Kalydeco t (ivacaftor) tablets 150 mg

Transcription

1 What effect does Kalydeco have? Why take it with fat-containing food? What are CFTR and gating mutations? HOW TO TAKE YOUR Kalydeco t (ivacaftor) tablets 150 mg When do I need to take the tablets? What if I miss a dose? What if I take other medicines? What about side effects? This booklet is for patients who have been prescribed Kalydeco tablets by their CF doctor. Please refer to the full Kalydeco tablets Patient Information Leaflet (PIL) for more information. Kalydeco tablets are indicated for the treatment of patients with cystic fibrosis (CF) aged 6 years and older and weighing 25 kg or more who have one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. Kalydeco tablets are also indicated for the treatment of patients with CF aged 18 years and older who have an R117H mutation in the CFTR gene. t This medicine is subject to additional monitoring. This will allow quick identification of new safety information. You can help by reporting any side effects you may get. If you get any side effects, talk to your doctor or pharmacist. This includes any possible side effects not listed in the patient information leaflet. You can also report side effects directly via the national reporting system

2 A few words about Cystic Fibrosis You may already know a lot about your condition, cystic fibrosis (CF), or you may not know that much about CF at all. CF is an inherited condition caused by a faulty gene. Over 10,500 people have cystic fibrosis in the UK. 1 Your body is made up of cells, and each cell contains around 22,000 genes. 2 One of these genes tells your body how to build a protein called cystic fibrosis transmembrane conductance regulator (CFTR). What is CFTR? The CFTR protein acts as a channel that controls the movement of salt and water in and out of your cells. 3,4 Every child inherits two CFTR genes one from each parent. For someone to have CF, they must inherit a faulty copy of the CFTR gene from both of their parents. Carrier parents Child Carrier Carrier Child Note: Explanations of words highlighted in green throughout this booklet appear on page Affected child Carrier Carrier Unaffected child

3 What is a gating mutation? CF can be caused by different genetic mutations (or faults) within the CFTR gene. 3,4 Gating is the name given to a specific group of faults within the CFTR gene. 4 This fault in your CFTR gene causes a salt and water imbalance in and out of your cells resulting in thick, sticky mucus in your lungs and pancreas. It is normal to have mucus in the lungs and pancreas, but in CF the mucus becomes thicker and stickier than normal, clogging up these organs and preventing them from working properly. This can then lead to symptoms such as: 3,4 Kalydeco tablets are approved for nine specific gating mutations: G551D, S549N, G551S, G1244E, S1255P, G178R, S549R, S1251N and G1349D. Kalydeco tablets are also approved for the treatment of patients with CF aged 18 years and older who have an R117H mutation in the CFTR gene. 5 The most common of these mutations is G551D. 1,6 You are one of a small group (approximately 5.7% of people with CF in the UK and 14.5% in Ireland) who has the gating mutation in at least one copy of your CFTR genes. 1,6 3 Shortness of breath Frequent coughing An increased chance of infection in your chest Difficulty digesting food (making it hard for your body to absorb fats and vitamins)

4 What happens with a faulty gating function? You may hear the words gating defect or gating function used when people talk about your mutation. Gating describes how the mutation disrupts the normal functioning of the CFTR channel across the outer surface of your cells. While a normal CFTR channel allows the movement of salt and water in and out of cells, a faulty gating -mutated CFTR channel acts like a gate that is usually locked and only opens intermittently. As a result, the salt/water balance of your cells is impaired causing mucus to become thicker and stickier, so that it clogs your lungs and pancreas. 3,4 Normal CFTR protein CFTR protein gating defect A normal CFTR gene makes normal CFTR proteins, which are like gates The gates don t work properly, so very few molecules (Cl - ions) move in or out of the cell 4

