Diamond sparkles for US MedTech
Diamond Pharma Services in Harlow, a leading technical services and regulatory affairs consulting group, provided EU regulatory support to American company Akcea Therapeutics in a key transaction.
It resulted 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.
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 was grateful for the opportunity to assist with the European Regulatory support.
“To help Akcea 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.”