Decision

Asciminib Treatment protocol Information for patients

Updated 13 December 2022

This decision was withdrawn on 13 December 2022

Withdrawn due to granting of marketing authorisation

Introduction

The aim of the Early Access to Medicines Scheme (EAMS) is to provide earlier availability of promising new unlicensed medicines (medicines that do not have a marketing authorisation or are used outside their licence) to UK patients that have a high unmet clinical need. The medicines included in the scheme after they have received a positive scientific opinion are those that are intended to treat, diagnose or prevent seriously debilitating or life-threatening conditions where there are no adequate treatment options. More information about the scheme can be found here: http://www.mhra.gov.uk/Howweregulate/Innovation/EarlyaccesstomedicinesschemeEAMS/index.htm

The information below is intended for you, the patient, and is provided by the pharmaceutical company (called scientific opinion holder) that manufactures the EAMS medicine. This medicine, which does not yet have a drug licence or is used outside its licence, may also be used in combination with other medicines. More information about medicines licensing can be found here: http://www.nhs.uk/conditions/medicines-information

This medicine can be prescribed for individual patients to meet specific needs provided they are given sufficient information about the medicine to make an informed decision. Your physician will be responsible for giving you all the information you need to make this decision and for obtaining informed consent from you prior to treatment. You will be asked to sign a form to confirm that you are providing informed consent to receiving the EAMS treatment. Information on consent can be found here: https://www.nhs.uk/conditions/Consent-to-treatment

The information below is provided to help you decide with your physician on whether to use the EAMS medicine and helps explain how to use it in accordance with the pharmaceutical company’s instructions for safe and proper use. A positive scientific opinion is not a recommendation for use of the medicine and should not be interpreted as such. Under EAMS, the risk and legal responsibility for prescribing the medicine remains with the physician, and the opinion and EAMS documentation published by the MHRA are intended only to inform physicians’ decision making and not to recommend use. An EAMS scientific opinion does not affect the civil liability of the manufacturer or any physician in relation to the product.

The information below may change during the time you are using the medicine if more data become available. Your physician will highlight to you any changes that you need to be aware of.

Whilst you are using this medicine, data will be collected on the use and safety profile of the medicine, to ensure that the benefits of taking the medicine continue to outweigh any potential risks. Your physician will answer all your questions during and after the treatment and will provide you with contact details that you should use in case of any events or problems.

Each patient enrolled in the scheme will continue to receive the EAMS product until the end of the treatment in line with prescribing and NHS guidance and as long as benefit is seen. In rare cases where the EAMS treatment may not be available anymore, your physician will discuss other options with you.   ##Information for the patient

Asciminib 20 mg film-coated tablets

Asciminib 40 mg film-coated tablets

Read all of this leaflet carefully before you start taking 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 asciminib is and what it is used for
2. What you need to know before you take asciminib
3. How to take asciminib
4. Possible side effects
5. How to store asciminib
6. Contents of the pack and other information

1. What asciminib is and what it is used for

What asciminib is

Asciminib contains the active substance asciminib, which belongs to a group of medicines called protein kinase inhibitors.

What asciminib is used for

Asciminib is used to treat adults with Philadelphia chromosome positive chronic myeloid leukaemia (Ph+ CML) in chronic phase that do not have a gene defect called T315I mutation and who are no longer benefiting from at least two previous medicines of a similar type called tyrosine kinase inhibitors

Ph+ CML is a type of blood cancer (leukaemia) in which the body produces too many abnormal white blood cells. Chronic phase is the first phase of this blood cancer.

How asciminib works

Asciminib blocks the action of a protein (BCR ABL1) produced by the abnormal white blood cells and stops their division and growth.

If you have any questions about how asciminib works or why this medicine has been prescribed for you, ask your doctor or nurse.

2. What you need to know before you take asciminib

You must not take asciminib

• if you are allergic to asciminib or any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions

Talk to your doctor or nurse before taking asciminib if any of the following applies to you: * if you have or have ever had pancreas problems (inflamed pancreas, pancreatitis). * if you have ever had or might now have a hepatitis B infection. This is because asciminib could cause hepatitis B to become active again. You will be carefully checked by your doctor for signs of this infection before treatment is started.