5 How Kalydeco works Kalydeco binds directly to the CFTR channel helping it to open and work properly. This is like opening a locked gate to allow the movement of salt through the channel and restore the normal salt/water balance. The exact mechanism by which it has this effect is not fully known. Gating-CFTR with Kalydeco It s important to take your Kalydeco correctly to achieve the best results. This booklet is designed to tell you what you need to know about taking Kalydeco. Please also see the Package Information Leaflet inside the Kalydeco (ivacaftor) pack. If you have any questions about your CF or treatment, don t hesitate to talk to your CF doctor or CF nurse they are there to help you. 5

6 An introduction to your Kalydeco prescription You have been prescribed Kalydeco 150 mg tablets 5 Kalydeco is taken orally (by mouth) 5 and is a small molecule that enters the bloodstream from the digestive system The scientific name of Kalydeco is ivacaftor, so you may sometimes hear it called ivacaftor or see that name written on prescriptions and notes Kalydeco tablets are indicated for the treatment of patients with cystic fibrosis (CF) aged 6 years and older and weighing 25 kg or more who have one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. Kalydeco tablets are also indicated for the treatment of patients with CF aged 18 years and older who have an R117H mutation in the CFTR gene. 6

7 How to take your Kalydeco Kalydeco should be taken as one 150 mg tablet, twice a day, ideally 12 hours apart. 5 Kalydeco is taken by mouth: swallow the tablets whole do not try to chew, break or dissolve the tablets. Kalydeco should be taken with fat-containing food 5 To give Kalydeco the best chance of working properly, it is very important to always take one 150 mg tablet, twice a day, ideally 12 hours apart, with fat-containing food. Kalydeco is fat-soluble which means it dissolves in fat. This makes it easier for your body to absorb/get the drug into your bloodstream. In fact, studies have shown that the absorption of Kalydeco increased approximately 2- to 4-times when given with fat-containing food. 5 Therefore, Kalydeco should be taken with fat-containing foods to help ensure you have the right amount of Kalydeco in your body. What to do if you miss a tablet If less than 6 hours have passed since the time you missed your dose, take the missed dose as normal with a fat-containing food (you can make this a snack e.g. muesli or cereal bar, if it is not at a meal time). 5 (See page 18 for a more detailed explaination of what to do if you have missed a dose) There is no specific recommended amount of fat to take with Kalydeco. However, for a guide to fat intake, take a look at standard nutritional guidelines or the Cystic Fibrosis Trust Factsheet: Eating well with Cystic Fibrosis. Eating_Well_Children_and_Parents_Sep_10.pdf You will also find some suggestions for fat-containing meals and snacks on the following pages of this booklet. 7

8 Examples of fat-containing foods to take with your Kalydeco Fat is an essential nutrient and an important part of a healthy diet. You may need more calories than other people who don t have CF because your body has a harder time absorbing the fat and nutrients in food. There are two types of fat saturated and unsaturated. In general, unsaturated fatty foods are better for your health than saturated fats. Saturated fats Found in meat, sausages, bacon, meat pies, butter, cheese (especially hard cheese), milk (except skimmed), cream, cakes and biscuits. Unsaturated fats Found in sunflower oil, olive oil, oily fish (such as: salmon, sardines and mackerel), nuts (such as peanuts, cashews, almonds, walnuts and macadamia nuts) and avocados. 8

9 Suggestions for healthy fat-containing meals or snacks 7 Breakfast Here is a list of suggested foods: Egg (hard-boiled, poached, fried, scrambled or 2-egg omelette) and toast with baked beans Bagel with cream cheese Full fat yoghurt Muesli with nuts and seeds Cereal or porridge with whole milk Toast with butter or margarine spreads Occasional lean bacon and/or sausage 9

10 Lunch Here is a list of suggested foods: Avocado salad with olive oil or dressing Cheese, salmon, tuna or chicken sandwich Cheese on toast Cheese and crackers Ham and cheese sandwich Egg salad sandwich (two large eggs plus mayonnaise) Bacon, salad and dressing 2 slices of cheese pizza Thick vegetable or chicken soup 10

