Revumenib’s approval in Europe and beyond: When is the breakthrough treatment for leukemia coming to you?

Last updated: 28 November 2024

Revumenib’s approval in Europe and beyond: When is the breakthrough treatment for leukemia coming to you?

You can legally access new medicines, even if they are not approved in your country.

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Revumenib's recent FDA approval marks a significant breakthrough in the treatment of KMT2A-rearranged acute leukemia. This rare and aggressive form of leukemia affects children and adults, including those whose acute myeloid leukemia (AML) develops as a side effect of chemotherapy for another cancer.

As the first targeted therapy for this type of leukemia, revumenib offers new hope for patients and their families. However, it is currently only available in the USA. When will this novel treatment be available to patients in Europe, the UK, and other regions worldwide?

What is revumenib used for?

Revumenib's indication is for relapsed or refractory acute leukemia with a KMT2A translocation. It can be used in both adults and children over the age of 1 1.

KMT2A abnormalities are present in 5-15% of the cases of acute lymphoblastic leukemia (ALL), and in 3% of adult cases with acute myeloid leukemia (AML) 2. As such, revumenib is not applicable for all leukemia cases, but it does address a challenging subset that is often resistant to standard therapies.

How does revumenib work for acute leukemia?

Revumenib is a menin inhibitor - the first medicine of this class indicated for the treatment of leukemia. The way it works is by blocking the menin-KMT2A interaction, which is critical in driving the proliferation of leukemia cells with this genetic abnormality. In this way, the medicine aims to help some patients achieve remission and proceed to potentially curative treatments like stem cell transplantation 3.

What is the success rate of revumenib in clinical trials?

Revuforj (revumenib) received its FDA approval on the basis of the results from the AUGMENT-101 trial. The key reported outcomes of the trial were:

  • 63% of patients had a partial or complete response to the treatment;
  • 21.2% of patients treated with revumenib achieved complete remission or complete remission with partial hematological recovery;
  • The median time to complete remission was 1.9 months;
  • The median duration of response was 6.4 months 4.

According to Dr. Ghayas C. Issa from MD Anderson Cancer Center, revumenib shows promise as a paradigm-changing therapy due to its ability to achieve deep responses, maintain remission after stem cell transplant, and allow for extended use with a manageable safety profile​ 5.

When will revumenib get EMA approval?

As of November 2024, there is no active marketing authorization application for revumenib in the EU. Unfortunately, this means that it's unlikely to be available to patients in Europe soon.

Typically, an EMA approval review takes up to 210 days. Theoretically, if revumenib's manufacturer submits an EMA approval application at the beginning of 2025, the medicine could be approved within the same year. However, whether that will happen, remains to be seen.

When will revumenib be available in the UK?

That's difficult to say. As of November 2024, no application has been submitted for the approval of revumenib by the MHRA. However, this isn't necessarily required. According to post-Brexit regulations, the MHRA may approve a medicine on the basis of another regulatory body's approval (e.g. the FDA). At least in theory, if the MHRA decides to go down that route, revumenib could be available in the UK even before it's available in Europe. However, we'll have to wait and see if that happens.

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

If you or a loved one have acute leukemia with KMT2A translocation, you may feel discouraged by the prospect of waiting for revumenib to become available in your country. The good news is, you don't have to wait. You have two safe, legal, and quick methods to access Revuforj (revumenib) 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 revumenib as a Named Patient. Here's what each option means.

Revumenib Expanded Access Program

The medicine's manufacturer, Syndax, has a Compassionate Use (Expanded Access) Program for patients based in a country where the medicine is not approved yet. In order for you to be considered for this program, your treating doctor would have to reach out to Syndax via [email protected].

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 and, according to the company's website, you will get a decision within 5 business days.

Buy revumenib as a Named Patient

If the Expanded Access Program for revumenib 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 revumenib before its approval in Europe, the UK, or another country outside the USA? 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 revumenib immediately.