Akcea Therapeutics and Ionis Pharmaceuticals have received positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for the marketing authorisation of tegsedi in the European Union.
The CHMP’s endorsement will now be referred to the European Commission, which will decide on whether to grant the drug authorisation to be marketed in European Union and European Economic Area countries.
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The companies are seeking approval for the drug to treat stage 1 or stage 2 polyneuropathy in adults suffering from rare disease hereditary transthyretin amyloidosis (hATTR).
Tegsedi is an antisense oligonucleotide being developed to decrease transthyretin production, which reduces the formation of amyloids and alleviates hATTR symptoms.
Akcea Therapeutics CEO Paula Soteropoulos said: “Today’s positive CHMP opinion is an important step toward making tegsedi available to people with this systemic, progressive and fatal hereditary disease that relentlessly deprives them of their independence and dignity.
“We are now anticipating approval in Europe shortly and we are ready to launch tegsedi to bring this new treatment to people with hATTR amyloidosis.”
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By GlobalDataThe company supported its marketing authorisation application with findings from the Phase III NEURO-TTR clinical trial and an ongoing open label extension study conducted in hATTR amyloidosis patients with polyneuropathy symptoms.
Subjects experienced substantial decrease in TTR protein levels, which had clinically relevant effects on neurological manifestations and their quality of life.
The most frequent adverse events were related to reactions at the injection site.
Previously, the drug gained orphan drug designation from the EMA, as well as from the US Food and Drug Administration (FDA), which also granted it fast track status.