Tell your doctor or nurse immediately if you get any of the following during treatment with asciminib:

• if you experience weakness, spontaneous bleeding or bruising and frequent infections with signs such as fever, chills, sore throat or muscle ulcers (signs of decreased bone marrow activity, resulting in a reduced number of white blood cells, red blood cells and platelets, also known known as myelosuppression).
• if blood tests show that you have high levels of enzymes called lipase and amylase (signs of damage to the pancreas, also known as pancreatic toxicity).
• if you have a heart disorder or a heart rhythm disorder, such as an irregular heartbeat or an abnormal electrical signal called prolongation of the QT interval.
• if blood tests show that you have a low level of potassium or magnesium (hypokalaemia or hypomagnesaemia).
• if you are being treated with medicines that may have an unwanted effect on the function of the heart (torsades de pointes) (see “Other medicines and Asciminib”)
• if you experience headache, dizziness, chest pain or shortness of breath (signs of high blood pressure, also known as hypertension).
• if you experience rash, itching, hives, breathlessness or difficulty breathing, wheezing or coughing, light headedness, dizziness, changes in levels of consciousness, low blood pressure, skin reddening, facial/throat swelling, blue discoloration of the lips, tongue or skin (signs of allergic reactions).You must stop taking asciminib and seek urgent medical help.

Monitoring during your treatment with asciminib

Your doctor will regularly monitor your condition to check that the treatment is having the desired effect. You will have regular tests including blood tests during treatment. These tests will monitor:

• the amount of blood cells (white blood cells, red blood cells and platelets)
• the levels of pancreas enzymes (amylase and lipase)
• the levels of electrolytes (potassium, magnesium)
• your heart rate and blood pressure.

Children and adolescents

This medicine is not recommended for patients aged under 18 years. No data are available in this age group.

Other medicines and asciminib

Tell your doctor or pharmacist if you are taking, have recently taken or might take any other medicines. In particular, tell your doctor or pharmacist if you are using:

• medicines used to treat seizures, such as carbamazepine, phenobarbital or phenytoin.
• medicines used to treat pain and or as sedatives before or during medical or surgical procedures, such as alfentanil or fentanyl.
• medicines used to treat migraine or dementia, such as dihydroergotamine or ergotamine.
• medicines that may have an unwanted effect on the electrical activities of the heart (torsades de pointes), such as bepridil, chloroquine, clarithyromycin, halofantrine, haloperidol, methadone, moxifloxacin or pimozide.
• medicines used to reduce the blood’s ability to clot, such as warfarin.
• St. John’s wort (also known as Hypericum perforatum), a herbal medicine used to treat depression and other conditions.

The medicines listed here may not be the only ones that could interact with asciminib.

You should also tell your doctor if you are already taking asciminib and you are prescribed any new medicine that you have not taken previously during asciminib treatment.

Ask your doctor or nurse if you are not sure whether your medicine is one of the medicines listed above.

Asciminib with food and drink

Do not take Asciminib with food. Take it at least 2 hours after and 1 hour before any food. For more information, see “When to take Asciminib” in section 3.

Contraception, pregnancy and breast feeding

If you are pregnant or breast feeding, think you may be pregnant or are planning to have a baby, ask your doctor or pharmacist for advice before taking this medicine.

Asciminib may harm your unborn baby. If you are a woman who could become pregnant, your doctor will discuss with you the potential risks of taking Asciminib during pregnancy or breast feeding.

If you are a woman who could become pregnant, your doctor will perform a pregnancy test if necessary before starting treatment with Asciminib.

If you do become pregnant, or think you may be pregnant, after starting treatment with Asciminib, tell your doctor straight away.

Breast feeding

It is not known if Asciminib passes into breast milk. It is recommended that you do not breast feed while you are taking Asciminib and for at least 3 days after you stop taking it.

Contraceptive advice for women

If you are a woman who could become pregnant, you should use an effective method of contraception during treatment with Asciminib and for at least 3 days after you stop taking it to avoid becoming pregnant. Ask your doctor about effective methods of contraception.

