When will nirogacestat get its EMA approval (and how to access it earlier)

Up until November 2023, the only treatment options for patients with desmoid tumors were surgery and radiation. With the FDA's approval of Ogsiveo (nirogacestat), this is no longer the case. The new medicine shows promise in alleviating pain symptoms and slowing down the progression of this rare disease¹. 

Currently, nirogacestat is only approved in the USA. What does this mean for non-US patients affected by desmoid tumors, and when will nirogacestat be available in Europe and beyond? Here's everything you need to know about nirogacestat's EMA approval. Including ways to access the medicine before it's available in your country. 

What is nirogacestat used for?

Ogsiveo (nirogacestat) is indicated for adults with progressing desmoid tumors, who require systemic treatment ². The medicine is available as tablets, taken orally twice a day. 

Nirogacestat inhibits a specific enzyme called gamma secretase. This enzyme is involved in activating a protein called Notch, which is believed to contribute to the growth of desmoid tumors. By blocking the enzyme, Ogsiveo (nirogacestat) intends to slow down tumor growth².

According to Ogsiveo's reported clinical trial results, the medicine may also alleviate pain symptoms, which are often associated with desmoid tumors and can greatly diminish patients' quality of life³.

nirogacestat EMA approval: What's the status?

Ogsiveo's manufacturer, SpringWorks Therapeutics, has shared its intention to apply for Marketing Authorization in the EU in the first half of 2024 ¹. 

According to the EMA's guidelines, assessing an application can take up to 210 days. At that point, the CHMP issues its recommendation. If positive, the new medicine should be granted marketing authorization within 67 days ⁴.

So, when will nirogacestat be available in Europe? It seems that the earliest timeline is somewhere in 2025. 

When will nirogacestat be available in the UK?

nirogacestat's approval in the UK is not bound anymore by the timelines of the EMA. While this could mean a shorter approval timeline, it doesn't have to.

At the moment, there is no active nirogacestat evaluation by the MHRA or NICE. If nirogacestat's manufacturer submits an authorization application to the MHRA, it typically takes 90 days to process. Once MHRA-approved, the NICE needs to evaluate the medicine as well for NHS coverage. In case of a positive recommendation by NICE, nirogacestat can be made available on the NHS within 3 months ⁵. 

With these timelines in mind, it seems that desmoid tumor patients in the UK are also unlikely to have access to nirogacestat before 2025. 

How to access nirogacestat before its EMA approval

Do you have to wait until nirogacestat is available in Europe or the UK to start your treatment? Fortunately, no. 

There are two main options to access nirogacestat immediately outside the USA. One is to join a clinical trial involving nirogacestat. The other is to buy nirogacestat as an Inidividual Named Patient. 

Join a nirogacestat clinical trial

You can try joining an ongoing clinical trial to get quick access to Ogsiveo (nirogacestat). Finding trials isn't easy, but it's possible. To participate, you must meet the eligibility criteria and have your doctor's support. Be aware that you might receive a placebo instead of the active ingredient.

Here are some good places to start looking for ongoing nirogacestat 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, there are no listed clinical trials involving Ryzneuta, but this can change at any point, so it's 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 and FindMeCure: This organization supports patients in finding treatment options in clinical trials.

Buy nirogacestat as an Individual Named Patient

Most countries in the world allow for individual patients to import medicines that can address serious conditions before they are available locally. The regulations guiding this process can vary in name, but they are typically referred to as Named Patient Import regulations. 

If you want to buy nirogacestat before its EMA approval or UK approval, this could be a good option for you and your doctor. 

To make use of the Named Patient Import regulation, you will need a prescription from your doctor. Based on your country, additional documentation may be required. However, you don't have to stress about it.

Buy nirogacestat

If you have a prescription for Ogsiveo (nirogacestat) and would like to buy the medicine right now, get in touch with us. We're qualified and certified to support you with importing medicines before they're locally available. 

References:

1. Eckford, Catherine. First pill for desmoid tumours approved,  European Pharmaceutical Review, 28 November 2023.
2. Reference ID: 5283221, Accessdata.fda.gov, Accessed 15 January 2024.
3. Dr Kasper on the Evaluation of Nirogacestat in Desmoid Tumors in the DeFi Trial, OncLive, 9 June 2023.
4. Presentation - Centralised procedure at the European Medicines Agency, European Medicines Agency, Accessed 15 January 2024.
5. Ewbank, Leo. Access to new medicines in the English NHS. The King's Fund, 28 October 2020.