Arimoclomol (oral route)


    Brand Name

    US Brand Name

    Mipylffa


    Description

    Arimoclomol is used in combination with miglustat to treat the neurologic symptoms of Niemann-Pick disease type C (NPC).

    This medicine is available only with your doctor's prescription.

    This product is available in the following dosage forms:

    • Capsule

    Before Using

    In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:

    Allergies

    Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

    Pediatric

    Appropriate studies have not been performed on the relationship of age to the effects of arimoclomol in children younger than 2 years of age. Safety and efficacy have not been established.

    Geriatric

    Appropriate studies on the relationship of age to the effects of arimoclomol have not been performed in the geriatric population.

    Breastfeeding

    There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

    Drug Interactions

    Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. When you are taking this medicine, it is especially important that your healthcare professional know if you are taking any of the medicines listed below. The following interactions have been selected on the basis of their potential significance and are not necessarily all-inclusive.

    Using this medicine with any of the following medicines is usually not recommended, but may be required in some cases. If both medicines are prescribed together, your doctor may change the dose or how often you use one or both of the medicines.

    • Dalfampridine
    • Dofetilide
    • Metformin

    Other Interactions

    Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.

    Other Medical Problems

    The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:

    • Kidney disease—Use with caution. The effects may be increased because of slower removal of the medicine from the body.

    Proper Use

    Take this medicine exactly as directed by your doctor. Do not take more of it, do not take it more often, and do not take it for a longer time than your doctor ordered. To do so may increase the chance of side effects.

    This medicine comes with a patient instructions. Read and follow these instructions carefully. Ask your doctor if you have any questions.

    You may take this medicine with or without food. Swallow the capsule whole.

    If you are unable to swallow the capsule whole:

    • Open the capsule and sprinkle the entire contents into 15 milliliters (mL) of water or apple juice of 15 mL of soft food (eg, applesauce, pudding, or yogurt). Do not mix with any other liquids.
    • Stir the mixture for 15 seconds. Swallow the mixture right away. Do not save the mixture for later use.

    You may also give the medicine through a nasogastric tube or gastric tube:

    • Open the capsule and sprinkle the contents into a cup with 20 mL water. Do not mix with any other liquids.
    • Stir the mixture for 15 seconds. Pull the plunger to withdraw the whole mixture into the feeding tube syringe. Attach the syringe to the feeding tube. Slowly push the plunger to give the mixture right away.
    • Do not save the mixture for later use. Add 5 mL of water and flush the feeding tube.

    Dosing

    The dose of this medicine will be different for different patients. Follow your doctor's orders or the directions on the label. The following information includes only the average doses of this medicine. If your dose is different, do not change it unless your doctor tells you to do so.

    The amount of medicine that you take depends on the strength of the medicine. Also, the number of doses you take each day, the time allowed between doses, and the length of time you take the medicine depend on the medical problem for which you are using the medicine.

    • For oral dosage form (capsules):
      • For Niemann Pick disease type C:
        • Adults and children 2 years of age and older—Dose is based on body weight and must be determined by your doctor.
          • Weighing more than 55 kilograms (kg)—124 milligrams (mg) 3 times a day.
          • Weighing 30 kg to 55 kg—93 mg 3 times a day.
          • Weighing 15 kg to 30 kg—62 mg 3 times a day.
          • Weighing 8 kg to 15 kg—47 mg 3 times a day.
        • Children younger than 2 years of age—Use and dose must be determined by your doctor.

    Missed Dose

    If you miss a dose of this medicine, skip the missed dose and go back to your regular dosing schedule. Do not double doses.

    Storage

    Keep out of the reach of children.

    Do not keep outdated medicine or medicine no longer needed.

    Ask your healthcare professional how you should dispose of any medicine you do not use.

    Store the medicine in a closed container at room temperature, away from heat, moisture, and direct light. Keep from freezing.

    Store in the original container.


    Precautions

    It is very important that your doctor check your or your child's progress at regular visits to make sure that this medicine is working properly. Blood tests may be needed to check for unwanted effects.

    Using this medicine while you are pregnant can harm your unborn baby. Use an effective birth control during treatment with this medicine. If you think you become pregnant while using the medicine, tell your doctor right away.

    This medicine may cause serious allergic reactions, including angioedema and urticaria, which can be life-threatening and require immediate medical attention. Check with your doctor right away if you have itching, large, hive-like swelling on the face, eyelids, lips, tongue, throat, hands, legs, feet, or sex organs, redness of the skin, or skin rash.

    If you plan to have children, talk with your doctor before using this medicine. Some men and women using this medicine have become infertile (unable to have children).

    Do not take other medicines unless they have been discussed with your doctor. This includes prescription or nonprescription (over-the-counter [OTC]) medicines and herbal or vitamin supplements.


    Side Effects

    Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

    Check with your doctor immediately if any of the following side effects occur:

    More common

    • Body aches or pain
    • chills
    • cough
    • ear congestion
    • fever
    • headache
    • large, hive-like swelling on the face, eyelids, lips, tongue, throat, hands, legs, feet, or sex organs
    • loss of voice
    • seizures
    • shakiness in the legs, arms, hands, or feet
    • sneezing
    • sore throat
    • stuffy or runny nose
    • trembling or shaking of the hands or feet
    • unusual tiredness or weakness

    Less common

    • Itching
    • redness of the skin

    Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

    More common

    • Decreased appetite
    • decreased weight
    • diarrhea

    Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

    Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.






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