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NICE has approved a first-order gene neutralization treatment in the fight against hereditary amyloidosis.
Alnylam UK Limited, a company specializing in NIB Interference Therapy (RNAi), is pleased with the decision of the National Institute for Excellence in Health and Care ( NICE) recommending the use of ONPATTRO.®▼ (patisiran) on the NHS in England for the treatment of a life-threatening progressive disease called hereditary amyloidosis mediated by transthyretin (hATTR amyloidosis).
Prior to this decision, there were few treatment options available for patients with hATTR amyloidosis in England. Patisiran provides eligible patients suffering from this disease a treatment option that attacks the underlying cause by reducing the production of an abnormal protein that damages the nerves and organs of the body.
The hATTR amyloidosis is caused by an abnormal deposition and accumulation of a protein called transthyretin (TTR). Patisiran works by using RNAi to block the production of the majority of the TTR protein prior to its manufacture. In 2006, the discovery of RNAi received the Nobel Prize in Physiology or Medicine. In hATTR amyloidosis, the accumulation of TTR protein and the damage it causes to tissues and organs, such as peripheral nerves and the heart, result in patients with neuropathy (Parkinson's disease). nerves) and cardiomyopathy (heart muscle disease).
In the patisiran pivotal clinical trial, APOLLO, 56% of the 225 patients in the hATTR amyloidosis study with polyneuropathy treated with patisiran found an improvement in their neuropathic symptoms compared with 4% of placebo-treated patients. In addition, the intermediate monitoring data presented to the Annual Nerve Peripheral Society Meeting in June 2019, patients on patisiran treatment for a total of 30 to 36 months demonstrated that improvements in neuropathic impairment and quality of life observed with patisiran continued during this period (compared with corresponding reference data from the parent study).
Professor Philip Hawkins, Director of the National Center for Amyloidosis at the Royal Free Hospital in London, said: "Today's decision marks an important step forward in the treatment of a disease that threatens both the lives of patients and families. Patisiran has shown in his main clinical study that he could stop or even improve the potentially debilitating symptoms of this disease in the majority of patients. This means that we now have a real opportunity to preserve the quality of life of eligible patients longer than has been possible until now. Gene silencing is a promising field of medicine and it is encouraging to see this science translate into treatments that could potentially help people with serious diseases such as hATTR amyloidosis. "
Carlos Heras-Palou, spokesman for the British ATTR Association Amyloidosis Patients' Association, said: "Today's announcement could potentially change the lives of British families. affected by this cruel illness, by offering them an option to reduce the burden of symptoms, this can be crippling for many. This disease also has enormous psychological consequences, with many patients fearing that they have pbaded on the defective gene to children or grandchildren. The availability of patisiran is good news and will greatly rebadure today's patients, as well as those who may need treatment in the future. We are grateful to all who have played a role in achieving this positive outcome and we are pleased that many patients can now have the chance to live a fuller life. "
Brendan Martin, general manager of Alnylam Pharmaceuticals for the UK and Ireland, said: "Over the past year, Alnylam and NICE have worked closely together to ensure access to patisiran on the NHS in England. We look forward to today's result, which we hope will now help many people affected by this devastating disease to better control their daily lives. At Alnylam, our goal remains to build on the groundbreaking science of RNAi and turn it into innovative medicines that can improve the lives of patients with serious illnesses. Today is another important step of this trip. "
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