MIPLYFFA is the first FDA-approved treatment for Niemann-Pick disease type C (NPC)

MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older.

Please see below for Important Safety Information.

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WHAT is MIPLYFFA [mye-plye’-fah]?

MIPLYFFA is prescription medicine used in combination with a drug called miglustat to treat neurological symptoms of Niemann-Pick disease type C (NPC) in patients 2 years of age and older.

IMPORTANT SAFETY INFORMATION

Before starting MIPLYFFA, tell your healthcare provider about all your medical conditions, including if you are pregnant or plan to become pregnant, breastfeeding or plan to breastfeed.

Tell your healthcare provider about all the medicines you take, including any prescription and over-the-counter medicines, vitamins, or herbal supplements. MIPLYFFA may affect how other medicines work.

What are the possible side effects of MIPLYFFA?

MIPLYFFA may cause serious side effects including:

Hypersensitivity reactions. Call your healthcare provider immediately if you get any of the following symptoms:
- urticaria (hives),
- shortness of breath,
- persistent cough, or
- facial swelling
Harm to your unborn baby. If you are of childbearing age, take precautions to prevent pregnancy. Tell your healthcare provider right away if you become pregnant or think you might be pregnant during treatment with MIPLYFFA.
Infertility. MIPLYFFA may affect your ability to have children.

The most common side effects of MIPLYFFA in patients also taking miglustat include upper respiratory tract infection, diarrhea and decreased weight.

These are not all the possible side effects of MIPLYFFA. Call your HCP for medical advice about side effects. You are encouraged to report side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Drug Interactions: MIPLYFFA can cause side effects if used together with certain drugs called OCT2 substrates. Talk to your healthcare provider about any drugs that you may be taking for other conditions.

MIPLYFFA capsules for oral use are available in the following strengths in a 90-count bottle: 47 mg, 62 mg, 93 mg, and 124 mg.

For more information, please see the Prescribing Information, including Instructions for Use.

IMPORTANT SAFETY INFORMATION

Before starting MIPLYFFA, tell your healthcare provider about all your medical conditions, including if you are pregnant or plan to become pregnant, breastfeeding or plan to breastfeed. Tell your healthcare provider about all the medicines you take, including any prescription and over-the-counter medicines, vitamins, or herbal supplements. MIPLYFFA may affect how other medicines work.... Continue Reading