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For the treatment of hereditary amyloid transthyretin (hATTR), Ema Chmp has given the nod to the approval of patisiran, an experimental therapy that acts through the mechanism of RNA interference . The drug received a positive opinion for use in adult patients with polyneuropathy grade 1 and 2.
For the treatment of hereditary transthyretin amyloidosis (hATTR), Ema Chmp gave the green light to the approval of patisiran, an experimental therapy that acts through the mechanism of RNA interference. The drug received a positive opinion for use in adult patients with polyneuropathy grade 1 and 2
A patisiran had already been granted an accelerated evaluation by Ema, which reduced the evaluation time of the European Agency of 210 to 150 days
Developed by the American biotech Alnylam, the definitively approved drug will be marketed under the brand name Onpattro
The positive opinion of the CHMP is based on data from the study of phase. 3 of APOLLO published on July 5 in the New England Journal of Medicine.
Patients were randomized in a ratio of 2: 1 to receive intravenous treatment (0.3 mg per kilogram of body weight) or placebo once every 3 weeks for 18 months. The study showed that patients improved measures of polyneuropathy, quality of life, activities of daily living, walking, nutritional status and autonomic symptoms in adult patients with high blood pressure. amyloidosis hATTR. The APOLLO study used the Neuropathy Impairment Score +7 (mNIS + 7) to badess motor strength, reflexes, sensation, nerve conduction, and postural blood pressure.
How patisiran works
Patisiran is a transthyretin-based (RTI) -retreated intravenous (RTI) therapy. It is designed to target and silence specific messenger RNA by blocking the production of TTR proteins prior to its formation. This can help eliminate TTR amyloid deposits in peripheral tissues and potentially restore the function of these tissues.
What is amyloidosis of hereditary accumulation of transthyretin
Hereditary TTR-mediated amyloidosis (hATTR) is a hereditary disease, gradually debilitating and often fatal, caused by mutations of the TTR gene. TTR protein is mainly produced in the liver and is normally a vitamin A transporter. Mutations in the TTR gene cause an abnormal accumulation of amyloid proteins that damage organs and tissues of the body, such as peripheral nerves and the heart, causing sensory neuropathy Peripheral neuropathy, autonomic and / or incurable cardiomyopathy.
HATTR amyloidosis represents a significant unmet clinical need, with significant morbidity and mortality, affecting approximately 50,000 people worldwide. The life expectancy of patients with hATTR amyloidosis ranges from 2.5 to 15 years after onset of symptoms and the only approved treatment options for the initial disease are liver transplantation and tafamidis (approved in Europe, Japan). and some countries in Latin America, the specific indication varies by region). Therefore, there is a significant need for new therapies to help treat patients with HATTR amyloidosis.
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