Driving, cycling and using machines

If you experience side effects (such as dizziness or visual disorders) with a potential impact on the ability to safely drive, cycle or use any tools or machines after taking this medicine, you should refrain from these activities until the effect has disappeared.

Asciminib contains lactose

If you have been told by your doctor that you have an intolerance to some sugars, contact your doctor before taking this medicine.

This medicine contains less than 1 mmol sodium (23 mg) per film-coated tablet, that is to say essentially ‘sodium-free’.

3. How to take asciminib

Always take this medicine exactly as your doctor has told you. Check with your doctor or nurse if you are not sure.

Do not exceed the recommended dose prescribed by your doctor.

Asciminib will only be prescribed to you by a doctor with experience in medicines to treat leukaemia.

How much asciminib to take

Your doctor will tell you exactly how many asciminib tablets you should take per day, and how to take them.

The usual total daily dose of asciminib is 80 mg (2 tablets of Asciminib 40 mg per day). You may take your daily dose:

• Once daily: Take 2 tablets together at approximately the same time each day, OR
• Twice daily: Take 1 tablet, then take another one approximately 12 hours later.

You should not change the asciminib dose or schedule without first talking to your doctor.

Depending on how you respond to treatment, your doctor may ask you to change to a lower dose or to temporarily or permanently stop the treatment.

When to take asciminib

Do not take asciminib with food.

Take asciminib:

• at least 2 hours after any food
• then wait at least 1 hour before eating again.

Taking asciminib at the same time each day will help you to remember when to take your medicine.

How to take asciminib

Swallow asciminib tablets whole. Do not break, crush or chew the tablets.

How long to take asciminib

Continue taking Asciminib for as long as your doctor tells you. This is a long term treatment, possibly lasting for months or years. Your doctor will regularly monitor your condition to check that the treatment is having the desired effect.

If you have questions about how long to take asciminib, talk to your doctor or nurse.

If you take more asciminib than you should

If you have taken more Asciminib than you should have, or if someone else accidentally takes your medicine, contact a doctor for advice straight away. Show them the pack of asciminib. Medical treatment may be necessary.

If you miss a dose of asciminib

If you take asciminib once daily

If you miss asciminib by more than 12 hours, skip the missed dose and take the next one as usual.

If you take asciminib twice daily

If you miss asciminib by more than 6 hours, skip the missed dose and take the next one as usual.

If you stop taking asciminib

Do not stop taking asciminib unless your doctor tells you to.

If you have any further questions on the use of this medicine, ask your doctor or nurse.

4. Possible side effects

Like all medicines, this medicine can cause side effects, although not everybody gets them. The following side effects have been reported with this medicine:

Serious side effects

If you experience any serious side effects, stop taking this medicine and tell your doctor immediately.

Very common (may affect more than 1 in 10 people)

• spontaneous bleeding or bruising (signs of low level of platelets, thrombocytopenia)
• fever, sore throat, frequent infections (signs of low level of white blood cells, neutropenia)

Uncommon (may affect up to 1 in every 100 people)

• irregular heart beat, (prolongation of the QT interval)
• fever above 38°C associated with a low level of white blood cells (febrile neutropenia)

Other side effects

Other side effects include the following listed below. If these side effects become severe, tell your doctor or nurse.

Very common (may affect more than 1 in 10 people)

• tiredness, fatigue, pale skin (potential signs of low level of red blood cells, anaemia)
• headache, dizziness, chest pain or shortness of breath (signs of high blood pressure, hypertension)
• headache
• dizziness
• cough
• vomiting
• diarrhoea
• nausea
• abdominal pain
• rash
• pain in muscles, bones or joints (musculoskeletal pain)
• joint pain (arthralgia)
• tiredness (fatigue)
• itching (pruritus)
• blurred vision
• dry eyes
• palpitations

Common (may affect up to 1 in every 10 people)

• fever, coughing, difficulty breathing, wheezing (signs of lower respiratory tract infections)
• influenza
• loss of appetite
• chest pain, cough, hiccups, rapid breathing, fluid collection between the lungs and chest cavity which, if severe, could make you breathless (pleural effusion)
• shortness of breath, laboured breathing (signs of dyspnoea)
• chest pain (non cardiac chest pain)
• severe upper stomach pain (sign of inflamed pancreas, pancreatitis)
• itchy rash (urticaria)
• fever (pyrexia)
• generalised swelling (oedema)

