Efmody (hydrocortisone modified release) / Abiogen, Neurocrine Biosciences - LARVOL DELTA

Once-daily, late-night, modified-release hydrocortisone rapidly normalizes menstrual cyclicity in women with non-classic congenital adrenal hyperplasia. (PubMed, Hum Reprod Open) - "A single nightly dose of MR-HC appears sufficient to achieve hormonal control and subsequent menstrual-cycle restoration in most women with NCCAH and may improve time to conception. These findings support MR-HC as a promising therapeutic option in the management of NCCAH."

Journal • Congenital Adrenal Hyperplasia • Endocrine Disorders • Women's Health

Switching from hydrocortisone to Modified-Release Hydrocortisone (Efmody) in patients with congenital adrenal hyperplasia: a paired analysis of dose and hormonal response at 6 months (ESPE 2026) - No abstract available

Clinical • Late-breaking abstract • Congenital Adrenal Hyperplasia • Endocrine Disorders

Modified-release hydrocortisone (Efmody®) in children with congenital adrenal hyperplasia: a retrospective registry study. (PubMed, Front Endocrinol (Lausanne)) - "Replacement therapy for congenital adrenal hyperplasia (CAH) in childhood includes hydrocortisone and, in salt-wasting forms, addition of fludrocortisone. No adrenal crisis was observed. Twice-daily HMRC therapy can also improve CAH therapy in children and adolescents in terms of hormonal control and growth."

Journal • Retrospective data • Congenital Adrenal Hyperplasia • Endocrine Disorders

Replacing Early Morning Cortisol Reduces Fatigue in Patients with Adrenal Insufficiency: Post-Hoc Analysis of CHAMPAIN, A Double-Blind Study Comparing Chronocort and Plenadren (ENDO 2026) - P2 | "Replacing the early morning cortisol improved health related QoL in AI based on the disease specific questionnaire AddiQoL. This was primarily due to reduced fatigue which was also seen in the SF-36 physical component score and fatigue specific questionnaire PROMIS 7b and there were fewer reports of fatigue on Chronocort compared to Plenadren."

Clinical • Retrospective data • Endocrine Disorders • Fatigue • Nephrology • Rare Diseases • Renal Disease

Real-World Outcomes with Modified-Release Hydrocortisone in Classic Congenital Adrenal Hyperplasia. (PubMed, Eur J Endocrinol) - "MR-HC enabled stable biochemical control with reduced glucocorticoid exposure and was associated with improvements in insulin sensitivity, blood pressure, and menstrual regularity, supporting its role as a safe and physiological treatment option in adults with CAH."

Journal • Real-world evidence • Congenital Adrenal Hyperplasia • Endocrine Disorders • Women's Health

Glucocorticoid Prescribing Trends in Congenital Adrenal Hyperplasia, 2017 to 2023. (PubMed, Endocr Connect) - "We found that despite increasing availability of new medications during the study time period, only a small number of patients (5%) switched to these newer treatments. This limited use is mainly due to high costs, problems with getting legal approval, and supply issues, highlighting unequal access to care worldwide."

Journal • Congenital Adrenal Hyperplasia • Endocrine Disorders • Pediatrics

Efmody - opinion on variation to marketing authorisation (European Medicines Agency) - "On 29 January 2026, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending a change to the terms of the marketing authorisation for the medicinal product Efmody....Efmody is indicated in adolescents aged 12 years and over and adults for the: Treatment of adrenal insufficiency (AI); Treatment of congenital adrenal hyperplasia (CAH)."

CHMP • Congenital Adrenal Hyperplasia

Long-term Safety Study of Chronocort in the Treatment of Participants With Congenital Adrenal Hyperplasia (clinicaltrials.gov) - P3 | N=76 | Completed | Sponsor: Neurocrine UK Limited | Enrolling by invitation ➔ Completed

Trial completion • Congenital Adrenal Hyperplasia • Endocrine Disorders

Effects of modified release hydrocortisone on restoration of early morning cortisol, quality of life, and fatigue in adrenal insufficiency (The CHAMPAIN study): a randomised, double-blind, double-dummy, cross-over study comparing Chronocort and Plenadren. (PubMed, EClinicalMedicine) - P2 | "Restoring the early morning cortisol levels with twice daily Chronocort 15 mg at night 10 mg in the morning improved health-related quality of life, fatigue and the immune profile compared with 25 mg daily Plenadren. Neurocrine UK Ltd."

