Miplyffa’s approval: How long until the first medicine for Niemann-Pick is available in your country?

Niemann-Puck Disease Type C (NPC) is a rare genetic condition affecting about 1 in 45,000-286,000 live births worldwide ¹. Up until this year, the treatment options for this progressive disease were largely symptomatic ².

With the recent FDA approval of Miplyffa (arimoclomol) and Aqneursa (levacetylleucine), NPC patients worldwide now have a new hope to slow the progression of the disease ³.

While this is good news, what does it mean for Niemann-Pick Type C patients outside the USA? When can you expect Miplyffa's EMA approval, MHRA approval, and more? And what are your options in the meantime?

What is the use of Miplyffa?

At the time of receiving its FDA approval, Miplyffa (arimoclomol) was the first and only treatment to address the neurological manifestations of Niemann-Pick disease type C ⁴. The medicine is intended for use together with miglustat, and is applicable for adults as well as children over the age of 2.

Just a few days after arimoclomol got its FDA approval, a second treatment for NPC was also approved - Aqneursa (levacetylleucine) ⁵.

Unlike previous symptomatic treatments, Miplyffa (arimoclomol) aims to halt the disease progression ⁶.

What is the mechanism of action of arimoclomol?

While it is not fully understood how Miplyffa (arimoclomol) works, it is thought to enhance lysosomal function. This potentially reduces cholesterol accumulation and activates genes that regulate lysosomal activity ⁷. 

NPC is caused by mutations in the NPC1 or NPC2 genes, leading to the buildup of cholesterol and lipids in cells, particularly in the brain. This results in impaired speech, movement, cognition, swallowing, and fine motor skills. By reducing cholesterol accumulation, arimoclomol may be able to alleviate the neurological symptoms of NPC.

What is Miplyffa's success rate in clinical trials?

Arimoclomol's FDA approval was based, among others, on the results of a 12-month clinical trial. In this trial, the effectiveness of Miplyffa was evaluated using the NPC Clinical Severity Scale (R4DNPCCSS).

The key reported outcome of this trial was:

• Miplyffa, taken in combination with miglustat, stopped disease progression through 12 months of treatment. This was seen in the 0.2 point decrease on the R4DNPCCSS scale. Patients treated only with miglustat showed a 1.9 points of progression on the same scale ⁶.

When is Miplyffa's EMA approval expected?

While Miplyffa's manufacturer had submitted a marketing authorization application to the EMA already back in 2022, it was subsequently withdrawn ⁸. As of November 2024, there is no new application for Miplyffa's EMA approval. Unfortunately, this means that it's unlikely that arimoclomol will be available to patients in Europe any time soon.

A typical review by EMA takes about 210 days. If theoretically a new marketing authorization application for arimoclomol is submitted in early 2025, then the medicine could be approved in the EU by the end of 2025. However, whether Zevra will submit a new EMA approval application and when, remains to be seen.

Is Miplyffa getting MHRA approval?

As of November 2024, there is no marketing authorization application for Miplyffa submitted to the MHRA in the UK. However, one is also not necessary for the medicine to get approval in the UK. According to post-Brexit regulations, the MHRA may grant approval to a medicine on the basis of its approval from a trusted regulatory body (which the FDA is considered to be).

Therefore, if the MHRA decides to, it could make arimoclomol available to NPC patients in the UK almost immediately. This would not automatically make it available on the NHS, as a NICE assessment is required for that. However, it at least would be possible for patients to access the treatment locally. Whether the MHRA takes this approval route for arimoclomol, only time will tell.

How to get Miplyffa before it's available in your country

If you or a loved one are affected by Niemann-Pick disease type C, it may be discouraging to hear that you need to wait to get the treatment, while it's already available to patients in the USA. The good news is, you don't have to wait. You can explore several safe, legal, and efficient methods to access Miplyffa (arimoclomol) prior to its EMA approval, MHRA approval, or any other local authorization.

One option is related to Expanded Access Programs, and another - to buying and importing Miplyffa as a Named Patient. Here's what each option means.

Miplyffa Expanded Access Program

The medicine's manufacturer, Zevra, currently has a limited Expanded Access Program in which over 150 patients are enrolled, across multiple locations. If you're based in a country where Miplyffa is not approved yet, you could try to join this Expanded Access Program.

Please keep in mind that there may be certain limitations to the program, and not all patients or locations may be eligible. However, it's always worth checking. If you're interested in the Miplyffa Expanded Access Program, your treating doctor will have to reach out to [email protected] on your behalf.

Buy Miplyffa for personal use

If the Expanded Access Program for Miplyffa is not an option for you, you can also directly buy and import the medicine for your personal use. This is allowed under the Named Patient regulation in effect in most countries.

The regulation applies when a medication is not yet approved or available in the patient's country, and:

• it is approved elsewhere;
• it has no local alternatives, and
• it is for personal use.

This process requires a prescription from your treating doctor. Other documentation requirements may apply, depending on your country.

Do you want to use the Named Patient Import regulation to get Miplyffa before its approval in Europe, the UK, or another non-US country? You will first need to consult your treating doctor and get a suitable prescription.

Already have a prescription? Share it with our team at Everyone.org, so we can support you with buying arimoclomol immediately.

References:

1. Niemann-Pick disease type C. Orphanet, Accessed 28 November 2024.
2. Soliman, Megan, and Rachael Ajmera. Niemann-Pick Disease Type C: Symptoms, Causes, Treatment. Healthline, 11 June 2024.
3. After prior snub, Zevra clinches FDA approval for rare lysosomal storage disorder drug Miplyffa. Fierce Pharma, Accessed 28 November 2024.
4. Zevra Therapeutics: FDA Approves First Treatment for Niemann-Pick Disease Type C, MIPLYFFA™ (Arimoclomol); Investigational in the EU. NPUK, Accessed 28 November 2024.
5. FDA approves first two drugs for rare Niemann–Pick disease. Nature, 27 September 2024.
6. Zevra Therapeutics’ MIPLYFFA™ (arimoclomol) Receives U.S. FDA Approval as Treatment for Niemann-Pick Disease Type C. Zevra Therapeurics, Accessed 28 November 2024.
7. Miplyffa. Drugs.com, Accessed 28 November 2024.
8. Orphazyme announces withdrawal of European Marketing Authorisation Application for arimoclomol for the treatment of Niemann-Pick disease type C. NPUK, 22 March 2022.