Abnormal blood test results

During asciminib treatment, the results of blood tests may be abnormal, which can give your doctor information on the function of your organs. For example:

Very common (may affect more than 1 in 10 people)

• high level of the enzymes lipase and amylase (pancreas function)
• high level of the enzymes transaminases, which include alanine aminotransferase (ALT), aspartate aminotransferase (AST) and gamma glutamyltransferase (GGT) (liver function)
• high level of fats/lipids (dyslipidaemia)

Common (may affect up to 1 in every 10 people)

• high level of bilirubin (liver function)
• high level of creatine phosphokinase (muscles function)

A decrese in phospate levels may also be obsvered.

Reporting of side effects

If you get any side effects, talk to your doctor or nurse. This includes any possible side effects not listed in this leaflet. You can also report side effects directly to MHRA via the Yellow Card scheme via www.mhra.gov.uk/yellowcard. By reporting side effects, you can help provide more information on the safety of this medicine.

5. How to store asciminib

Keep this medicine out of the sight and reach of children.

Do not use this medicine after the expiry date which is stated on the carton after EXP. The expiry date refers to the last day of that month.

Do not store above 25°C. After first opening of the bottle, the tablets can be stored for 3 months. Do not use this medicine if you notice any damage to the packaging or if there are any signs of tampering. Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to throw away medicines you no longer use. These measures will help protect the environment.

6. Contents of the pack and other information

What Asciminib contains

• The active substance is asciminib.
  • Each 20 mg film coated tablet contains 20 mg asciminib (as hydrochloride).
  • Each 40 mg film coated tablet contains 40 mg asciminib (as hydrochloride).
• The other ingredients are: lactose monohydrate, microcrystalline cellulose (E460i), hydroxypropylcellulose(E463), croscarmellose sodium (E468), polyvinyl alcohol (E1203), titanium dioxide (E171), magnesium stearate, talc (E553b), colloidal silicon dioxide, iron oxide (E172, yellow and red for 20 mg film coated tablets, black and red for 40 mg film coated tablets), lecithin (E322), xanthan gum (E415).

What Asciminib looks like and contents of the pack

Asciminib 20mg film-coated tablets are pale yellow, round curved with bevelled edges 6mm film-coated tablets debossed with NVR on one side ad “20” on the other.

Asciminib 40mg film-coated tablets are violet white, round curved with bevelled edges 8mm film-coated tablets debossed with NVR on one side ad “40” on the other.

Asciminib film coated tablets are supplied in HDPE bottles fitted with child-resistant closure containing 30 film coated tablets.

Scientific Opinion Holder and Manufacturer

Novartis Pharmaceuticals UK Ltd 2nd Floor, The WestWorks Building White City 195 Wood Lane London W12 7FQ UK

Manufacturer

Novartis Pharma AG Lichtstrasse 35, Basel, CH-4056 Switzerland

This protocol was revised in November 2021

Additional information

Informed Consent Form

You will have the Early Access to Medicines Scheme explained to you using the informed consent form. You will be asked to sign this form and a copy of the signed consent form will be given to you to keep.

Patient Alert Card

You will also be given a Patient Alert Card before you start treatment with asciminib. You must keep this Patient Alert Card with you at all times during the treatment and for at least 30 days after completing your treatment with asciminib. The card alerts any other healthcare professional who may treat you that you are currently receiving asciminib through an early access scheme, provides information regarding the important known side effects for which you should seek assistance should they occur, contact details for the doctor managing your treatment and the company’s contact details.

Patient data to be collected

Information collected during the scheme will mostly be used for safety surveillance and cannot replace a proper clinical trial to support a marketing authorisation. These data are required by the MHRA to help verify that your condition complies with the EAMS indication and help interpret the side effects and other events occurring during and after the EAMS treatment. These data include your initials, year of birth, gender, information about cancer, comorbidities, response data and any medications you may be taking.

Contact information

Novartis Pharmaceuticals UK Limited, medical information, Tel 01276698370 or email medinfo.uk@novartis.com