HEOR • Journal • Endocrine Disorders • Fatigue • Nephrology • Renal Disease

The Current Treatment Landscape for Congenital Adrenal Hyperplasia. (PubMed, Drugs) - "Modified-release hydrocortisone formulations, such as Efmody® and Plenadren®, as well as continuous subcutaneous infusion therapies, have been developed to help better mimic physiological cortisol rhythms...This review provides an overview of current standard-of-care treatments for CAH and highlights recent advancements in therapeutic strategies. By addressing the limitations of traditional glucocorticoid replacement, these innovations have the potential to improve long-term outcomes and quality of life for patients with CAH."

Journal • Review • Congenital Adrenal Hyperplasia • Endocrine Disorders • CYP1A2 • CYP21A2

A Double-blind Study of Modified-release Hydrocortisones, Chronocort versus Plenadren, in Adrenal Insufficiency (CHAMPAIN) (ENDO 2025) - "In adrenal insufficiency Chronocort provides physiological waking cortisol levels and is associated with reduced fatigue and improved QoL as compared to Plenadren."

Clinical • Endocrine Disorders • Fatigue • Nephrology • Renal Disease

Long-term outcomes in patients with congenital adrenal hyperplasia treated with hydrocortisone modified-release hard capsules. (PubMed, Eur J Endocrinol) - "MRHC treatment resulted in hydrocortisone dose reduction followed by a stable dose with improved biochemical control associated with fertility. Biochemical control could be reliably monitored by a single blood sample taken between 09:00h and 13:00h. The incidence of adrenal crises was below that reported previously in patients with CAH."

Journal • Congenital Adrenal Hyperplasia • Endocrine Disorders • Nephrology • Renal Disease

Switching Patients With Congenital Adrenal Hyperplasia to Modified-Release Hydrocortisone Capsules: Relative Bioavailability and Disease Control. (PubMed, Clin Endocrinol (Oxf)) - "MRHC showed comparable bioavailability to IRHC based on cortisol AUC after 20 mg administration. Switching patients treated with IRHC to a twice daily MRHC regimen on the same daily dose (giving approximately two thirds of the dose at night) is an effective protocol for starting MRHC treatment."

Journal • Congenital Adrenal Hyperplasia • Endocrine Disorders • Nephrology • Renal Disease

Achieving Spontaneous Pregnancy in a Patient with Classic Congenital Adrenal Hyperplasia through Modified-Release Hydrocortisone (ESPE-ESE 2025) - "To optimise disease control her treatment was adjusted multiple times, including the use of prednisolone and dexamethasone, dose escalations and additional fludrocortisone. The presented case of a female patient with classic CAH and infertility for over three years highlights the challenges of fertility treatment in CAH. The normalisation of morning follicular progesterone concentrations and the successful spontaneous pregnancy under MR-HC treatment, indicate that due to the circadian release MR-HC normalises adrenal steroid precursor synthesis in a more physiologic manner. Therefore, it could be a promising therapeutic option for fertility treatment in women with classic CAH."

Clinical • Congenital Adrenal Hyperplasia • Endocrine Disorders • Gynecology • Infertility • Sexual Disorders

A Double-blind Study of Modified-release Hydrocortisones, Chronocort versus Plenadren, in Adrenal Insufficiency (CHAMPAIN) (ESPE-ESE 2025) - "In adrenal insufficiency** Chronocort provides physiological waking cortisol levels and is associated with reduced fatigue and improved QoL as compared to Plenadren."

Clinical • Endocrine Disorders • Fatigue • Nephrology • Renal Disease

Treatment of children and adolescents with congenital adrenal hyperplasia with hydrocortisone modified-release hard capsules (ESPE-ESE 2025) - "Context: The standard treatment for congenital adrenal hyperplasia (CAH) in childhood includes hydrocortisone and, for salt-wasting CAH, fludrocortisone supplementation...Conventional fast- acting hydrocortisone often fails to adequately suppress the early morning surge of 17-OH progesterone (17OHP), and the requirement for three daily doses poses adherence challenges... HMRC treatment in children with CAH improves morning pre-dose 17OHP levels, while growth rates align more closely with the parental target height. No significant adverse effects or severe complications were observed. Further research is needed to assess long-term outcomes and quality of life."

Clinical • Congenital Adrenal Hyperplasia • Endocrine Disorders

17OHP and cortisol day curve profiles for children using hard-capsule modified release hydrocortisone as compared with immediate release hydrocortisone (ESPE 2024) - "Patient characteristics are shown in Table 1. Subject 8 (with SAI) stopped MRH due to a lack of symptomatic improvement after 1 month so had no cortisol profiles. Subject 9 (with SAI) had no cortisol day curves obtained before transitioning to adult services."