11 Dinner Here is a list of suggested foods: Chicken and vegetables Salmon fillet with salad Fish with mashed potato Quiche and salad Roast dinner Lasagne Pizza Curry and rice Chocolate pudding Yoghurt Ice cream 11

12 Snacks If you are in a hurry or on the go here are some suggested snacks: Handful of peanuts/almonds Slice of hard cheese Croissant Slice of buttered toast Medium avocado Muesli with whole milk Whole milk yoghurt Large glass of whole milk or milkshake Muesli or cereal bar Bag of microwave popcorn Ice cream Biscuits Chocolate bar Slice of cake RemembeR to eat a healthy balanced daily diet. If you are unsure of what you should be eating and how much, ask your CF doctor or CF nurse for meals and snack advice. You can also ask to see a dietician who can help you plan what to eat. 12

13 On Kalydeco, you should avoid these fruits Foods containing grapefruit or Seville oranges (also known as bitter oranges) should be avoided as the chemicals they contain can increase the amount of Kalydeco in your bloodstream above desirable levels. 5 What s the difference between a Seville orange and an ordinary orange? Common foods/drinks to avoid: Seville oranges Marmalade Cakes that contain Seville oranges Grapefruit Grapefruit juice It can be tricky to tell the difference, so always try to double check the label or ask the shop or restaurant which type of orange is being sold/ served. Usually, Seville oranges have a rougher, bumpier skin, are highly fragrant and sometimes bitter in taste. 13

14 Why it is so important to take your Kalydeco correctly Although there is currently no treatment available that can cure CF, Kalydeco may help treat and improve your symptoms. To help achieve the best results, it is important to try to take your Kalydeco as prescribed: every day, twice a day, ideally 12 hours apart. 5 This will help maintain the right amount of Kalydeco in your body for it to keep working effectively. If I feel better after taking Kalydeco, do I have to keep taking it? If you notice an improvement, it s tempting to think that you can ease off or stop taking your tablets. Kalydeco is intended as a twice daily on-going treatment. Therefore, you should not stop taking Kalydeco as prescribed without first consulting your CF doctor or CF nurse. 14

15 I feel the same as before I started taking Kalydeco, so why should I keep taking it? Although you may not notice any improvement in how you feel, this does not mean that Kalydeco is not working. To improve the likelihood of Kalydeco working appropriately, take it as prescribed by your doctor. Is it important to stick to my CF regimen and other medicines while taking Kalydeco? Kalydeco works together with your overall CF treatment regimen. However well you feel, do not be tempted to stop your physiotherapy or your other medicines and just take Kalydeco, as it works best when taken as part of a whole treatment regimen. If, for any reason, you are finding it tricky to stick to your CF regimen, have an open discussion with your CF doctor or CF nurse they want to know and want to help. 15

16 Tips to help you take Kalydeco correctly as part of your daily routine/lifestyle Set up a reminder on your phone or set up your alarm clock Put a reminder on your phone for times to take your Kalydeco in the morning and evening, or set your alarm clock to the times to take Kalydeco If you are not going to have easy access to fat-containing food when you are due to take your Kalydeco, pack a fat-containing snack to take with you. Or download the CF MedCare App from the App Store, or on the Android App on Google play. The CF MedCare App is a handy App that lets you set customisable reminders for when to take your different cystic fibrosis treatments 16

17 Sleepovers and sleeping in If you are going on a sleepover, remember to take enough Kalydeco with you for the time you will be away from home. If you plan to have a lie in on weekends or holidays, plan the time you take your Kalydeco so that you don t miss a dose. Holidays and school trips If you are travelling, always make sure you pack enough Kalydeco to last your entire trip plus a couple of extra days worth, in case you are delayed in returning home for any reason. Make sure you never run out of your Kalydeco Each prescription should give you 28 days supply of Kalydeco. Please ensure that you have requested a repeat prescription from your doctor, at the CF centre where you are treated, if you are planning to be away when your next prescription is due. If you think you may run out of tablets for any reason, tell your CF doctor, CF nurse or CF pharmacist immediately to arrange a new prescription. 17

