Akcea Therapeutics and Ionis Pharmaceuticals have announced that Tegsedi (inotersen) has received European marketing authorisation approval for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).
approval
This decision by the European Commission (EC) has followed the positive opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), which recommended the approval earlier this year.
“With the EC’s decision, inotersen is now the world’s first and only RNA-targeted therapeutic approved for patients with hATTR amyloidosis. With subcutaneous delivery, inotersen puts treatment in the patients’ hands while bringing the significant benefits shown in the NEURO-TTR study in both measures of neuropathy and quality of life for people living with this serious and fatal disease. This is an important day for the hATTR amyloidosis community as we believe inotersen enables people and their families impacted by this disease to move forward with their lives,” said Paula Soteropoulos, chief executive officer at Akcea Therapeutics. “Today is a milestone for Akcea with our first drug approval. It is an achievement we share with the courageous hATTR patient community in Europe and around the globe. We are ready to launch inotersen along with our patient and physician support services across Europe.”
The underlying cause of hATTR amyloidosis is the abnormal formation and aggregation of transthyretin (TTR) protein giving rise to TTR amyloid deposits throughout the body. In the Phase III NEURO-TTR study — which is what the EC based its approval on — substantial reductions in the TTR protein levels were seen after treatment with inotersen, regardless of mutation type or stage of disease.
“hATTR amyloidosis is an inherited, progressive and fatal disease for which treatment options are limited. The approval of inotersen brings us into a new era of treatment with an efficacious and disease modifying medicine that potentially allows patients to achieve a greater degree of independence,” explained Dr Teresa Coelho, neurologist and neurophysiologist at Santo António Hospital, Porto, Portugal. “Inotersen has demonstrated rapid and sustained benefits in improving the course of this disease and preserving quality of life.”
“Today, we are thrilled to see our successful research and development efforts result in the approval of an important new medicine for patients with hATTR amyloidosis. Using our antisense technology platform, we set out to design a therapy to block the production of the underlying cause of this disease, the TTR protein,” added Brett P. Monia, PhD, chief operating officer at Ionis Pharmaceuticals. “Approval of inotersen further establishes Ionis as a multi-product company. We are confident that the experienced team at Akcea will deliver on the promise of inotersen. We are grateful to all of the physicians and patients who participated in the inotersen clinical programme and who made this landmark approval possible.”