Country: European Union
Language: English
Source: EMA (European Medicines Agency)
patisiran sodium
Alnylam Netherlands B.V.
N07
patisiran
Other nervous system drugs
Amyloidosis, Familial
Onpattro is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy.
Revision: 11
Authorised
2018-08-27
25 B. PACKAGE LEAFLET 26 PACKAGE LEAFLET: INFORMATION FOR THE PATIENT ONPATTRO 2 MG/ML CONCENTRATE FOR SOLUTION FOR INFUSION patisiran READ ALL OF THIS LEAFLET CAREFULLY BEFORE YOU ARE GIVEN THIS MEDICINE BECAUSE IT CONTAINS IMPORTANT INFORMATION FOR YOU. • Keep this leaflet. You may need to read it again. • If you have any further questions, ask your doctor or nurse. • If you get any side effects, talk to your doctor or nurse. This includes any possible side effects not listed in this leaflet. See section 4. WHAT IS IN THIS LEAFLET: 1. What Onpattro is and what it is used for 2. What you need to know before you are given Onpattro 3. How Onpattro is given 4. Possible side effects 5. How to store Onpattro 6. Contents of the pack and other information 1. WHAT ONPATTRO IS AND WHAT IT IS USED FOR The active substance in Onpattro is patisiran. Onpattro is a medicine that treats an illness which runs in families called hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis). hATTR amyloidosis is caused by problems with a protein in the body called ‘transthyretin’ (TTR). • This protein is made mostly in the liver and carries vitamin A and other substances around the body. • In people with this illness, abnormally shaped TTR proteins clump together to make deposits called ‘amyloid’. • Amyloid can build up around the nerves, heart, and other places in the body, preventing them from working normally. This causes the symptoms of the illness. Onpattro works by lowering the amount of TTR protein that the liver makes. • This means there is less TTR protein in the blood that can form amyloid. • This can help to reduce the effects of this illness. Onpattro is used in adults only. 2. WHAT YOU NEED TO KNOW BEFORE YOU ARE GIVEN ONPATTRO YOU MUST NOT BE GIVEN ONPATTRO • if you have ever had a severe allergic reaction to patisiran, or any of the other ingredients of this medicine (listed in section 6). If you are not sure, talk to your doctor or nurse before you are given Onpattro. 27 WARNINGS AND Read the complete document
1 ANNEX I SUMMARY OF PRODUCT CHARACTERISTICS 2 1. NAME OF THE MEDICINAL PRODUCT Onpattro 2 mg/mL concentrate for solution for infusion 2. QUALITATIVE AND QUANTITATIVE COMPOSITION Each mL contains patisiran sodium equivalent to 2 mg patisiran. Each vial contains patisiran sodium equivalent to 10 mg patisiran formulated as lipid nanoparticles. Excipients with known effect Each mL of concentrate contains 3.99 mg sodium. For the full list of excipients, see section 6.1. 3. PHARMACEUTICAL FORM Concentrate for solution for infusion (sterile concentrate). White to off-white, opalescent, homogeneous solution (pH approximately 7). 4. CLINICAL PARTICULARS 4.1 THERAPEUTIC INDICATIONS Onpattro is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy. 4.2 POSOLOGY AND METHOD OF ADMINISTRATION Therapy should be initiated under the supervision of a physician knowledgeable in the management of amyloidosis. Posology The recommended dose of Onpattro is 300 micrograms per kg body weight administered via intravenous (IV) infusion once every 3 weeks. Dosing is based on actual body weight. For patients weighing ≥ 100 kg, the maximum recommended dose is 30 mg. Treatment should be initiated as early as possible after symptom onset (see section 5.1). The decision to continue treatment in those patients whose disease progresses to stage 3 polyneuropathy should be taken at the discretion of the physician based on the overall benefit and risk assessment (see section 5.1). Vitamin A supplementation at approximately 2 500 IU vitamin A per day is advised for patients treated with Onpattro (see section 4.4). _ _ 3 _Required premedication _ _ _ All patients should receive premedication prior to Onpattro administration to reduce the risk of infusion-related reactions (IRRs) (see section 4.4). Each of the following medicinal products should be given on the day of Onpattro infusion at least 60 minutes prior to the start of infusion: • Intravenous Read the complete document