European regulators have approved Akcea and Ionis’ Tegsedi for patients with hereditary transthyretin amyloidosis (hATTR), a rare disease causing build up of amyloid in the peripheral nervous system and multiple organs.

The progressive accumulation of TTR amyloid deposits in tissues and organs leads to sensory, motor and autonomic dysfunction often having debilitating effects - causing loss of sensation in limbs, weight loss and chest.

hATTR can result in death within three to fifteen years of symptom onset, and currently there are no disease-modifying drugs approved to treat the condition, which affects an estimated 50,000 patients worldwide.

Tegsedi (inotersen) is designed to block production of the TTR protein, and in clinical trials (NEURO-TTR) treatment with the drug produced substantial reductions in the levels of the protein regardless of mutation type or stage of disease, the firms noted.

EU approval enables doctors to prescribe the drug to treat stage 1 or stage 2 polyneuropathy (damage to peripheral nerves) in patients with the disease.

“With the EC’s decision, inotersen is now the world’s first and only RNA-targeted therapeutic approved for patients with hATTR amyloidosis,” said Paula Soteropoulos, Akcea’s chief executive.

“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.”

“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,” added 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.”