Seladelpar's approval around the world: How long will you have to wait?

Primary biliary cholangitis (PBC) is a rare autoimmune disease affecting 1 in 3,000 to 4,000 people, primarily women ¹.

Until 2024, only two FDA-approved treatments existed: ursodeoxycholic acid and obeticholic acid, which have limited effectiveness for about 40% of patients ². However, the FDA's approval of seladelpar in August 2024 marks a significant shift in PBC treatment options.

While this is promising for PBC patients, those outside the USA will have to wait for local approvals. If you're in Europe, the UK, or elsewhere, here’s what to expect regarding approval timelines.

What is seladelpar's indication?

Seladelpar (sold under the brand name Livdelzi) is indicated for treating adult patients with PBC. The medicine can be used in combination with ursodeoxycholic acid (UDCA) in those cases where patients have an insufficient response to UDCA alone. If the patient is unable to tolerate UDCA, seladelpar may also be used as monotherapy ³. 

What is seladelpar's success rate in clinical trials? 

 The FDA's decision to grant seldelpar an accelerated approval was based on the results of the Phase 3 RESPONSE study. The key reported results include:

• 25% of participants achieved normalization of alkaline phosphatase (ALP), a key predictor of liver health. In comparison, no patients in the placebo group achieved this result;
• 62% of participants taking Livdelzi reported a composite biochemical response 12 months after start of treatment,  compared to 20% in the placebo group;
• The most common adverse effects were headache, abdominal pain, nausea, abdominal swelling, and dizziness ⁴.

Seladelpar's approval status around the world

In August 2024, Livdelzi (seladelpar) got its first global approval by the FDA in the USA. But what approval timelines are patients elsewhere facing?

When will seladelpar get EMA approval?

Seladelpar's marketing authorization application in the EU has been in review since February 2024. Typically, the EMA approval process takes up to 210 days. Therefore, assuming the best case scenario, seladelpar's EMA approval could be a fact by the end of 2024. Only time will tell if this is indeed the case. 

When will seladelpar be available in Europe?

If we assume the best-case scenario, in which seladelpar gets its European approval before the end of 2024, that still doesn't mean that it will become available across Europe at the same time.

Each EU member state has its own local process and timeline for launching newly approved medicines. The EU average is 511 days, with Germany being the fastest (133 days) and Romania - the slowest (899 days) ⁵.

In other words, even looking at the most hopeful scenario, the earliest some countries in Europe may make seladelpar available is mid-2025. Patients in other countries may be facing over 2 years of additional waiting time.

Is MHRA approval coming for seladelpar?

As of August 2024, there is no active application for the MHRA approval of seladelpar. However, that doesn't necessarily have to spell a long waiting time for UK patients. Based on the International Reliance Procedure in place after Brexit, medicine approvals by another trusted regulatory body (e.g. FDA, EMA) can be directly adopted by the MHRA. Whether this will be the case with seladelpar, of course, is for the MHRA to decide.

When will seladelpar be available in the UK?

Let's assume the best-case scenario, in which the MHRA adopts an EMA or FDA approval of Livdelzi. That would mean that within 2024, seladelpar could theoretically be available in the UK.

However, for it to be included on the NHS, a positive evaluation from NICE is needed. As of August 2024, NICE has not yet initiated a review of the medicine, so it's difficult to predict a decision date. If and when NICE gives the green light, seladelpar should become available on the NHS within three months. 

How to get seladelpar before it's approved in your country

Are you a PBC patient? If your doctor is of the opinion that you might benefit from treatment with Livdelzi (seladelpar), 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 seladelpar via Named Patient Import.

Join a Livdelzi clinical trial

One way to access Livdelzi 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 and accept the risk that you may be assigned to the control group in the trial.

Here are some good places to start looking for ongoing seladelpar 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: This organization supports patients in finding treatment options in clinical trials.

Buy seladelpar via Named Patient Import

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

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 seladelpar before its EU approval (or its approval in the UK, China, Canada, Japan, Australia, or elsewhere)? 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 Livdelzi, wherever you are. Get in touch with us for more information.

References:

1. Primary biliary cholangitis. Wikipedia, Accessed 20 August 2024.
2. Management of Primary Biliary Cholangitis: Current Treatment and Future Perspectives. PubMed, Accessed 20 August 2024.
3. Highlights of prescribing information. Accessdata.fda.gov, Accessed 20 August 2024.
4. FDA Grants Accelerated Approval to Gilead’s Livdelzi for the Treatment of Primary Biliary Cholangitis. PharmExec.com, Accessed 20 August 2024.
5. EFPIA Patients W.A.I.T. Indicator 2021 Survey. EFPIA, Accessed 20 August 2024.