Pronunciation: mye plye' fah
Generic name: arimoclomol
Dosage form: Capsules (47 mg, 62 mg, 93 mg and 124 mg)
Drug class: Miscellaneous metabolic agents

Medically reviewed by Melisa Puckey, BPharm. Last updated on Sep 24, 2024.

What is Miplyffa?

Miplyffa (arimoclomol) is a medication for Niemann-Pick disease type C (NPC) to improve neurological symptoms and stop disease progression. Miplyffa may help to improve speech, fine motor skills, body movement, swallowing, and cognition (thinking, understanding, and learning). Miplyffa capsules are taken 3 times daily and are used in combination with miglustat (Zavesca) capsules.  

Niemann-Pick disease type C is caused by a genetic mutation that affects lysosomes, which are part of the cells that transport material into cells and break them down. Changes in the genes (NPC1 or NPC2) stop cholesterol and other fatty substances (lipids) from being able to be transported inside cells. This means that lipids build up in areas of the body, including the brain, causing problems with speech, cognition, swallowing, movement, and fine motor skills. It is not fully understood how Miplyffa works but is thought to improve the function of lysosomes by reducing unesterified cholesterol in the lysosomes of NPC fibroblasts and upregulation of coordinated lysosomal expression and regulation (CLEAR) genes.

Miplyffa FDA approval was received on Sept. 20, 2024, for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. It is to be used in combination with miglustat.

AmplifyAssist is a support program by Zevra Therapeutics, Inc., for patients and caregivers with NPC who are taking Miplyffa. The program provides clinical education, insurance coverage support, access support, and help with prescription fulfillment.

Miplyffa side effects  

Miplyffa side effects include upper respiratory tract infections (stuffy nose, sneezing, sore throat), diarrhea, weight loss, tremors, hives, headache, lower respiratory tract infections, and seizures. These side effects occurred in 8% or more of patients in clinical trial 1.

Serious Miplyffa side effects are hypersensitivity reactions, including urticaria and angioedema.

This is not a complete list of side effects, and others may occur. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

Related/similar drugs

Warning 

Hypersensitivity Reactions 

Hypersensitivity reactions, including urticaria and angioedema, have been reported in patients treated with this medicine in Trial 1. Two patients (6%) reported both urticaria (hives) and angioedema (swelling of the eyes, lips, genitals, hands, and feet), and one patient (3%) experienced hives alone. These reactions occurred within the first two months of treatment. In patients who develop severe hypersensitivity reactions, discontinue Miplyffa. If a mild or moderate hypersensitivity reaction occurs, stop this medicine and treat it promptly. Monitor the patient until signs and symptoms have resolved.

Increased Creatinine without Affecting Glomerular Function 

In clinical trials of Miplyffa, mean increases in serum creatinine of 10% to 20% compared to baseline were reported. These increases occurred mostly in the first month of treatment and were not associated with changes in glomerular function. During Miplyffa treatment, use alternative measures that are not based on creatinine to assess renal function. Increases in creatinine were reversed upon Miplyffa discontinuation.

Embryofetal Toxicity

Miplyffa may cause embryofetal harm if taken during pregnancy,  based on findings from animal studies. Females should be advised of the potential risk to the fetus and consider pregnancy planning and pregnancy prevention.

Before taking this medicine

Pregnancy

Tell your healthcare provider if you are pregnant or planning to become pregnant, as Miplyffa may cause harm to your unborn baby. 

Breastfeeding 

Tell your healthcare provider if you are breastfeeding or plan to breastfeed. It is not known if Miplyffa passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby if you take Miplyffa.

Infertility 

This medicine may impair fertility in females and males of reproductive potential, based on findings from animal studies. In a animal fertility study, oral administration of arimoclomol resulted in decreased male and female fertility at 9-fold and increased pre-implantation loss at 5-fold the human exposure. It is not known if these effects are reversible.

How should I take Miplyffa?

Take or give capsules exactly as instructed by your healthcare provider.  Your healthcare provider will tell you the number of capsules to take or give for the prescribed dose.  

 Miplyffa capsules can be swallowed whole with or without food.  

If you or your child have difficulty swallowing capsules, they can be opened and: 

• can be taken or given by mouth by adding 15 mL of water, apple juice, apple sauce, yogurt, or pudding in a small cup, stirring well, and swallowing straight away.
• through a feeding tube (see package insert for full instructions with diagrams).  

The inactive ingredients are not water soluble and will remain undissolved if the contents of the capsule are added to beverages or soft food.

If you miss a dose, skip the missed dose and take or give the next dose of Miplyffa at your regularly scheduled time. 

 Miplyffa Dosing information

Miplyffa recommended dose is based on weight.

Miplyffa Dose:

8 kg to 15 kg: 47 mg three times a day  

16 kg to 30 kg: is 62 mg three times a day  

31 kg to 55 kg: is 93 mg three times a day 

Heavier than 56 kg: is 124 mg three times a day 

Miplyffa capsules are available as 47 mg, 62 mg, 93 mg and 124 mg of arimoclomol.

What other drugs will affect this medicine? 

Miplyffa is an inhibitor of the organic cationic transporter 2 (OCT2) transporter and may increase the exposure of drugs that are OCT2 substrates. When Miplyffa is used concomitantly with OCT2 substrates, monitor for adverse reactions and reduce the dosage of the OCT2 substrate. 

Other drugs may interact with Miplyffa, including prescription and over-the-counter medicines, vitamins, and herbal products. Not all possible drug interactions are listed here.

Storage

Store at 20°C to 25°C (68°F to 77°F); excursions permitted between 15°C to 30°C (59°F to 86°F) 

Store in the original container. Protect from light. 

Miplyffa Ingredients

Active ingredients: arimoclomol 

Inactive ingredients: microcrystalline cellulose (MCC) and magnesium stearate

Capsule shells: hypromellose, titanium dioxide, and one or more of the following: Brilliant Blue FCF-FD&C Blue 1, Yellow iron oxide and Red iron oxide. 

Manufacturer

Zevra Therapeutics Inc. Celebration FL, USA 

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

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