Lurbinectedin's EMA approval: Is it coming (and when)?

Lurbinectedin was designated an orphan drug by the FDA and EMA. It was also included in the FDA's Project Orbis for accelerated concurrent oncological medicine approval ⁶. These were all signs of hope for small cell lung cancer patients everywhere, suggesting the possibility of a faster approval process for the medicine.

However, over 5 years after its orphan drug designation and 3 years after its accelerated FDA approval, Zepzelca (lurbinectedin) is still waiting for its EMA approval.

How long do lung cancer patients in Europe and the UK still have to wait to access this treatment? Find out the latest news, including your options for getting lurbinectedin before it's widely available.

What is lurbinectedin used for?

Zepzelca (lurbinectedin) is a prescription medicine for the treatment of metastatic small cell lung cancer (SCLC) in adults. It's specifically intended for patients whose disease has progressed after chemotherapy ¹.

What is lurbinectedin's success rate?

The FDA granted Zepzelca an accelerated approval on the basis of the PM1183-B-005-14 trial. This is what the reported results tell us about lurbinectedin's success rate in SCLC:

• 35% of patients had their tumors shrink or disappear;

• The median response duration was 5.3 months;

• Some of the most common adverse reactions included leukopenia, lymphopenia, fatigue, anemia, neutropenia, increased creatinine, alanine aminotransferase or glucose ¹.

Lurbinectedin confirmatory trials

In order to maintain its FDA approval, lurbinectedin's manufacturer was required to conduct a confirmatory study. This Phase 3 study, called ATLANTIS, failed to demonstrate a statistically significant improvement in overall survival compared to chemotherapy alone.

However, this outcome may have been due to the study design. For example, it tested a lower dosage of lurbinectedin than the one approved, and combined it with a chemotherapy agent. With this in mind, the FDA has decided to maintain Zepzelca's approval. In order to confirm its efficacy, the manufacturer must provide data from a new confirmatory trial with an appropriate design.

At the moment, two confirmatory trials for lurbinectedin are ongoing. The LAGOON trial will finalize in June 2025, and the IMForte trial will complete in March 2026. Hopefully, the data from these trials will support the medicine's prolonged approval in the USA, and help speed up its approval in other regions, such as Europe.

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Lurbinectedin's EMA approval: What's the status?

In 2019, the EMA granted lurbinectedin an orphan drug designation for treating SCLC ². However, no formal marketing authorization application has been submitted to the EMA since then.

Typically, from the moment a marketing authorization application is submitted, it takes up to 210 days for an approval decision to be made. This process may be a bit quicker for orphan medicines. In any case, even if PharmaMar submits its EMA approval application today, it would still be looking at a potential approval no earlier than end of 2024-beginning of 2025.

An exception to this timeline is Switzerland, where the medicine was granted temporary approval in March 2023 ⁴.

When will lurbinectedin be available in Europe?

It's difficult to say. If an EMA approval application for lurbinectedin is submitted before the end of the year, by mid-2025 there could theoretically be a decision on it. If positive, it would take another 67 days for Zepzelca's EMA approval to become official.

From that point on, each EU member state can initiate their own procedures for local price negotiation, reimbursement, and local market launch. How long these procedures take depends on the country.

Germany tends to be the fastest to launch newly approved orphan medicines, within an average of 102 days. At the other end of the spectrum is Estonia, where it takes an average of 1,081 days for a new orphan medicine to become available ³.

In other words, it seems like lurbinectedin will be available in the EU no earlier than late 2025 or early 2026.

When will Zepzelca be available in the UK?

The MHRA granted Zepzelca the Innovative Medicine designation in 2022. In the same year, PharmaMar submitted a formal application for conditional marketing authorization in the UK ⁵.

However, since then, no decisions have been made. As of April 2024, the MHRA has not yet approved lurbinectedin. The NICE has also suspended its assessment of the medicine for potential inclusion on the NHS. There is no timeline provided by either agency regarding decisions.

How to safely get lurbinectedin before its MHRA and EMA approval

Are you a patient with SCLC? If your doctor is of the opinion that you might benefit from treatment with Zepzelca, you may not need to wait for its approval in Europe or the UK. Instead, your doctor and you could consider joining a lurbinectedin clinical trial. Or, buying the medicine on an Individual Named-Patient basis.

Join a lurbinectedin clinical trial

One way to access Zepzelca (lurbinectedin) is to enroll in a clinical trial. This can often be easier said than done, as you and your doctor would need to find a trial that is currently recruiting participants in your country. You would also have to meet their eligibility criteria. For all of this, you will need your treating doctor's support.

Here are some good places to start looking for ongoing lurbinectedin 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 LAGOON clinical trial. It's US-based, but also open to patients across Europe and Asia.

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

Buy lurbinectedin as a Named Patient

In most countries, patients are allowed to buy and import medicines that could enhance their quality of life or address life-threatening conditions. If you want to access Zepzelca before it's available in your country, this might be the quickest 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 lurbinectedin 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 accessing Zepzelca. We specialize in sourcing and delivering medicines not yet available in your country. Get in touch with us for more information.

References:

1. Reference ID: 4624945. Accessdata.fda.gov, Accessed 23 April 2024.
2. EU/3/19/2143 - orphan designation for treatment of small cell lung cancer. European Medicines Agency, 9 April 2019.
3. EFPIA Patients W.A.I.T. Indicator 2021 Survey. Efpia, Accessed 23 April 2024.
4. PharmaMar announces the approval of Zepzelca® (lurbinectedin) for the treatment of metastatic Small Cell Lung Cancer in Switzerland. Pharmamar, Accessed 23 April 2024.
5. PharmaMar has filed for approval of lurbinectedin for the treatment of metastatic Small Cell Lung Cancer in the UK. Pharmamar, Accessed 23 April 2024.
6. Project Orbis. FDA, Accessed 23 April 2024.