The first treatment for a rare disease characterised by high levels of triglycerides in blood is among eight new medicines put forward by the European Medicines Agency’a human medicines committee (CHMP) for approval.
A conditional marketing authorisation was recommended for Akcea Therapeutics’ Waylivra (volanesorsen), the first medicine for the treatment of the familial chylomicronaemia syndrome (FCS), a rare genetic disease that prevents the body from breaking down fats (lipids).
Patients with the condition have extremely high levels of triglycerides in their blood that can cause a range of symptoms, including severe abdominal pain, potentially fatal attacks of acute pancreatitis, hepatosplenomegaly, diabetes, lack of concentration, memory loss and fat-filled spots on the skin (called xanthomas).
There is currently no authorised medicine available to treat this rare disease, with current treatment approaches focusing on a strict diet with extremely limited fat intake.
The Agency highlighted that existing lipid-lowering medications are only “minimally effective” to reduce triglyceride levels in patients with FCS, and there is “an urgent unmet medical need” for new treatments for the disease.
Elsewhere, conditional marketing authorisations were also recommended for Portola Netherlands’ Ondexxya (andexanet alfa), as the first antidote for adults taking the anticoagulants apixaban or rivaroxaban, for life-threatening or uncontrolled bleeding, and Pfizer’s Lorviqua (lorlatinib) for the treatment of patients with anaplastic lymphoma kinase (ALK)-positive advanced non-small cell lung cancer (NSCLC).
A positive opinion was also given for: BioMarin International’s Palynziq (pegvaliase), a new medicine for patients aged 16 and older with phenylketonuria, a rare but potentially serious inherited metabolic disease; AbbVie’s Skyrizi (risankizumab) for moderate to severe psoriasis; Sanofi’s Zynquista (sotagliflozin) for certain patients with type I diabetes; and GlaxoSmithKline’s Dectova (zanamivir) – under exceptional circumstances – for the treatment of complicated and potentially life-threatening influenza.
The Committee also backed clearance of Teva’s generic Pazenir (paclitaxel), for the treatment of metastatic breast cancer and non-small cell lung cancer.
The February meeting was the last to take place in London; as of March 2019, all CHMP meetings will take place at the new EMA headquarters in Amsterdam.
