The European Medicines Agency’s Decision and Nitisinone
The AKU Society is proud to announce that the European Medicines Agency (EMA), which regulates medications across Europe, has given a positive opinion on the extension of the use of nitisinone to treat AKU. This means the EMA feels that nitisinone can be used to treat AKU and that it should be used for both AKU and the disease it was initially developed to treat, Hereditary Tyrosinemia Type 1.
The EMA came to this decision because of the positive results of the final trial in the DevelopAKUre programme, SONIA 2 (Suitability of Nitisinone In Alkaptonuria 2). SONIA 2 showed that nitisinone reduces HGA (homogentisic acid), the acid that cause the damage in AKU, by 99.7% and can stop the symptoms of AKU getting worse. It also showed that nitisinone is safe for use in AKU patients and noted that one of its side effects is tyrosine keratopathy, a build-up of the level of an amino acid called tyrosine leading to tiny crystals forming in the eyes. When caught early enough, this is fully reversible if the user stops taking the drug.
The Next Steps
Now the EMA has made this decision, nitisinone is sent to the European Commission (EC) for it to decide on whether the drug should receive marketing authorisation. This means the EC will need to say whether it wants to advise each member state of the EU to adopt the use of nitisinone in treating AKU. This decision should happen within the next few months.
What This Means For Your Country
Once the EC has agreed the marketing authorisation, each member state of the EU will then need to decide how much they will charge for nitisinone and how this will be covered by socialised health care or insurance. This process is typically undertaken by each country’s HTA (health technology assessment) agency. These are often non-governmental organisations that help the health department of each member state’s government decide on what drugs they should give to people with diseases and how much they should cost. An example of an HTA is the National Institute for Health and Care Excellence (NICE) in the UK.
This process may take some time. However, the AKU Society, along with our sister societies, will assist, where possible, for AKU patients in each country to get access to the drug. We hope this process will be as quick and painless as possible.
What This Means For The UK
The AKU Society and the NAC (National AKU Centre) are in contact with NICE as part of NICE’s horizon scanning process. NICE’s decision only commonly affects the provisioning of care in England. Each devolved nation has its own method of assessing drugs. However, a positive indication from the EMA, NICE or another English HTA will hopefully make it easier for Welsh, Scottish and Northern Irish patients to get access and begin nitisinone treatment. This decision or any future decision will not affect the operation of the NAC. Once a decision has been reached, we will communicate it to all UK patients directly.
Brexit in any form will not affect the NAC’s use of nitisinone. Due to the timing of the EMA’s decision and the January deadline for Brexit, we expect the EC’s decision to still apply in the UK as it should be announced before Christmas. We hope that all nations of the UK will be able to access nitisinone soon.
We want to thank all members of the DevelopAKUre consortium for their hard work throughout the trials. We would especially like to thank the patients who volunteered to make these trials a reality.
The AKU Society appreciates all your patience during these exciting times and will endeavour to publish blogs frequently that update everyone on the progress of nitisinone. If you do have a question, please email [email protected].