Onpattro (patisiran) lipid complex injection 10mg/5ml

Onpattro (patisiran) is a lipid complex injection used to treat hereditary transthyretin-mediated (hATTR) amyloidosis in adults. This condition is characterized by the accumulation of amyloid proteins in various organs and tissues, leading to a wide range of debilitating symptoms. Onpattro is an innovative RNA interference (RNAi) therapeutic that targets the root cause of the disease, offering a novel approach to treatment. How It Works Onpattro (patisiran) employs RNA interference (RNAi) technology to reduce the production of transthyretin (TTR) protein. In patients with hATTR amyloidosis, mutations in the TTR gene lead to the production of misfolded TTR proteins, which aggregate and form amyloid deposits in tissues throughout the body. These deposits cause progressive organ damage, primarily affecting the nervous system and the heart. Targeting mRNA: Patisiran specifically targets and binds to the messenger RNA (mRNA) that encodes the TTR protein. By doing so, it prevents the mRNA from being translated into the TTR protein, effectively reducing the levels of both wild-type and mutant TTR proteins in the bloodstream. Lipid Nanoparticle Delivery System: Patisiran is encapsulated in a lipid nanoparticle, which facilitates its delivery to the liver—the primary site of TTR protein synthesis. This delivery system ensures that patisiran reaches its target effectively, allowing it to exert its therapeutic action. Reduction of Amyloid Deposits: By lowering TTR protein levels, Onpattro reduces the formation of amyloid deposits, thereby alleviating symptoms, slowing disease progression, and improving the quality of life for patients. Clinical Efficacy Clinical trials have demonstrated that Onpattro significantly reduces serum TTR levels and amyloid deposits, leading to improvements in neurological function, quality of life, and overall survival in patients with hATTR amyloidosis. Its ability to target the underlying genetic cause of the disease makes it a powerful therapeutic option for this rare and progressive condition. Side Effects While Onpattro offers significant benefits, it is associated with various side effects that patients and healthcare providers should be aware of. Side effects can range from mild to severe and may vary from person to person. Common Side Effects Infusion-related reactions: These can occur during or shortly after the administration of Onpattro and may include:

• Flushing
• Back pain
• Nausea
• Abdominal pain
• Fatigue
• Headache
• Chills
• Dizziness
• Dyspnea
• Upper respiratory tract infections: Common colds, nasal congestion, and sore throat.

Gastrointestinal symptoms: Nausea, diarrhea, vomiting, and abdominal pain. Serious Side Effects Peripheral edema: Swelling of the legs, ankles, or feet due to fluid retention. Cardiac issues: Some patients may experience worsening of heart-related symptoms, especially those with existing cardiac conditions. Hepatic effects: Elevated liver enzymes, which may indicate liver inflammation or damage. Hypersensitivity reactions: Serious allergic reactions that require immediate medical attention. Patients experiencing severe side effects or signs of an allergic reaction (such as difficulty breathing, swelling of the face or throat, or severe rash) should seek emergency medical attention immediately. Indications Onpattro is specifically indicated for the treatment of polyneuropathy caused by hereditary transthyretin-mediated amyloidosis in adult patients. Approved Indications Hereditary Transthyretin-Mediated Amyloidosis (hATTR): Onpattro is used to treat polyneuropathy of hATTR amyloidosis, a genetic condition characterized by the buildup of amyloid deposits in the peripheral nerves, heart, and other organs. Polyneuropathy: This condition involves damage to multiple peripheral nerves, resulting in symptoms such as: By addressing the underlying cause of TTR amyloid formation, Onpattro helps manage and alleviate these symptoms, improving patients’ overall quality of life. Contraindications Hypersensitivity: Patients with known hypersensitivity to patisiran or any of the excipients in Onpattro should not receive this medication. Hypersensitivity reactions may include anaphylaxis or severe allergic reactions. Severe Liver Impairment: Onpattro is primarily metabolized by the liver. Patients with severe liver impairment may not process the medication effectively, leading to increased risk of side effects or toxicity. Pregnancy and Breastfeeding: The effects of Onpattro during pregnancy and breastfeeding are not well established. Therefore, it should be used with caution in pregnant or breastfeeding women, only if the potential benefit justifies the potential risk to the fetus or infant. Cardiac Disease: Patients with advanced cardiac amyloidosis should be monitored closely during treatment, as Onpattro may exacerbate cardiac symptoms. Price in Different Countries The cost of Onpattro can vary widely depending on the country, healthcare system, and insurance coverage. The table below provides an overview of the approximate price of Onpattro in various countries.

Country	Price (USD) Per 10 mg/5 ml	Reference Link
United States	$15,000 – $17,000	Drugs.com
Canada	$13,000 – $16,000	Health Canada
United Kingdom	£11,500 – £14,000	NHS
Australia	$14,500 – $16,500	PBS
Germany	€13,000 – €15,500	Apotheken Umschau

  Top 5 Global Brands Onpattro is part of the growing field of RNA interference therapeutics, and several global pharmaceutical companies are involved in developing similar treatments. Here are the top 5 global brands known for their contributions to RNAi-based therapies: Alnylam Pharmaceuticals: The developer of Onpattro and a leader in RNAi therapeutics, with a strong focus on innovative treatments for genetic diseases. Ionis Pharmaceuticals: A pioneer in RNA-targeted drug discovery and development, with a robust pipeline of RNA-based therapies for various diseases. Novartis: A global pharmaceutical company known for its research and development in innovative medicines, including RNA-based therapies. Pfizer: Engaged in the development of RNA-based treatments and vaccines, Pfizer is a key player in the field of biopharmaceuticals. Regeneron Pharmaceuticals: Known for its focus on innovative genetic medicines, including RNAi and gene-editing technologies.