CHMP adopts positive opinion for orphan drug to treat rare hereditary disease

Akcea Therapeutics, has announced that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion, recommending the use of its orphan drug for the treatment of Stage 1 or Stage 2 polyneuropathy in adults with hereditary transthyretin (hATTR) amyloidosis.

The fatal hereditary disease, hATTR amyloidosis, is caused by abnormal formation and build-up of TTR amyloid deposits in the body, which leads to symptoms such as loss of sensation in the limbs, chest pain, palpitations, weight loss, nausea, weakness and fatigue. Usually, people with hATTR amyloidosis die within three to 15 years after developing symptoms.

Akcea Therapeutics’ medicine, Tegsedi (inotersen), is an ‘antisense oligonucleotide’, a very short piece of synthetic DNA designed to attach to the genetic material of the cells responsible for producing the transthyretin protein. Through this mechanism it is expected to decrease transthyretin production, thereby reducing the formation of amyloids and relieving the symptoms of hATTR. As such, the therapy aims to affect the course of the disease and improve quality of life.

This recent positive opinion has been based on results from the Phase III NEURO-TTR study and open label extension (OLE) study in patients with hATTR amyloidosis with symptoms of polyneuropathy. The results demonstrated a significant benefit to patients administered with inotersen when compared with those taking a placebo for both co-primary endpoints.

“We are delighted at this positive opinion, which is an important milestone for people suffering with this disease. Patients currently have no treatment options and the prognosis is potentially devastating,” explained Luke Robinson, general manager, Akcea Therapeutics, UK, Ireland & Nordics. “This encouraging announcement from CHMP offers hope to these patients and their families, who may soon have access to an innovative therapy that can help improve their lives.”

Currently, therapeutic options for patients with hATTR include liver transplant, treatment with tafamidis and off-label use of a non-steroidal anti-inflammatory drug (NSAID). Each of these options have significant limitations for patients with stage 2 or 3 polyneuropathy and as such indicates a clear unmet medical need. In light of this fact, the CHMP considered Tegsedi a medicine of major interest for public health and agreed to the applicant’s request for an accelerated assessment of the medicine.

It was designated as an orphan medicine in 2014 as hATTR is a rare diseases that is diagnosed in an estimated three cases per 10 million people. A review of the orphan designation will be performed by the European Medicines Agency’s (EMA’s) Committee for Orphan Medicinal Products (COMP) at the time of the approval to determine if it is still valid and granting the medicine 10 years of market exclusivity.

The CHMP opinion will be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation.