Orphan drug inotersen receives positive CHMP opinion

Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion, recommending approval of its orphan drug Tegsedi™ (inotersen), for the treatment of Stage 1 or Stage 2 polyneuropathy in adults with hereditary transthyretin amyloidosis (hATTR amyloidosis).

hTTAR amyloidosis is a fatal hereditary disease caused by abnormal formation and build-up of TTR amyloid deposits in the body, leading to debilitating symptoms, such as loss of sensation in the limbs, chest pain, palpitations, weight loss, nausea, weakness, fatigue and chest pain. People with hATTR amyloidosis die within three to fifteen years of developing symptoms.

Inotersen aims to affect the course of the disease and improve quality of life.

The positive opinion is based on results from the Phase 3 NEURO-TTR study and Open Label Extension (OLE) study in patients with hATTR amyloidosis with symptoms of polyneuropathy. The results showed that inotersen resulted in significant benefits for patients, compared with placebo on both co-primary endpoints: the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy score, and the modified Neuropathy Impairment Score + 7, a measure of neuropathic disease progression.

Luke Robinson, General Manager, Akcea Therapeutics, UK, Ireland & Nordics said, “We are delighted at this positive opinion. Patients currently have no treatment options and the prognosis is potentially devastating. This offers hope to these patients and their families, who may soon have access to an innovative therapy.”