Vimseltinib's approval worldwide: When is the new TGCT treatment coming to you?

Last updated: 17 September 2024

Tenosynovial giant cell tumor (TGCT) is a rare tumor that typically forms in tendons or around joints. Although usually benign, it can be aggressive and significantly impact movement and quality of life.

Currently, Turalio (pexidartinib) is the only targeted therapy approved for TGCT in the USA. Unfortunately, it was not approved by the EMA in Europe ¹. However, vimseltinib is emerging as a promising new treatment for TGCT, with favorable prospects for approval by both the FDA and EMA.

Here's what you need to know about the approval timelines for patients in Europe, the UK, and the USA.

What is vimseltinib?

Vimseltinib is a medicine being studied for the treatment of Tenosynovial Giant Cell Tumor (TGCT) and other conditions. It is a selective small molecule inhibitor that targets colony-stimulating factor 1 receptor (CSF1R). CSF1R is involved in the growth and survival of cells associated with TGCT. By inhibiting CSF1R, vimseltinib aims to reduce tumor growth and inflammation.

It is currently undergoing clinical trials to determine its effectiveness and safety. If successful, vimseltinib could become a new treatment option for TGCT. Particularly for patients who do not respond to or are ineligible for existing therapies.

Vimseltinib vs pexidartinib: What's the difference?

Both vimseltinib and pexidartinib are CSF1R inhibitors intended to treat TGCT. However, there are some key differences:

Selectivity: Vimseltinib has a unique switch-control design, which helps it be more selective in targeting CSF1R. In contrast, pexidartinib also inhibits closely related kinases such as KIT, PDGFRA, PDGFRB and FLT3, in addition to CSF1R ².
Safety: Additional clinical trial data are required to compare the safety profiles of vimseltinib and pexidartinib. However, preliminary trial results indicate that vimseltinib may have a more manageable side effect profile. Notably, vimseltinib does not seem to be linked to potential liver toxicity, which is a concern associated with pexidartinib and contributed to its failure to receive EMA approval ².

How successful is vimseltinib in clinical trials?

In August 2024, the FDA granted priority review to vimseltinib's approval application on the basis of the Phase III MOTION study ⁴. The key findings of the study included:

At Week 25 of the treatment, vimseltinib had an overall response rate of 40% (compared to 0% for placebo);
Vimseltinib showed improvement in tumor volume score (67% ORR) and active range of motion (18.4% improvement). Improvements were also reported in additional measures such as pain and quality of life;
There was no evidence of severe liver toxicity associated with vimseltinib ³.

When is vimseltinib expected to get FDA approval?

According to the FDA, the expected decision date on its priority review of vimseltinib is February 17, 2025 ⁴. Clearly, this doesn't mean that FDA approval will be granted. However, at this point there is no reason to expect otherwise.

When will vimseltinib get EMA approval?

In July 2024, the EMA accepted vimseltinib's marketing authorization application ⁵. EMA decisions typically take up to seven months. This suggests potential approval by February 2025—aligning closely with the anticipated FDA approval timeline.

Of course, once the medicine is approved in Europe, it won't be immediately available in all EU countries. That process can take from a few additional months to a couple of years.

When will vimseltinib be available in the UK?

As of September 2024, the MHRA hasn't received an official marketing authorization application for vimseltinib. This, however, doesn't have to mean a longer approval waiting time for UK patients. After Brexit, the MHRA has the option to approve medicines as soon as they are vetted by another trusted agency (e.g. the FDA or EMA). If the MHRA takes this road, it could theoretically approve vimseltinib in early 2025. Afterwards, NICE would have to give a positive assessment of the medicine, so that it can become available on the NHS. Assuming the best-case scenario, where a positive NICE assessment is available at the same time as an MHRA approval, we could theoretically see vimseltinib available in the UK in the second half of 2025.

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

Are you a patient with TGCT? If your doctor is of the opinion that you might benefit from treatment with vimseltinib, you may not need to wait for its local approval. Instead, your doctor and you could consider joining a clinical trial. Or, buying and accessing the medicine via Named Patient Import. The latter option is possible as soon as vimseltinib gets an approval somewhere in the world.

Join a clinical trial

To access the new TGCT medicine, enrolling in a clinical trial is one option. However, this can be challenging, as you and your doctor will need to locate a trial that is currently accepting participants in your country. Additionally, you must meet the eligibility requirements and understand that you may be placed in the control group of the trial.

Here are some good places to start looking for ongoing vimseltinib clinical trials:

ClinicalTrials.gov: This is a database with all clinical trials in the USA. Some of the trials are also open to international participants, so it's a list worth keeping an eye on.
EUClinicaltrials.eu: This is a database with all clinical trials in the European Union. Currently, it contains limited information on trials launched before 31 January 2022. For those trials, you can refer to the EU Clinical Trials Register.
myTomorrows and FindMeCure: These organizations support patients in finding treatment options in clinical trials.

Buy vimseltinib for personal use

In most countries, patients are allowed to buy and import medicines for personal use, even if they are not approved or available in their country. The regulation making this possible is known as the Named Patient Import. There may be variations across countries in terms of the specific administrative requirements. However, in all cases these criteria must be met:

The medicine in question has market approval in another country and is not (yet) approved or available in the patient's country;
There is no alternative on the local market;
The medicine is for personal use;
The patient has a prescription from their treating doctor;
The doctor takes responsibility for the treatment. This might require different documentation from country to country.

Do you want to use the Individual Named Patient Import regulation to get vimseltinib before its FDA, MHRA, or EU approval? You will first need to consult your treating doctor and get a suitable prescription.

Already have a prescription? Our team can support you with buying vimseltinib, as soon as it gets its first approval somewhere in the world. Get in touch with us for more information.

Get in touch

References:

Turalio | European Medicines Agency (EMA). European Medicines Agency, 18 December 2020.
The MOTION Study: A Randomized, Phase III Study of Vimseltinib for the Treatment of Tenosynovial Giant Cell Tumor. Future Oncology, 18 August 2023.
Efficacy, safety, and patient-reported outcomes of vimseltinib in patients with tenosynovial giant cell tumor: Results from the phase 3 MOTION trial. Journal of Clinical Oncology, 29 March 2024.
FDA accepts Deciphera’s vimseltinib NDA for priority review. Pharmaceutical Technology, 16 August 2024.
EU Approval Sought for Vimseltinib in Tenosynovial Giant Cell Tumor. OncLive, 19 July 2024.