How to Navigate the EMA's Adakveo Revocation and Its Impact on You

When it was approved by the FDA in 2019, Adakveo (crizanlizumab) was the first new sickle cell disease treatment to be introduced in decades ¹. With that came high expectations, both from the medical community and from patients ².

Fast forward to the present day and the EMA has officially revoked its conditional marketing authorization for Adakveo ³. If you're a patient who has been using the treatment, you probably have a lot of questions. 

Does Adakveo's revocation mean it's unsafe? Will you still be able to access the medicine? What are potential Adakveo alternatives to discuss with your doctor?

In this article, we'll tell you everything you need to know about Adakveo's revocation by the EMA. 

Why was Adakveo revoked in the EU?

The EMA provided a number of reasons for its decision to revoke crizanlizumab's conditional marketing authorization. The main ones are:

• The Committee for Medicinal Products for Human Use (CHMP) found that Adakveo's clinical benefits did not outweigh its risks.
• The results from the STAND clinical trial did not demonstrate Adakveo's efficacy. More specifically, its ability to decrease the number of painful crises.
• The STAND trial showed a higher frequency of serious side effects for Adakveo compared to placebo.
• Additional clinical studies, a managed access program, and real-world data failed to counterbalance the negative results of the STAND study.

Since the STAND trial results did not confirm the medicine's benefits, the CHMP recommended revocation ³. 

Is Adakveo still available in the UK?

After its revocation in the European Union (EU), you may be wondering if Adakveo is still available to sickle cell patients in the UK.

Currently, UK patients' access to Adakveo does not seem to be affected. Adakveo is available as part of a Managed Access agreement between Novartis and NICE. This program is currently set to run until January, 2025. At that point, the gathered data will be re-evaluated by NICE. Based on this evaluation, a final recommendation will be made for routine NHS funding ⁵.

Is Adakveo unsafe?

In the current circumstances, you may be wondering if Adakveo is hazardous. 

There are always risks involved in treatment with newly approved medicines. That's why it's always important to discuss the full prescribing information with your doctor. 

It's good to know that the STAND clinical trial for Adakveo did not show any new safety concerns. It did reveal that known serious side effects may be more common in Adakveo patients compared to those taking a placebo ⁴.

Since the trial did not prove the medicine's effectiveness in reducing painful crises, the increased risk of side effects was considered unjustified. The EMA's decision may have been different if Adakveo's efficacy results had been more positive in the STAND trial.

In other words, the more important question is not whether Adakveo is unsafe, but whether it's efficient enough to justify its adverse effects. Based on the current data, this doesn't seem to be the case.

Adakveo alternatives to discuss with your doctor

Adakveo's revocation in the EU has left patients like you in need of alternative treatment options. 

Only your doctor is competent to choose the right Adakveo alternative in your case. Here, we're simply offering a shortlist of medicines approved for sickle cell disease, which you may want to discuss with your doctor:

• Xyndari (L-glutamine):  Like Adakveo, Xyndari also aims to reduce the frequency of painful crises. Xyndari is approved in the USA (under the commercial name Endari). Its marketing authorization application to the EMA was withdrawn in 2019 ⁷. However, if your doctor considers Xyndari a suitable Adakveo alternative for you, you can access the medicine in Europe within the Named Patient Import regulation. Get in touch with our team for more information. 
• Oxbryta (voxelotor): This medicine aims to reduce low red blood cell count (anemia). Oxbryta is approved in the USA and Europe ⁶. Here's some more information on how Oxbryta compares to Adakveo.
• Hydroxyurea: A well-established treatment option that can help to reduce the frequency of sickle cell pain crises. In the EU, there are two approved medicines with this active ingredient - Siklos and Xromi. 

Can you still access Adakveo in Europe after its revocation?

If you've been treated with Adakveo and had beneficial results, you may not be interested in Adakveo alternatives. Instead, you’re probably wondering if you can still buy crizanlizumab after its revocation in the EU. 

The good news is, you can, as long as your doctor maintains the opinion that crizanlizumab is the right course of treatment for you. 

When a medicine is unapproved in a patient's country, or it's approved but not available (as is the case with Adakveo in Europe), you can access it via the Named Patient Import regulation.

Everyone.org specializes in helping people access the latest medicines via this regulation. If you have a prescription from your treating doctor for Adakveo (crizanlizumab) and would like us to help you access the medicine immediately, contact us.

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References:

1. FDA approves crizanlizumab-tmca for sickle cell disease. FDA, 15 November 2019.
2. Carvalho, Joana. Adakveo 'Important' and Safe New Treatment for VOC Crises in Sickle Cell. Sickle Cell Disease News, 22 November 2019.
3. Adakveo | European Medicines Agency. European Medicines Agency, Accessed 3 October 2023.
4. Adakveo - EMEA/H/A-20/1525/C/4874/0013. European Medicines Agency, 3 August 2023.
5. Crizanlizumab - A Simple Guide. Sickle Cell Society, Accessed 3 October 2023.
6. Oxbryta | European Medicines Agency. European Medicines Agency, Accessed 3 October 2023.
7. Xyndari: Withdrawn application. European Medicines Agency, Accessed 3 October 2023.