Geron's Imetelstat: When is EMA approval coming for the newest MDS treatment?

Myelodysplastic syndromes (MDS) are a rare type of blood cancer. There have been no new curative treatments for MDS approved in the last 10 years. And despite the fact that many MDS patients don't respond to first-line treatment, there have been no approved second-line therapies until recently ¹.

With Ryvelo (imetelstat)'s FDA approval in June 2024, the treatment landscape for MDS patients may be changing.

Although the news is positive, it does raise timing questions for patients outside of the US. When will Geron's imetelstat be approved in Europe, the UK, and other countries?

Here's everything you need to know.

What is Rytelo (imetelstat)?

Imetelstat is a first-in-class medicine used to treat adults with certain types of myelodysplastic syndromes (MDS). Specifically, it helps patients with low- to intermediate-1 risk MDS who suffer from anemia that requires frequent blood transfusions. Rytelo is intended for patients who need at least 4 units of red blood cells over 8 weeks and have not responded to, stopped responding to, or cannot use other treatments that stimulate red blood cell production ².

How does imetelstat work?

Imetelstat works by blocking an enzyme called telomerase, which cancer cells need to grow. It attaches to a part of the telomerase enzyme and stops it from working. This leads to shorter telomeres (protective caps on the ends of chromosomes). It also slows down the growth of cancer cells, and eventually causes them to die. This is especially important in treating myelodysplastic syndromes (MDS), where telomerase activity is higher in cancer cells ².

What is imetelstat's success rate in clinical trials?

Imetelstat's FDA approval was informed by the results of the Phase 3 IMerge trial. The trial compared the safety and efficacy of imetelstat against placebo in patients with ESA-relapsed, ESA-refractory, or ESA-ineligible low- or intermediate-1 risk MDS ³.

Some of the key reported results were:

• 40% of imetelstat patients had a transfusion independence of at least 8 weeks, compared to 15% of the placebo group;
• 91% of imetelstat patients (compared to 47%) of placebo patients experienced adverse effects of grade 3 or 4. The most common adverse effects of imetelstat were neutropenia and thrombocytopenia ³;
• The treatment with Imetelstat resulted in a notable decrease in variant allele frequency (VAF) in specific genes often mutated in MDS. This outcome correlated with a prolonged period of transfusion independence and elevated levels of hemoglobin ⁴.

Will imetelstat be approved in Europe?

After imetelstat gained FDA approval in June 2024, the logical question for patients elsewhere is when will it be approved in their countries, too.

When it comes to Europe, the process of gaining EMA approval for imetelstat is already underway. Geron's marketing authorization application for the medicine was validated in October 2023 ⁵.

Typically, the EMA approval process takes up to 210 days (or less for orphan medicines such as imetelstat). With this timeline in mind, Rytelo should have been EMA-approved by now. However, as of July 2024, that is not the case. Hopefully, the approval in Europe is just a matter of a bit more time and we will have some positive news soon.

When will imetelstat be available across the EU?

Approval and availability are unfortunately not the same thing. While imetelstat's EMA approval seems just around the corner, it will still take some time until the treatment is available across Europe.

Each EU member state launches EMA-approved medicines at their own pace. Some, like Germany, are relatively quick, with an average launch time of 100 days for oncological treatments. At the other end of the spectrum is Romania, where it takes an average of 964 days for a new cancer treatment to become available ⁶.

In other words, imetelstat could theoretically become available in some EU countries towards the end of 2024. That's assuming that the medicine will get EMA approval by September 2024. Other EU countries may have to wait for another couple of years.

When will imetelstat be approved in the UK?

In the UK, imetelstat holds the Innovation Passport Designation. This designation offers an expedited review pathway ⁷.

While this is positive news, there is no specific timeline for imetelstat's MHRA approval. Theoretically, a UK approval could be a fact within 2024, if the MHRA follows the International Reliance Procedure ⁸. However, only time will tell if that's the direction they go.

When will imetelstat be available to UK patients?

If the MHRA approves Rytelo soon, the medicine will then require a NICE assessment before becoming available on the NHS. If approved, the medicine should be available to UK patients within three months of the NICE's decision date.

NICE has already initiated an assessment of imetelstat. However, it is currently suspended, meaning that there is no decision-making timeline available at the moment.

Assuming a best-case scenario, where the MHRA approves imetelstat by the end of 2024, and NICE makes a positive decision soon after, the treatment could be available in the UK by mid-2025. However, note that this is only a theoretical timeline and the MHRA's actions in the next months will determine whether it's realistic.

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

If you're based outside the USA, having to wait for Rytelo's local approval may be discouraging. Fortunately, waiting is not the only thing you can do. With your treating doctor's support, you could try and access imetelstat via a Compassionate use program. Or, you can buy imetelstat immediately via Named Patient Import.

Imetelstat compassionate use

Geron started an early access program (EAP) for imetelstat in June 2023. The program offers free access to the medicine before it's locally approved. Note that this program and its inclusion criteria may change now that imetelstat has FDA approval.

For more information on the program, your treating doctor will have to submit a request to Geron directly via [email protected]. Note that submitting a request doesn't guarantee access to the program.

Buy imetelstat 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. The regulation making this possible is known as 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 Named Patient Import regulation to get imetelstat before its EMA approval (or its approval in the UK 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 Rytelo immediately. Get in touch with us for more information.

 References:

1. Emerging treatments for myelodysplastic syndromes: Biological rationales and clinical translation. NCBI, 13 February 2023.
2. HIGHLIGHTS OF PRESCRIBING INFORMATION. Accessdata.fda.gov, Accessed 16 July 2024.
3. Imetelstat in patients with lower-risk myelodysplastic syndromes who have relapsed or are refractory to erythropoiesis-stimulating agents (IMerge): a multinational, randomised, double-blind, placebo-controlled, phase 3 trial. The Lancet, Accessed 16 July 2024.
4. Eckford, Catherine. Phase III data for novel telomerase inhibitor released. European Pharmaceutical Review, 5 December 2023.
5. EMA Validates MAA for Imetelstat for Transfusion-Dependent Anemia in Lower-Risk MDS. OncLive, 3 October 2023.
6. EFPIA Patients W.A.I.T. Indicator 2021 Survey. Efpia, Accessed 16 July 2024.
7. Geron Enters New Innovative Licensing and Access Pathway in the United Kingdom for Imetelstat. Geron, 25 October 2021.