How to access pirtobrutinib before its approval in Europe & the UK

In early 2023, Jaypirca (pirtobrutinib) gained the FDA's approval for treating relapsed or refractory mantle cell lymphoma after two prior lines of therapy ¹. Mantle cell lymphoma (MCL) is a rare type of B-cell non-Hodgkin lymphoma. It affects around 1 in 200,000 people per year. Like many rare diseases, there weren't many treatment options available before.

For all patients with this rare disease, any new treatment option is a major milestone. However, while pirtobrutinib has been in the US for almost a year, European patients must wait for EU approval.

What if you can't wait? In this article, we'll explore the legal and safe ways to access pirtobrutinib before it's fully approved by the EMA.

Pirtobrutinib approval in Europe: What's the status?

Good news! Jaypirca (pirtobrutinib) is likely to get full marketing authorization in the EU. The medicine was recommended for conditional marketing authorization by the CHMP in April 2023 ³.

The less positive news is that it's unclear when the marketing authorization is going to be granted ².

Normally, if the CHMP recommends it, the European Commission should approve a medicine within 67 days. As of October 2023 (>100 days after the recommendation), this is still not the case for Jaypirca.

Approval doesn't equal availability

Unfortunately, pirtobrutinib won't be available right away in Europe, even if it gets approved. Each member state must make decisions about local approval, prices, and health insurance coverage. As a result, each country within Europe is likely to make Jaypirca available at a different time.

Oncology drugs take on average 545 days to become available in Europe after EMA approval. This timeline is dramatically different per country. It ranges from 100 days in Germany to 964 days in Romania ⁴.

What about pirtobrutinib approval in the UK?

After Brexit, the MHRA and NICE don't have to follow the EMA's marketing authorization timelines. This means that they can independently approve a medicine for use in the UK.

When it comes to pirtobrutinib, however, the MHRA and NICE have not made any approval decisions yet. The medicine is in review and no timeline is provided for the communication of an outcome ⁵.

This means that, unfortunately, pirtobrutinib is not currently available in the UK.

Ways to safely access Jaypirca (pirtobrutinib) before its EMA approval

Are you an MCL patient in Europe or the UK? If your doctor thinks pirtobrutinib could help you, you might not have to wait for Jaypirca's EMA approval. Instead, your doctor and you could explore Jaypirca clinical trials. Or, you can buy Jaypirca right away as an Individual Named Patient.

Access pirtobrutinib via a clinical trial

You can join a clinical trial to get Jaypirca (pirtobrutinib) or other unapproved medicines. Finding a trial recruiting participants in your country can be challenging, but not impossible. You would have to meet the trial's eligibility criteria. And, you will need your treating doctor's support.

Here are some good places to start looking for ongoing pirtobrutinib 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. An example is the NCT04662255 clinical trial with Jaypirca. It's US-based, but also open to patients in the UK, Netherlands, Germany, and multiple other European countries ⁶.

• 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: This organization supports patients in finding treatment options in clinical trials.

Buy pirtobrutinib on an Individual Named-Patient basis

In most countries, patients are legally allowed to buy and import medicines that could improve their life or address life-threatening conditions. If you want to access Jaypirca before its approval in Europe, this might be an option for you and your doctor.

The regulation making this possible is known as the Individual Named Patient Import regulation. 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 letter 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 pirtobrutinib before its 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 sourcing Jaypirca immediately.

Buy Jaypirca

Final thoughts on accessing pirtobrutinib before its EU approval

Patients with rare diseases often find the chance to try new treatments invaluable. These options can greatly improve their quality of life.

Approval processes for new medicines in Europe and elsewhere are slow. Speeding access might seem attractive. But, like any other medicine, Jaypirca also has side effects. You need to get yourself informed about them by your doctor. You'll need them on board whether you want to explore a clinical trial or buy pirtobrutinib on a Named-Patient basis.

If you and your doctor decide to try the medicine after being well-informed, there are options available. Reach out to our team for help.

References:

1. FDA grants accelerated approval to pirtobrutinib for relapsed or refractory mantle cell lymphoma. FDA, 27 January 2023.
2. Applying for EU marketing authorisation for medicinal products for human use. European Medicines Agency, Accessed 17 October 2023.
3. Jaypirca: Pending EC decision | European Medicines Agency. European Medicines Agency, 26 April 2023.
4. EFPIA Patients W.A.I.T. Indicator 2021 Survey. EFPIA, Accessed 17 October 2023.
5. Project information | Pirtobrutinib for treating relapsed or refractory mantle cell lymphoma [ID3975] | Guidance. NICE, Accessed 17 October 2023.
6. Study of BTK Inhibitor LOXO-305 Versus Approved BTK Inhibitor Drugs in Patients With Mantle Cell Lymphoma (MCL) (BRUIN-MCL-321). ClinnicalTrials.gov, Accessed 17 October 2023.