UNITED KINGDOM EUROPE

INDUSTRY KEYWORD: HEALTH CLINICAL TRIALS GENETICS PHARMACEUTICAL RESEARCH SCIENCE

SOURCE: Diamond Pharma Services

Copyright Business Wire 2019.

PUB: 03/25/2019 05:30 AM/DISC: 03/25/2019 05:31 AM

http://www.businesswire.com/news/home/20190325005278/en