The MHRA has approved eplontersen (Wainzua) for treating adults with polyneuropathy associated with hereditary transthyretin amyloidosis (ATTRv), a rare genetic condition caused by the transthyretin protein
The condition causes tiny fibres of TTR protein to clump together to make deposits called ‘amyloid’. Amyloids can build up around or within the nerves, heart, and other organs, stopping them from working correctly.
Eplontersen works by lowering the amount of TTR protein made by the liver, meaning there is less TTR protein in the blood to form amyloid deposits.
Enabling safe access to effective medicines
Eplontersen is recommended for individuals whose disease is causing polyneuropathy, which is damage to multiple nerves outside of the brain and central nervous system, resulting in pain, discomfort, progressive weakness and loss of sensation in the legs and arms, and mobility difficulties.
The recommended dose is one 45mg dose every month, administered as an injection under the skin using a pre-filled pen.
Julian Beach, MHRA Interim Executive Director of Healthcare Quality and Access, said: “Enabling safe access to high quality, safe and effective medicines is a key priority for us.
“We’re assured that the appropriate regulatory standards of safety, quality and effectiveness for the approval of this new formulation have been met.
“As with all products, we will keep its safety under close review.”
Eplontersen helps reduce symptoms of the disease
The NEURO-TTRansform study showcased the efficiency of the medicine. In this trial, 164 adults with hereditary transthyretin-mediated amyloidosis were treated with a 45 mg subcutaneous injection of eplontersen every 4 weeks. The results were compared to data from a historical placebo group in the 2017 NEURO-TTR study and to patients receiving the medication inotersen at a dose of 284 mg weekly.
The study looked at TTR levels in the patient’s blood to measure the protein levels contributing to the disease. It used questionnaires to rate symptoms of nerve damage. The study also tracked these changes from the start of the trial to 35 and 66 weeks after the patients had received eplontersen.
The trial showed that patients receiving the medicine experienced more significant reductions in TTR levels and less disease worsening from baseline compared to the placebo group.
Sami Khella, M.D., Chief of the Department of Neurology at Penn Presbyterian Medical Center, Professor of Clinical Neurology at the Perelman School of Medicine at the University of Pennsylvania School of Medicine and a Principal Investigator on the NEURO-TTRansform trial, said: “The totality of positive, consistent eplontersen data-position this therapy, which can be self-administered, to be an important and empowering potential new medicine for treating hereditary transthyretin-mediated amyloid polyneuropathy.
“Without treatment, hereditary transthyretin-mediated amyloid polyneuropathy is a debilitating and devasting disease that can ultimately result in death. The JAMA publication reinforces the growing evidence showing that eplontersen significantly reduces serum transthyretin concentration, may halt the progression of neuropathy impairment, and improves overall patient quality of life, providing hope to this community.”
Sharon Barr, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: “Eplontersen demonstrated improvements in a wide array of disease-related endpoints across transthyretin-mediated amyloid polyneuropathy in patients with a diverse range of disease characteristics. Publication of these positive results provides evidence of eplontersen’s ability to achieve consistent and sustained benefit in this patient population and its potential to be an important treatment option across all types of transthyretin-mediated amyloidosis.”
