Safety and effectiveness of MIPLYFFA were studied in a 12-month multi-center, randomized, double-blind, placebo controlled trial in patients with NPC aged 2-19 years.
*Throughout the trial, patients stayed on their ongoing prescribed background treatment, including miglustat and symptom management. Randomization was stratified by miglustat treatment, which is considered routine clinical care in some countries. In Trial 1, 76% of patients in the MIPLYFFA group and 81% of those in the placebo group received miglustat as part of their routine care.
There were insufficient data to determine the effectiveness of the use of MIPLYFFA without miglustat for treatment of the neurological manifestations in patients with NPC.
Side Effects
The most common adverse reactions in Trial 1 (≥15%) in MIPLYFFA-treated patients who also received miglustat were upper respiratory tract infection, diarrhea, and decreased weight.
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.
