Scemblix is a prescription medicine used to treat adults with:

• Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with 2 or more tyrosine kinase inhibitor (TKI) medicines.
• Ph+ CML in CP with the T315I mutation.

It is not known if this medicine is safe and effective in children.

Scemblix was approved in October 2021, and works by specifically targeting the ABL myristoyl pocket (STAMP). It represents an important development in addressing TKI resistance for patients with CML who suffer intolerance or an inadequate response after at least two previous TKI treatments. It may also be given to Ph+ CML patients in CP with the T315I mutation.

Scemblix is an oral tablet that is taken once or twice daily without food. Common side effects include upper respiratory tract infections, musculoskeletal pain, fatigue, nausea, rash, diarrhea, and abnormal laboratory test results. Serious adverse reactions include bone marrow suppression, pancreatic toxicity, high blood pressure, hypersensitivity reactions, and cardiovascular toxicity.

• Take Scemblix exactly as your healthcare provider tells you.
• Do not change your dose or schedule or stop treatment unless your healthcare provider tells you to.
• Take Scemblix tablets without food. You should avoid eating for at least 2 hours before and 1 hour after taking Scemblix.
• Swallow the tablets whole. Do not break, crush, or chew the tablets.
• If you take Scemblix 1 time a day and miss a dose by more than 12 hours, skip the missed dose and take your next dose at your regular time.
• If you take Scemblix 2 times a day and miss a dose by more than 6 hours, skip the missed dose and take your next dose at your regular time.

Usual Adult Dose for Philadelphia Chromosome-Positive Chronic Myeloid Leukemia (Ph+ CML)

Recommended Dosage in Ph+ CML in CP: 80 mg orally once daily or 40 mg twice daily.
Recommended Dosage in Ph+ CML in CP with the T315I Mutation: 200 mg orally twice daily.

Use: adult patients with:
- Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with two or more tyrosine kinase inhibitors (TKIs).
- Ph+ CML in CP with the T315I mutation.

Before you start treatment, tell your healthcare provider about all of your medical conditions, including if you:

• have a history of inflammation of your pancreas (pancreatitis)
• have a history of heart problems or blood clots in your arteries and veins (types of blood vessels)
• are pregnant or plan to become pregnant. Scemblix can harm your unborn baby.
  • Your healthcare provider will do a pregnancy test before you start treatment.
  • Females who are able to become pregnant should use effective birth control during treatment and for 1 week after your last dose. Talk to your healthcare provider about birth control methods that may be right for you.
  • Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment.
• are breastfeeding or plan to breastfeed. It is not known if Scemblix passes into your breast milk. Do not breastfeed during treatment and for 1 week after your last dose.

Scemblix may cause serious side effects, including:

• Low blood cell counts. Scemblix may cause low platelet counts (thrombocytopenia), low white blood cell counts (neutropenia), and low red blood cell counts (anemia). Your healthcare provider will do blood tests to check your blood cell counts every 2 weeks for the first 3 months of treatment and then monthly or as needed during treatment. Tell your healthcare provider right away if you have unexpected bleeding or easy bruising, blood in your urine or stools, fever, or any signs of an infection.
• Pancreas problems. Scemblix may increase enzymes in your blood called amylase and lipase, which may be a sign of pancreatitis. Your healthcare provider may do blood tests monthly or as needed during treatment to check for problems with your pancreas. Tell your healthcare provider right away if you have sudden stomach-area pain or discomfort, nausea, or vomiting.
• High blood pressure. Your healthcare provider may check your blood pressure and treat any high blood pressure during treatment as needed. Tell your healthcare provider if you develop elevated blood pressure or symptoms of high blood pressure including confusion, headaches, dizziness, chest pain, or shortness of breath.
• Allergic reaction. Stop taking Scemblix and get medical help right away if you get any signs or symptoms of an allergic reaction, including:
  • trouble breathing or swallowing
  • swelling of the face, lips, or tongue
  • skin rash or flushing of your skin
  • feeling dizzy or faint
  • fever
  • fast heartbeat
• Heart and blood vessel (cardiovascular) problems. Scemblix may cause heart and blood vessel problems, including heart attack, stroke, blood clots or blockage of your arteries, heart failure, and abnormal heartbeat, which can be serious and may sometimes lead to death. These heart and blood vessel problems can happen in people with risk factors or a history of these problems, and/or previously treated with other TKI medicines. Your healthcare provider may monitor you for heart and blood vessel problems and treat you as needed during treatment. Tell your healthcare provider or get medical help right away if you get:
  • shortness of breath
  • chest pain or pressure
  • feeling like your heart is beating too fast or you feel abnormal heartbeats
  • swelling in your ankles or feet
  • dizziness
  • weight gain
  • numbness or weakness on one side of your body
  • decreased vision or loss of vision
  • trouble talking
  • pain in your arms, legs, back, neck or jaw
  • headache
  • severe stomach area pain

The most common side effects include:

• nose, throat, or sinus (upper respiratory tract) infections
• muscle, bone, or joint pain
• tiredness
• nausea
• rash
• diarrhea
• decreased blood platelet counts, white blood cell counts, and red blood cell counts
• increased blood fat (triglycerides) levels
• increased blood creatine kinase levels
• increased blood liver enzyme levels
• increased blood pancreas enzyme (amylase and lipase) levels

Your healthcare provider may change your dose or temporarily or permanently stop treatment if you have certain side effects.

Scemblix may cause fertility problems in females. This may affect your ability to have a child. Talk to your healthcare provider if this is a concern for you.

These are not all of the possible side effects. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800 FDA-1088.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Scemblix and other medicines may affect each other causing side effects.

Especially tell your doctor if you take:

• antibiotics - clarithromycin
• antifungal medicines - itraconazole, ketoconazole, posaconazole, voriconazole
• HIV medicines containing atazanavir, darunavir, indinavir, nelfinavir, ritonavir, saquinavir, cobicistat
• mifepristone
• nefazodone