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Uses

What is Miplyffa used for?

Miplyffa (arimoclomol) is commonly used to help slow the worsening of a rare genetic disorder called Niemann-Pick disease.

Miplyffa may also be used for other conditions as determined by your healthcare provider.

How does Miplyffa work (mechanism of action)?

Niemann-Pick disease involves accumulation of fat in certain cells. Miplyffa helps keep cells working properly and stops them from dying in people who have fat buildup.

How is Miplyffa supplied (dosage forms)?

Miplyffa is available in the following dosage forms that are taken by mouth.

  • 47 mg oral capsules
  • 62 mg oral capsules
  • 93 mg oral capsules
  • 124 mg oral capsules

How should I store Miplyffa?

MIplyffa should be stored in the original container at room temperature, between 68 F to 77 F (20 C to 25 C). It can be exposed to temperatures between 59 F to 86 F (15 C to 30 C) for shorter periods of time, such as when transporting it. Store in a cool, dry place. Protect from light. 

Side Effects

What are the most common side effects of Miplyffa?

The most common side effects of Miplyffa are listed below. Tell your healthcare provider if you have any of these side effects that bother you.

  • Infection in the nose or throat, sore throat, or common cold symptoms
  • Diarrhea
  • Weight loss

There may be other side effects of Miplyffa that are not listed here. Contact your healthcare provider if you think you are having a side effect of a medicine. In the U.S., you can report side effects to the FDA at www.fda.gov/medwatch or by calling 800-FDA-1088. In Canada, you can report side effects to Health Canada at www.health.gc.ca/medeffect or by calling 866-234-2345.

What are the serious side effects of Miplyffa?

While less common, the most serious side effects of Miplyffa are described below, along with what to do if they happen.

Severe Allergic Reactions. Miplyffa may cause allergic reactions, which can be serious. Stop taking Miplyffa and get help right away if you have any of the following symptoms of a serious allergic reaction.

  • Breathing problems or wheezing
  • Racing heart
  • Fever or general ill feeling
  • Swollen lymph nodes
  • Swelling of the face, lips, mouth, tongue, or throat
  • Trouble swallowing or throat tightness
  • Itching, skin rash, or pale red bumps on the skin called hives
  • Nausea or vomiting
  • Dizziness, feeling lightheaded, or fainting
  • Stomach cramps
  • Joint pain

Warnings & Precautions

Who should not use Miplyffa?

Allergies to Ingredients. People who are allergic to any of the following should not take Miplyffa.

  • Arimoclomol
  • Miplyffa
  • Any of the ingredients in the specific product dispensed

Your pharmacist can tell you all of the ingredients in Miplyffa.

What should I know about Miplyffa before using it?

Do not take Miplyffa unless it has been prescribed to you by a healthcare provider. Take it as prescribed.

Do not share Miplyffa with other people, even if they have the same condition as you. It may harm them.

Keep Miplyffa out of the reach of children.

Miplyffa should be swallowed whole. If you have difficulty swallowing the capsule or you have a feeding tube, the capsules can be opened and mixed with liquid or soft foods and then taken. Ask your pharmacist for specific directions to do this if you are not able to swallow the capsules or if you have a feeding tube.

If you can get pregnant, talk with your healthcare provider about ways to prevent pregnancy while taking Miplyffa. 

Miplyffa may affect your ability to become pregnant or get your partner pregnant. Talk with your healthcare provider if you have any concerns.

Miplyffa may increase levels of creatinine (a chemical your muscles make). Creatinine is usually used to see how well your kidneys are working. Miplyffa seems to increase creatinine without affecting your kidneys. Your healthcare provider may need to use other ways to check to see how well your kidneys are working. Your dose of Miplyffa depends on how much you weigh and on how well your kidneys are working. Keep all appointments, so your healthcare provider can give you the right dose.

What should I tell my healthcare provider before using Miplyffa?

Tell your healthcare provider about all of your health conditions and any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. This will help them determine if Miplyffa is right for you.

In particular, make sure that you discuss any of the following.

Current and Past Health Conditions. Tell your healthcare provider if you have kidney problems.

Other Medicines and Supplements. Miplyffa may interact with other medicines and supplements. Before taking Miplyffa, tell your healthcare provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. See the Interactions section for more details.

Pregnancy. Miplyffa may cause harm to an unborn baby. Tell your healthcare provider if you are pregnant, think you are pregnant, or plan to become pregnant. Your healthcare provider will advise you if you should take Miplyffa while you are pregnant or trying to get pregnant.

Breastfeeding. It is not known if Miplyffa passes into breast milk. Tell your healthcare provider if you are breastfeeding or plan to breastfeed. Your healthcare provider will advise you if you should take Miplyffa while breastfeeding.

How can I get more information about Miplyffa?

For more information about Miplyffa, you can visit the manufacturer’s website at www.miplyffa.com or call them at 888-668-4198.

Interactions

Does Miplyffa interact with foods or drinks?

There are no known interactions between Miplyffa and foods or drinks.

It is unknown if drinking alcohol will affect MIplyffa.

Does Miplyffa interact with other medicines (drug interactions)?

Miplyffa may alter the blood levels of some other medicines. Always tell your healthcare provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. Do not take double or extra doses.

Does interact with other drugs you are taking?

Enter your medication into the WebMD interaction checker

Overdose/Missed Dose

What should I do if I accidentally use too much Miplyffa?

If you or someone else has used too much Miplyffa, get medical help right away, call 911, or contact a Poison Control center at 800-222-1222.

What should I do if I miss a dose of Miplyffa?

If you miss a dose, skip that dose. Take the next dose when you normally would. Do not take double or extra doses.