Akcea and Ionis’ Waylivra nets CHMP approval in ultra-rare familial chylomicronaemia syndrome

pharmafile | March 5, 2019 | News story | Manufacturing and Production, Sales and Marketing Akcea, FCS, Ionis, familial chylomicronemia syndrome, pharma, rare disease 

Akcea Therapeutics and Ionis Pharmaceuticals have announced that their investigational therapy Waylivra (volanesorsen) has been recommended by the EMA’s Committee for Medicinal Products for Human Use (CHMP) for conditional marketing authorisation as a supplement to diet in genetically confirmed familial chylomicronaemia syndrome (FCS) in adult patients who have not responded adequately to diet and triglyceride lowering therapy and who are at high risk of pancreatitis.

An ultra-rare condition, FCS is caused by impaired function of the enzyme lipoprotein lipase (LPL) and is characterised by hypertriglyceridemia and the risk of unpredictable acute pancreatitis which can lead to the patient’s death.

Waylivra secured the positive opinion on the back of Phase 3 data collected through the largest-ever study into FCS, demonstrating a 77% reduction in triglycerides after three months of treatment compared to placebo.  

 “The FCS community is encouraged by the positive CHMP opinion and we remain very hopeful that people living with FCS will soon have an approved treatment available in the EU,” said Jill Prawer, Founder and Chair of the Familial Lipoprotein Lipase Deficiency (LPLD) Alliance. “Without a treatment, people living with FCS have debilitating daily symptoms as well as the constant fear of pancreatitis. This is a positive step forward for people living with FCS who currently have no treatment options. Patients around the world need and deserve a treatment for FCS.”

Dr Brett P Monia, Chief Operating Officer at Ionis, also remarked: “If approved, volanesorsen will be the third medicine for which we will have received approval in the last two years. Our antisense technology platform continues to deliver many important scientific and medical advances that should support continuing growth. We look forward to continuing to work with the community of patients and the dedicated clinicians and researchers around the world to make this treatment available to patients who need it as quickly as possible.”

Matt Fellows

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