Miplyffa: A New Treatment for Niemann-Pick Disease Type C Approved on September 20, 2024
What is the name of the drug and what does it do?
Miplyffa (pronounced MYE-pli-fah), generic name arimoclomol, is a medication used to treat neurological symptoms of Niemann-Pick disease type C (NPC). NPC is a rare genetic disorder that affects the body’s ability to process cholesterol and other fats, leading to damage in the brain and other organs. Miplyffa is prescribed for adults and children aged 2 years and older and is used together with another drug called miglustat.
How does it work?
Although the exact way Miplyffa works isn’t fully understood, it is believed to help stabilize proteins in cells, reducing the damage caused by NPC. It appears to protect cells from stress and slow the progression of neurological symptoms like trouble walking, speaking, or swallowing.
What did the research discover?
Clinical trials studied Miplyffa in 50 patients with NPC, aged 2 to 19 years, over 12 months. Patients treated with Miplyffa showed slower progression of neurological symptoms compared to those who received a placebo. On average, patients taking Miplyffa had milder increases in their symptom severity scores than those in the placebo group. Additionally, patients who used both Miplyffa and miglustat experienced the most significant improvements in managing NPC symptoms like speech, swallowing, and movement. These findings suggest that Miplyffa can help slow the progression of neurological effects in NPC when combined with miglustat.
What are some of the side effects?
The most common side effects of Miplyffa include:
- Upper respiratory tract infections (colds)
- Diarrhea
- Weight loss
- Decreased appetite
- Headache
Rare but serious side effects include allergic reactions such as hives or swelling (urticaria and angioedema) and increases in creatinine levels, which may indicate kidney issues. Patients should inform their doctor if they experience unusual symptoms.
What are the dosage recommendations and how is it prescribed?
Miplyffa is taken by mouth as a capsule three times a day. The exact dose depends on body weight:
- 8–15 kg: 47 mg per dose
- 15–30 kg: 62 mg per dose
- 30–55 kg: 93 mg per dose
- Over 55 kg: 124 mg per dose
For patients who have difficulty swallowing, the capsule can be opened, and the contents mixed with water, apple juice, or soft food like applesauce or yogurt. It can also be given through a feeding tube.
Source:
U.S. Food and Drug Administration. Full prescribing information: [Label for NDA 214927]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/214927s000lbl.pdf. Accessed 2024 Nov 21.
