Research Update: Efmody®. Cortisol replacement, is there a better way? For nearly 10 years, pharmaceutical company Diurnal Ltd has been developing Efmody®, a hydrocortisone modified-release hard capsule. Many of you will also be familiar with Efmody® by its development name, Chronocort®, from previous ADSHG updates over the years. Excitingly, this medication has now been approved for those living with congenital adrenal hyperplasia (CAH), a condition where people also have adrenal insufficiency. A clinical trial has taken place in people with Addison’s disease. This was also made possible thanks to the ADSHG members who answered our request for volunteers for the clinical trial in October 2022. This important research is led by Dr Alessandro Prete, Associate Professor of Endocrinology at the University of Birmingham and ADSHG Medical Trustee. Following Dr Prete’s presentation on Efmody® at our 2024 London Addison’s Roadshow, we wanted to answer your questions on this promising new treatment. What is Efmody®? Efmody® is hydrocortisone (the same medication that many people with Addison’s and adrenal insufficiency already take) but designed in a very clever way! The tablet is a modified-release hard capsule. When the patient takes the capsule, the capsule resists the acid of the stomach, so it is not broken down and instead starts to be released when it goes into the small intestine. This differs from hydrocortisone, where cortisol levels start to go up immediately - with Efmody®, they start to go up very gradually. Efmody® is taken twice a day, while the standard hydrocortisone replacement is taken twice or thrice a day. Efmody® is taken following a “toothbrush” regimen, so the patient takes a tablet at bedtime and on waking. Approximately two-thirds of the daily dose is taken at bedtime, and one-third in the morning. This benefits the patient as the cortisol levels rise gradually through the night to replicate the natural pre-dawn rise, which is otherwise not possible with immediate-release hydrocortisone. The graph below shows the normal cortisol production in red. The green line shows cortisol levels during twice-daily Efmody® treatment: this closely replicates the normal cortisol production. Efmody® is available in doses of 5mg and 10mg. Current treatment – the problem to be solved Current generic hydrocortisone replacement takes effect in the hour after waking, once we are awake and have taken our morning medication. This means that we wake up with almost no cortisol in the body at a time when it’s designed to be at its peak. We think this is the reason why many people struggle with quality-of-life issues in the early morning, such as fatigue, and some people find they cannot get out of bed until that morning dose has kicked in. Again, in this graph, the red line shows the normal cortisol production. Here, the green line shows the levels of cortisol in the blood of someone who takes standard hydrocortisone three times a day. The main problem Efmody® addresses is the pre-dawn rise of cortisol (shaded blue in the graph), which we, as people with Addison’s and adrenal insufficiency, miss out on as we’re still sleeping! Research results Dr Prete and his team have completed their clinical trial sponsored by Diurnal Ltd, known as CHAMPAIN, which compared the efficacy, safety, and tolerability of Efmody® with Plenadren®, another modified-release hydrocortisone formulation. This trial involved over 40 patients with Addison’s disease, many from the ADSHG. Those of you who attended our 2024 London Addison’s Roadshow will have had a sneak peek at the initial results of the trial from Dr Prete himself, which are extremely promising. In CAH patients, for whom Efmody® is now approved, not only has it shown better control of the condition - it has also enabled patients to reduce their dose (mitigating the long-term negative impact of steroid use), as well as reducing stress dosing and adrenal crises so improved overall quality of life. It’s possible that with this overnight, slow-release formula, we could avoid not only those morning symptoms but also experience a safer, improved quality of life. All with the additional benefit of only having to take two tablets a day – waking and bedtime. The official results of the CHAMPAIN study should be released in 2025: watch this space for more updates! Thank you to Dr Alessandro Prete and his team for their work on this research to improve the future for those with Addison’s and adrenal insufficiency. You can hear more from Dr Prete in his presentation “Adrenal Insufficiency: Looking to the Future” by watching the recorded videos available on the forum. Watch online Author: Dr Alessandro Prete Consultant Endocrinologist, University Hospitals Birmingham NHS Foundation Trust. Whether you're newly diagnosed or have lived with the condition for years - please join our community and support our cause! You'll receive the latest expert advice, guidance and ADSHG news, whilst being part of our inspiring and supportive community. Become a member today! Join the ADSHG Connect on social media! Follow us on X/Twitter, Bluesky, Facebook, Instagram, LinkedIn, Threads, TikTok and YouTube. Manage Cookie Preferences