Givinostat's EMA approval: How long do you have to wait (and how not to)?

In March 2024, the FDA approved Duvyzat (givinostat) as the first nonsteroidal treatment for Duchenne Muscular Dystrophy (DMD) ¹. While this is positive news for all patients and their families, those outside the USA are facing the usual question - when will givinostat for DMD be approved in their countries, too?

For patients based in the EU and the UK, here's everything you need to know regarding givinostat's MHRA and EMA approval timelines. Including ways to access the treatment before it's available in your country.

What is givinostat used for?

Duvyzat(givinostat) is indicated for the treatment of Duchenne Muscular Dystrophy (DMD) in patients over the age of 6 ². Because of the way givinostat works, it's potentially a treatment for Becker Muscular dystrophy (BMD), as well, as indicated by the medicine's manufacturer, Italfarmaco ³. However, the medicine is not currently approved for treating BMD.

How does givinostat work?

Givinostat is an HDAC inhibitor. It blocks a type of enzymes called histone deacetylases (HDACs). HDACs play a role in activating and deactivating genes that can reduce muscle regeneration in Duchenne. By blocking HDACs, givinostat aims to stimulate muscle repair, increase muscle fiber regeneration, reduce inflammation and fibrosis ⁴.

Givinostat's EMA approval status

Duvyzat's marketing authorization application has been in review by the EMA since August 2023. Typically, a review takes up to 210 days, excluding any pauses to request additional information from the manufacturer. According to this standard timeline, a decision on givinostat's application should be available by mid-2024. However, whether that happens remains to be seen. 

When will givinostat be available in Europe?

Unfortunately, even if the givinostat gets EMA approval by mid-2024, that doesn't mean it will be available across the EU immediately. The time needed from EMA approval to market launch varies greatly across member states. For orphan medicines, such as givinostat, the average time to launch ranges from 102 days in Germany to 1,081 days in Estonia, with an EU average of 636 days ⁴. 

In other words, some countries within the EU may have access to givinostat already within 2024, but the majority of EU patients will be facing at least another year of waiting time before the medicine is available on their local markets. 

When will givinostat be available in the UK?

As of April 2024, givinostat is not yet approved or available in the UK. However, this may not mean that UK patients will have to wait for a long time.

Since early 2024, medicines that are already approved by a trusted regulator (e.g. the FDA or EMA) can benefit from accelerated review and approval by the MHRA, on the basis of the International Recognition Procedure. This doesn't automatically happen. The medicine's manufacturer, Italfarmaco, should initiate an approval process. To support in this process, the Duchenne UK organization is also launching its own campaign to push for the MHRA to expediate their recognition of the FDA's decision on givinostat.

With the above in mind, it is theoretically possible that givinostat gets MHRA approval within 2024. Whether that happens in practice remains to be seen.

Will givinostat be available on the NHS?

To be included on the NHS, givinostat will require a positive review by NICE. The review process has already been initiated, but there is no date provided for the decision publication.

Assuming both an MHRA approval and a positive NICE review, givinostat should become available on the NHS within 3 months from the NICE's decision date. 

How to safely get Duvyzat (givinostat) before its MHRA or EMA approval

Waiting for treatment can be challenging, especially without a clear timeline. The good news is, there are safe and legal ways to start your treatment with givinostat before it's available in your country. 

One of your options is to find and join a clinical trial. Another option is to buy and import givinostat for your personal use. 

Here's how each option works.

Join a givinostat clinical trial

To get quick access to Duvyzat, you can try joining an ongoing clinical trial. To do so, you must meet the eligibility criteria. You will also need your treating doctor's support. Good to keep in mind is that a trial doesn't guarantee you will be assigned to the treatment group. You may receive placebo instead. 

Here are some good places to start looking for ongoing givinostat clinical trials:

• ClinicalTrials.gov: This is a database with all clinical trials in the USA. However, some of the trials are also open to international participants. At the moment, this givionostat trial is about to start recruiting patients across Europe. It may be worth keeping an eye on.
• 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 givinostat as a Named Patient

In most countries, patients are legally allowed to buy and import medicines that could improve their lives or address life-threatening conditions. Specifically if these medicines are not approved or available locally yet.

If you want to access givinostat before it's approved in Europe and the UK (or anywhere else), this might be an option for you and your doctor.

The regulation that makes this possible is known as Named Patient Import. Under this regulation, patients can import a medication not yet approved or available in their country if:

• it is approved elsewhere;
• it has no local alternatives, and
• it is for personal use.

This process requires a prescription from the patient’s treating doctor. Other documentation requirements may apply, depending on your country.

Do you want to use the Named Patient Import regulation to get givinostat before its MHRA or EMA approval? You will first need to consult your treating doctor and get a suitable prescription.

Already have a prescription? Share it with our team at Everyone.org, so we can support you with buying givinostat immediately.

Buy Duvyzat

References:

1. FDA Approves Nonsteroidal Treatment for Duchenne Muscular Dystrophy. FDA, 21 March 2024.
2. Reference ID: 5351230. Accessdata.fda.gov, Accessed 16 April 2024.
3. Italfarmaco > Activities > Pipeline > About muscular distrophies > Givinostat. ITALFARMACO SpA, Accessed 16 April 2024.
4. EFPIA Patients W.A.I.T. Indicator 2021 Survey. EFPIA, Accessed 16 April 2024.