18 What to do if you take too many Kalydeco tablets, or forget to take one It is important to take your Kalydeco 150 mg tablets, every day, twice daily, ideally 12 hours apart, to ensure that the right amount of Kalydeco to be effective remains in your body. If you have taken too many Kalydeco tablets, you may experience some side effects (see page 24). If so, contact your CF doctor, CF nurse or CF pharmacist AS SOON AS POSSIBLE to ask for their advice. 18

19 However, if you have forgotten to take one of your Kalydeco 150 mg tablets in the morning or evening If less than 6 hours have passed... since the time you missed your dose, take the missed dose as normal with a fat-containing food 5 (you can make this a snack e.g. muesli or cereal bar, if it is not at a meal time) If more than 6 hours have passed... wait until your next scheduled dose and take that, but NEVER TAKE A DOUBLE DOSE (two Kalydeco 150 mg tablets) to make up for the forgotten tablet 5 EXAMPLE SITUATION If you have taken your Kalydeco at 7am, ideally the best time for your next dose is 7pm. However, if you forget at 7pm, it is okay to take your second dose any time up until midnight (6 hours later). 5 REMEMBER Don t stop taking Kalydeco without speaking to your CF doctor or CF nurse first. They can help if you are finding it tricky to remember when and how to take your tablets 19

20 Precautions when taking Kalydeco together with other medicines, or if you have specific other medical conditions Kalydeco might interact with other medicines. Tell your doctor or pharmacist if you are taking or have recently taken any other medicines, including medicines obtained without a prescription, such as herbal supplements. Anticonvulsant medicines (used for the treatment of epileptic seizures): phenobarbital, carbamazepine, phenytoin Herbal medicines: St. John s Wort (Hypericum perforatum) Tell your doctor if you take any of the following medicines: 8 Antifungal medicines (used for the treatment of fungal infections): ketoconazole, itraconazole, posaconazole, voriconazole, fluconazole Antibiotic medicines (used for the treatment of bacterial infections): telithromycin, clarithromycin, erythromycin, rifampicin, rifabutin 20

21 Benzodiazepines (used for the treatment of anxiety, insomnia, agitation, etc.): midazolam, alprazolam, diazepam, triazolam Immunosuppressants (used after an organ transplantation): ciclosporin, tacrolimus Cardiac glycosides (used for the treatment of mild to moderate congestive heart failure and an abnormal heart rhythm called atrial fibrillation): IMPORTANT Talk to your doctor if you have been told you have liver or kidney disease, as your doctor may need to adjust the dose of Kalydeco. 8 Tell your doctor or pharmacist if you are taking or have recently taken any other medicines, including medicines obtained without a prescription, such as herbal supplements. 8 digoxin Anticoagulants (used to prevent blood clots from forming or growing larger in blood and blood vessels): warfarin 21

22 Other things you need to know before taking Kalydeco If you are thinking of drinking alcohol If you are at, or above, the legal drinking age for alcohol, you may wonder if you can drink alcohol while on medication. The effects of alcohol on Kalydeco have not been studied, so talk to your CF doctor or CF nurse for advice and guidance. If you drive or operate machinery Dizziness has been reported in patients receiving Kalydeco, which could influence the ability to drive or operate machines. If you experience dizziness, you should not drive or operate machines until these symptoms disappear. 8 Cataracts... Abnormality of the eye lens (cataract) without any effect on vision has been noted in some children aged up to 12 years old receiving Kalydeco. 8 22

23 If you are pregnant or breast-feeding If you are pregnant or breast-feeding, think you may be pregnant or are planning to have a baby, ask your doctor for advice before taking this medicine. Your doctor will help you decide what is best for you and your child. It is unknown whether Kalydeco is excreted in human milk. If you plan to breast-feed, ask your doctor for advice before taking Kalydeco. 8 If you are lactose intolerant, or have pre-diagnosed kidney or liver conditions Talk to your CF doctor or CF nurse before taking Kalydeco if: 8 You have intolerance to lactose or other sugars You have any pre-diagnosed kidney or liver condition 23

