Erdafitinib's EMA approval: Where are we five years after its first global approval

The highest rates of bladder cancer in the world are found in Europe ¹. With only 8% of late-diagnosed patients expected to survive beyond five years, the need for innovative therapies is urgent ⁶. In this context, Balversa's accelerated FDA approval in 2019 was positive news and a sign of hope for bladder cancer patients.

Unfortunately, 5 years later, Balversa (erdafitinib) is still not available to patients outside the US.

If you're based in Europe or the UK, here's everything you need to know about erdafitinib's EMA and MHRA approval timelines. Including ways to get the treatment before it's locally available.

What is Balversa(erdafitinib) for?

Balversa is a prescription medicine for adults with bladder cancer (urothelial cancer) that has spread or cannot be removed by surgery. The treatment is intended for patients with an abnormal FGFR gene, who have had at least one round of systemic therapy that did not work or stopped working.

Balversa (erdafitinib) is not suitable for patients who are eligible for and have not received prior PD-1 or PD-L1 inhibitor therapy ².

What is the success rate of Balversa?

According to the clinical study results reported for Balversa(erdafitinib), this is its success rate:

• 30.1% of patients treated with erdafitinib had their tumor partially shrink, compared with 7.7% of people on chemotherapy;
• 5.1% of patients treated with erdafitinib had their tumor disappear completely, compared with 0.8% of people on chemotherapy;
• The median overall survival with erdafitinib was 12.1 months vs 7.8 months with chemotherapy ³.

What is the time to response to erdafitinib?

According to reported clinical trial data, the median time to reponse to erdafitinib was 1.4 months ⁴.

How long do you take Balversa for?

You can take Balversa (erdafitinib) continuously, until tumor progression or unacceptable toxicity occur ⁵. Your treating doctor will be monitoring you closely. They will be in the best position to know whether the treatment should be paused or discontinued in your case.

Is Balversa (erdafitinib) EMA approved?

As of March 2024, not yet. The EMA accepted a marketing authorization application for Balversa in September 2023. The application is under review, which can take up to 210 days, excluding any pauses to request additional information from the manufacturer.

Realistically, if all goes well, Balversa (erdafitinib) could be EMA approved around mid-2024.

When will erdafitinib be available in Europe?

Unfortunately, EMA approval doesn't automatically mean market availability. If erdafitinib gets a positive recommendation by the CHMP in mid-2024, it will take another 67 days for the EMA approval to become official. After that, each EU member state will start its own local procedures to make the medicine available on their market. How long these procedures takes varies greatly across countries - from 100 days in Germany to 964 days in Romania ⁶.

Therefore, depending on where you are in Europe, erdafitinib may be available to you before the end of 2024 or you may have to wait for another couple of years.

Does Balversa have UK approval?

There is currently no active application for the UK marketing authorization of Balversa(erdafitinib). Once an application is submitted, it takes a similar amount of time to approve as the EMA's 210 days. Following an MHRA approval, erdafitinib will have to undergo a NICE review to decide on its inclusion on the NHS. If the NICE's review is positive, then the medicine should be available on the UK market within 3 months.

With this in mind, it doesn't seem likely that Balversa(erdafitinib) will be available in the UK soon.

Ways to safely get erdafitinib before its EMA and MHRA approvals

If you're a bladder cancer patient, waiting for over a year to get access to Balversa may not be an option. The good news is, you don't have to wait.

There are two options available for accessing edafitinib before its approval in Europe and the UK: participating in a clinical trial or buying Balversa as an Individual Named Patient.

Join an erdafitinib clinical trial

To get quick access to Balversa (erdafitinib), you can try joining an ongoing clinical trial. Finding one isn't always straightforward, but with the right guidance from your treating doctor, it can be done. In order to participate in a clinical trial, you must meet the eligibility criteria. And you need to be aware you might be assigned to the placebo group instead of the working ingredient group in the trial.

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

• ClinicalTrials.gov: This is a database with all clinical trials in the USA. However, some of the trials are also open to international participants. At the moment, there are multiple trials involving Balversa, which are recruiting participants - including trials in Spain, Japan, and Canada.
• EUClinicaltrials.eu: This database contains 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: Both organizations support patients in finding treatment options in clinical trials.

Buy Balversa(erdafitinib) as a Named Patient

In most countries, patients are legally allowed to buy and import medicines that could improve their lives or address life-threatening conditions. Especially if these medicines are not approved or available locally yet.

If you want to access erdafitinib before its MHRA or EMA approval, this might be the fastest option for you and your doctor.

The regulation that makes this possible is known as the Named Patient Import regulation. Under this regulation, patients can import a medication not yet approved or available in their country if:

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

This process requires a prescription from the patient’s treating doctor. The doctor assumes responsibility for the treatment. Per country, specific documentation requirements can vary.

Do you want to use the Named Patient Import regulation to get erdafitinib before its approval in Europe or the UK? 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 - we can support you with buying erdafitinib immediately.

Buy Balversa

References:

1. Global, regional and national burden of bladder cancer and its attributable risk factors in 204 countries and territories, 1990–2019: a systematic analysis for the Global Burden of Disease study 2019, BMJ Global Health, Accessed 05 March 2024.
2. Homepage | BALVERSA® (erdafitinib), Accessed 5 March 2024.
3. How was BALVERSA® studied? | BALVERSA® (erdafitinib). Balversa.com, Accessed 5 March 2024.
4. Treatment approaches for FGFR-altered urothelial carcinoma: targeted therapies and immunotherapy. NCBI, 22 August 2023.
5. Dosing and Administration | BALVERSA® (erdafitinib) HCP. Balversa.com, Accessed 5 March 2024.
6. Survival Rates for Bladder Cancer. American Cancer Society, Accessed 5 March 2024.