Miplyffa
Miplyffa (arimoclomol) is the first FDA-approved medication used to treat neurological symptoms (e.g., muscle weakness, balance problems, difficulty speaking) associated with Niemann-Pick disease type C (NPC) in children and adults ages 2 and older. This medication is available as an oral capsule that’s taken by mouth three times a day, along with another medication called miglustat. Common side effects include upper respiratory tract infections, diarrhea, and weight loss.
What is Miplyffa (arimoclomol)?
What is Miplyffa (arimoclomol) used for?
- Neurological symptoms (e.g., muscle weakness, balance problems, difficulty speaking) of Niemann-Pick disease type C (NPC) in children and adults ages 2 and older, taken together with miglustat
How Miplyffa (arimoclomol) works
NPC is a genetic condition where the body can’t break down fats properly. Usually, a part of the cell called the lysosome helps with this. But people with NPC, have faulty proteins that cause fats to build up in cells.
Miplyffa (arimoclomol) is a heat shock protein amplifier. Heat shock proteins are made by the body to help fix faulty proteins. While the exact way this medication works is unknown, it's thought to boost the activity of heat shock proteins, which help fix the faulty proteins that cause NPC. As a result, this helps the lysosomes manage the fat buildup, which can then reduce symptoms of NPC.
Drug facts
Side effects of Miplyffa (arimoclomol)
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
Common Side Effects
- Upper respiratory tract infection (31%)
- Diarrhea (23%)
- Weight loss (15%)
Less Common Side Effects
- Reduced appetite
- Headache
- Tremors
- Lower respiratory tract infection
- Seizure
Side effects listed might not be from Miplyffa (arimoclomol) alone, as studies also included miglustat.
Miplyffa (arimoclomol) serious side effects
Contact your healthcare provider immediately if you experience any of the following.
- Severe allergic reactions: hives; trouble breathing; swelling of the lips, face, or throat
The following Miplyffa (arimoclomol) side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions or angioedema—skin rash, itching or hives, swelling of the face, eyes, lips, tongue, arms, or legs, trouble swallowing or breathing
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
- Cough
- Diarrhea
- Headache
- Loss of appetite with weight loss
- Runny or stuffy nose
- Sore throat
- Tremors or shaking
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Pros and cons of Miplyffa (arimoclomol)
Pros
- Taken by mouth
- Can be taken with or without food
- Capsule can be opened and mixed with certain liquids and foods for those who have trouble swallowing
- Approved for ages 2 and older
Cons
- Doesn’t cure NPC
- Must be taken three times a day
- Can cause side effects such as weight loss, diarrhea, and headaches
- Must be taken together with miglustat; unclear whether it can treat NPC on its own
Pharmacist tips for Miplyffa (arimoclomol)
- Miplyffa (arimoclomol) must be taken three times a day in order for it to work properly. You can take it with or without food.
- Consider keeping Miplyffa (arimoclomol) and miglustat close to each other to remind you that they’re taken for the same condition. You can even take them at the same time to make sure both medications are taken appropriately.
- If you’re having trouble swallowing the Miplyffa (arimoclomol) capsule, open the capsule carefully and sprinkle the contents into 15 mL of water, apple juice, or soft food (apple sauce, pudding, or yogurt) in a small cup. Stir the mixture for 15 seconds then eat or drink it right away.
- If using a feeding tube through the nose or the stomach, open the Miplyffa (arimoclomol) capsule and sprinkle the contents into 20 mL of water only. Don’t use any other liquid. Stir the mixture for 15 seconds and give it immediately through the feeding tube. Afterwards, flush it out with 5 mL of water.
- If you miss a dose of Miplyffa (arimoclomol), skip it and take a dose at your next scheduled time. Don’t double up on a dose since that can increase side effects from the medication.
- Miplyffa (arimoclomol) can interact with certain medications, like metformin, and can raise their levels in the body, leading to more harmful side effects. Let your prescriber know about everything that you’re taking, even if it’s over the counter, to help check for medication interactions.
- Miplyffa (arimoclomol) can cause some severe allergic reactions. Talk to your healthcare professional if you experience symptoms such as hives or swelling in the lips, face, or throat.
- Store Miplyffa (arimoclomol) at room temperature in a dry and safe place away from children or pets. Keep it in its original container and protect it from light.