24 Side effects you may experience with Kalydeco Like all medicines, this medicine can cause side effects, although not everybody gets them. As everybody reacts to treatment differently, it is not possible to say whether or not you will experience side effects and, if so, what they will be. However, the table to the right lists some of the most commonly reported side effects to look out for. Report side effects or unusual symptoms to your doctor, including any not listed in the Patient Information Leaflet. If you experience any side effects or unusual symptoms, tell your CF doctor or CF nurse immediately, as they can offer help and advise. Very common side effects (may affect more than 1 in 10 people): 8 Upper respiratory tract infection (the common cold), including sore throat and nasal congestion Headache Dizziness Abdominal pain (stomach ache) Diarrhoea Rash Please see the Patient Information Leaflet for more information about side effects. 24

25 A few explanations of scientific words used in this booklet CFTR protein CFTR gene Acts as a channel across the membrane of cells that produce mucus, sweat, saliva, tears and digestive enzymes 9 A gene in your cells that provides instructions for making a protein called the cystic fibrosis transmembrane conductance regulator (CFTR) 9 G551D mutation A fault (mutation), sometimes called a gating defect, which occurs when the amino acid (G), in position 551 of the CFTR protein, is replaced with aspartic acid (D). This produces a CFTR protein channel that does not open in the correct way. It acts like a locked gate, restricting the flow of salt and water through the CFTR channel 10 Gating mutation The group of mutations also called class III mutations, which produce CFTR protein channels that do not open in the correct way. The most common is the G551D mutation Mucus Cl - ions A naturally occurring bodily fluid that lubricates and protects the lining of the airways, digestive system, and other organs and tissues 11 Chloride ions are chemicals in your body that make up salt (sodium chloride). The transport of sodium chloride across cell membrane is very important. When the CFTR channel protein is faulty it cannot transport chloride ions across the membrane. This fault can be one of the underlying problems in CF 12 Ivacaftor Ivacaftor is the scientific name for Kalydeco (which is the brand name) 8 25

26 References 1. Cystic Fibrosis Trust Annual Data Report 2014; Published August Pertesa M, Salzberg SL. Genome Biol 2010;11: Orenstein DM. Cystic Fibrosis: A Guide for Patient and Family. 3rd ed. Philadelphia, PA: Lippincott Williams & Wilkins; Welsh MJ et al. Cystic fibrosis: membrane transport disorders. In: Valle D, Beaudet A, Vogelstein B et al., eds. The Online Metabolic & Molecular Bases of Inherited Disease. The McGraw-Hill Companies Inc; 2004:part 21, chap KALYDECO tablets. Summary of Product Characteristics. November Vertex Pharmaceuticals (Europe) Limited. 6. Annual report from Cystic Fibrosis Registry of Ireland; Published CF Trust. Nutrition A Guide For Children and Parents; April Available at: media/185765/fs_nutrition_children_and_parents_13_5_13.pdf. Last accessed January KALYDECO tablets. Patient information leaflet. September Vertex Pharmaceuticals (Europe) Limited. 9. U.S. National Library of Medicine Genetics Home Reference. CFTR. Available at: Last accessed January Sermet-Gaudelus I. Eur Respir Rev 2013;22: Mucus definition. Available at: Last accessed January U.S. National Library of Medicine Genetics home reference, Cystic fibrosis, Available at: condition/cystic-fibrosis. Last accessed January Last accessed January

27 Kalydeco is a trademark of Vertex Pharmaceuticals Incorporated. Kalydeco, VERTEX and the VERTEX triangle logo are registered trademarks of Vertex Pharmaceuticals Incorporated Vertex Pharmaceuticals Incorporated. VXR-UK a Date of preparation: January 2016