Clinical • Congenital Adrenal Hyperplasia • Endocrine Disorders • Nephrology • Pediatrics • Rare Diseases • Renal Disease

Effects of the modified release hydrocortisone preparation Efmody® on hormones, spermatogenesis and body weight in males with congenital adrenal hyperplasia (ESPE 2024) - "The median Efmody® dose required for optimal cortisol replacement was 16 mg/m2 (10-22.7), corresponding to 30 mg/day (25-50), with one third of the daily dose taken at 7:00 a.m. and two thirds taken at 11.00 p.m. The dose of fludrocortisone was not changed. The modified release hydrocortisone preparation Efmody® seems to be a valid alternative to conventional glucocorticoid replacement in men with CAH, with potential beneficial effects on hypothalamic-pituitary-adrenal and -gonadal axis hormone concentrations, semen quality and body weight."

Congenital Adrenal Hyperplasia • Endocrine Disorders • Musculoskeletal Diseases • Musculoskeletal Pain • Oncology • Orthopedics • Pain • CYP1A2

3D printed, personalized hydrocortisone for hypocortisolemia. (ESPE 2024) - "3D printed SR and DR hydrocortisone tablets were developed for patients with AI. The quality of 3D tablets is higher than pharmacy compounded capsules, while manufacturing costs are feasible. The 3D therapy is once-daily, compared to three times a day of the SOC, reducing pill-frequency."

Endocrine Disorders • Nephrology • Pediatrics • Renal Disease

Long-term Safety Study of Chronocort in the Treatment of Participants with Congenital Adrenal Hyperplasia (clinicaltrials.gov) - P3 | N=76 | Enrolling by invitation | Sponsor: Diurnal Limited | Trial completion date: Dec 2024 ➔ Dec 2025 | Trial primary completion date: Dec 2024 ➔ Dec 2025

Trial completion date • Trial primary completion date • Congenital Adrenal Hyperplasia • Endocrine Disorders

Comparison of modified-release hydrocortisone capsules versus prednisolone in the treatment of congenital adrenal hyperplasia. (PubMed, Endocr Connect) - "MRHC reduces 17OHP at 09:00h compared to prednis(ol)one and the dose of MRHC can be down-titrated over time in the majority of patients."

Journal • Congenital Adrenal Hyperplasia • Endocrine Disorders

CHAMPAIN Study: Initial Results From A Phase II Study Of Efficacy, Safety And Tolerability Of Modified-release Hydrocortisones: Chronocort® (Efmody®) Versus Plenadren®, In Primary Adrenal Insufficiency (ENDO 2024) - "Abstract is embargoed at this time."

Clinical • Late-breaking abstract • P2 data • Endocrine Disorders • Nephrology • Renal Disease

CHAMPAIN Study: Initial Results From A Phase II Study Of Efficacy, Safety And Tolerability Of Modified-release Hydrocortisones: Chronocort® (Efmody®) Versus Plenadren®, In Primary Adrenal Insufficiency (ENDO 2024) - "Chronocort® provides more physiological waking cortisol levels than Plenadren®. Further analysis will test the hypothesis that waking with physiological cortisol levels improves fatigue and QoL in patients with PAI. 1.Mah PM, et al."

Clinical • Late-breaking abstract • P2 data • Congenital Adrenal Hyperplasia • Endocrine Disorders • Fatigue • Nephrology • Rare Diseases • Renal Disease

Incidence of Adrenal Crisis in Congenital Adrenal Hyperplasia (CAH) Patients During a Prospective Monitored Long-Term Study of Modified-Release Hydrocortisone (MRHC) Capsules, (Efmody) (ENDO 2024) - "Data from this longest prospectively monitored study in CAH suggest that the incidence of adrenal crisis on MRHC is at the low end of that reported in retrospective studies and the safety profile of MRHC is otherwise similar to immediate release hydrocortisone. Unless otherwise noted, all abstracts presented at ENDO must not be released to the press or the public until the date and time of presentation. For oral presentations, the abstracts are embargoed until the session begins."

Clinical • Cardiovascular • Congenital Adrenal Hyperplasia • Endocrine Disorders • Fatigue • Musculoskeletal Pain • Myocardial Infarction • Nephrology • Renal Disease

Morning Cortisol Levels in Patients With Established Primary Adrenal Insufficiency (ENDO 2024) - "We have developed a modified-release formulation of hydrocortisone to replace the physiological cortisol circadian rhythm and are undertaking a Double-Blind, Double-Dummy, Two-Way Cross-Over, Randomised, Phase II Study of Modified-Release Hydrocortisones: Chronocort® Versus Plenadren® in PAI... In patients with an established diagnosis of PAI, the majority had undetectable morning cortisol, but cortisol was detectable in 41% of patients and above 140 nmol/L in 13% confirming previous publications 2. Retesting patients with a cortisol >50nmol/L showed very similar results suggesting that the detectable cortisol was not an artefact and likely due to background cortisol secretion. 1."

Clinical • Endocrine Disorders • Immunology • Nephrology • Renal Disease