What is MIPLYFFA [mye-plye’-fah]?
MIPLYFFA is a 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.
MIPLYFFA is an oral medication that can be taken at home
- MIPLYFFA capsules are taken 3x per day, either by swallowing the whole capsule, by adding the contents to a small amount of water, apple juice, or soft foods, or via a feeding tube
- MIPLYFFA can be taken with or without food
- Dosing for MIPLYFFA is based on body weight and should be determined by your doctor
- If not taking whole, be sure to take the medication immediately after preparation
For more details about dosing and administration, please talk to your doctor or refer to the Instructions for Use.
How MIPLYFFA may help
In the clinical trial, MIPLYFFA in combination with miglustat stopped disease progression at 12 months as measured by the R4DNPCCSS. The R4DNPCCSS measures changes in ambulation, speech, swallow, and fine motor skills, the areas identified as some of the most important to measure by NPC expert clinicians, caregivers, and patients. Download the NPC Assessment Guide
The domains measured in the R4DNPCCSS were determined to be some of the most important measurements to assess NPC symptom progression.
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.
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.
