- Levoketoconazole (Recorlev) was associated with improvements in mean 24-hour urinary free cortisol (mUFC) and acceptable safety in a phase 3 trial of patients with Cushing's syndrome.
Why this matters
- Levoketoconazole may represent a useful therapeutic option.
- 81% of patients who advanced to the maintenance phase had a complete response (mUFC ≤upper limit of normal) by end-of-dose titration.
- 31% of patients showed a response at the end of the 6-month maintenance phase.
- The least-squares mean estimate of the proportion of responders was 0.30 (P=.0154 vs null hypothesis of ≤0.20).
- Significant mean improvements were observed for:
- Fasting blood glucose (P<.0001>
- HbA1c (P<.0001>
- Total and low-density lipoprotein cholesterol (both P<.0001>
- Body weight (P<.0001>
- Acne global score (P=.0063).
- Hirsutism total score in women (P=.0008).
- Peripheral edema total score (P=.030).
- 94 patients with Cushing's syndrome were treated with levoketoconazole and analyzed for efficacy and safety outcomes.
- Funding: Strongbridge Biopharma.
- Small patient sample size.
- Open-label study.
- Nonrandomized, single-group study.