Recently, the US FDA announced the approval of Onpattro (patisiran) infusion therapy for adult patients with peripheral neuropathy (polyneuropathy) caused by hereditary transthyretin amyloidosis (hATTR). It is worth mentioning that this is the first drug developed in the world based on the Nobel Prize-winning RNA interference technology. It is the first FDA-approved therapy for patients with polyneuropathy caused by hATTR. It is also the first FDA approved. Small interfering RNA (siRNA) drugs.
It is known that hATTR is a rare, debilitating and often fatal hereditary disease. Its main feature is the formation of abnormal deposits of protein fibers called amyloid in organs and tissues in the body, which interfere with the normal organs and tissues. Functionally, these protein deposits most often occur in the peripheral nervous system, causing loss of sensation, pain or inability to move arms, legs, hands and feet. Amyloid deposits also affect the function of the heart, kidneys, eyes, and gastrointestinal tract. Current treatments often focus on symptom management, and there is a need for medical needs in this area.
Developed by Alnylam, Onpattro is a siRNA therapy targeting transthyretin (TTR), which works by silencing a portion of the RNA involved in pathogenesis. Specifically, Onpattro encapsulates siRNA in lipids. In the nanoparticle, the drug is delivered directly to the liver during infusion therapy, interfering with RNA production from the abnormal form of TTR. By preventing the production of TTR, Onpattro can help reduce the accumulation of amyloid deposits in the peripheral nerves, improve symptoms, and help patients better control their condition. Previously, Onpattro received FDA-approved breakthrough therapy certification, priority review qualifications, fast-track qualifications, and orphan drug qualifications.
The efficacy of Onpattro has also been confirmed in clinical trials. In a study of 225 patients, 148 patients were randomized to receive Onpattro infusions every three weeks for a total of 18 months, and another 77 patients were randomized to receive the same frequency of placebo infusion. The results showed that patients receiving Onpattro had better outcomes in measuring multiple neuropathy, including muscle strength, sensation (pain, body temperature, numbness), reflexes, and autonomic symptoms compared with patients receiving placebo infusion. (blood pressure, heart rate, digestion). Patients who received Onpattro also had higher scores in their assessment of walking, nutritional status, and ability to perform daily activities.
It is also known that according to the prediction of the Clinical and Economic Review Institute, the average cost of the Patisiran drug may be $450,000/year, at least in six digits. The assessment also indicates that if the drug price is less than $300,000, it may not be possible. Benefits (assessment of the lowest discount price of about $345,000), however, Alnylam expressed its willingness to discuss with the medical insurance company to establish a reimbursement mechanism based on clinical efficacy, if the drug has no clinical effect on a patient, then Full refund.
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