Common questions about Miplyffa (arimoclomol)
Niemann-Pick disease type C (NPC) is a very rare genetic condition that typically affects children and young adults. It’s caused by a mutation which makes it hard for the body to transport cholesterol and fats within cells. This leads to fat build up in the body. When fats build up in the brain, neurological symptoms can occur, such as muscle weakness, balance problems, difficulty speaking and walking, and problems with learning and memory. Miplyffa (arimoclomol) is the first FDA-approved medication available to treat these symptoms.
No. Miplyffa (arimoclomol) shouldn’t be taken by itself for NPC. It’s only FDA approved when taken together with miglustat because it was studied that way. Though miglustat on its own is approved in Europe to slow the worsening of neurological symptoms in NPC. Speak to your prescriber if you have questions about these two medications.
In a clinical study, Miplyffa (arimoclomol) was shown to delay neurological symptoms of NPC after 1 year. This was measured by using a score which kept track of swallowing, walking, speaking, and other muscle movements. Higher scores mean a more severe condition. At the end of the study, people who took Miplyffa (arimoclomol) with miglustat had lower scores compared to people who took placebo with miglustat. This showed that taking Miplyffa (arimoclomol) with miglustat can slow down the worsening of neurological symptoms.
No. Miplyffa (arimoclomol) doesn’t cure Niemann-Pick disease type C. In fact, there isn’t a cure for this condition. But this medication is used to help improve the neurological symptoms linked to this condition and slow it down from getting worse.
Yes. In studies, people who experienced this side effect typically lost an average of 6% of their body weight over the course of 1 to 2 months. Their body weight stabilized without any extra treatment or medication, and they were able to keep taking Miplyffa (arimoclomol) without further weight loss. Also, it’s important to know that the people in the studies were also taking miglustat with Miplyffa (arimoclomol), so weight loss might not be due to Miplyffa (arimoclomol) alone.
Talk to your healthcare professional before taking Miplyffa (arimoclomol) during pregnancy. It hasn't been studied in human pregnancy, but some animal studies showed that this medication can cause miscarriages and birth defects. To be safe, it’s best to avoid becoming pregnant while taking this Miplyffa (arimoclomol). There are many ways to do so, such as birth control.
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Risks and warnings for Miplyffa (arimoclomol)
Miplyffa (arimoclomol) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Allergic Reactions
Miplyffa (arimoclomol) can cause some serious allergic reactions, such as hives and angioedema. Angioedema is a life-threatening reaction causing swelling of the lips, throat, and face that can lead to trouble breathing. In clinical trials, these allergic reactions occurred within the first two months of treatment. If you experience symptoms of an allergic reaction, stop this medication and get medical help right away.
Harm to unborn baby
Miplyffa (arimoclomol) hasn’t been studied in pregnant people, but animal studies suggest it might raise the risk of miscarriages and birth defects. To be safe, it’s best to avoid pregnancy while taking this medication. Contact your prescriber if you have any questions or concerns about this medication in pregnancy.
Abnormal kidney labs
In clinical studies, Miplyffa (arimoclomol) caused an increase in serum creatinine levels, which can be a marker for kidney health. This typically happened in the first month of treatment and didn’t cause further kidney problems. But it generally went back to normal after the medication was stopped.
Miplyffa (arimoclomol) dosage
Capsule| Dosage | Quantity | Price as low as | Price per unit |
|---|---|---|---|
| 47mg | 30 capsules | $13,192.68 | $439.76 |
| 62mg | 90 capsules | $52,173.84 | $579.71 |
| 93mg | 30 capsules | $26,092.92 | $869.76 |
| 124mg | 90 capsules | $104,335.68 | $1,159.29 |
Typical dosage for Miplyffa (arimoclomol)
Miplyffa (arimoclomol) is available as an oral capsule that’s taken with another medication called miglustat.
The dose depends on your weight:
- 18 lbs to 33 lbs: The typical dose is 47 mg by mouth three times a day.
- More than 33 lbs to 66 lbs: The typical dose is 62 mg by mouth three times a day.
- More than 66 lbs to 121 lbs: The typical dose is 93 mg by mouth three times a day.
- More than 121 lbs: The typical dose is 124 mg by mouth three times a day.
Your dose might differ if you have kidney problems.
How much does Miplyffa (arimoclomol) cost?
$13,192.68
What are alternatives to Miplyffa (arimoclomol)?
Miplyffa (arimoclomol) images
