Strongbridge Biopharma has just announced that the FDA has decided to review the New Drug Application for RECORLEV, a therapy for endogenous Cushing’s syndrome. The target action date is January 1st, 2022.
This acceptance for review is based on the results from two Phase 3 investigations which both produced positive results.
Researchers are hopeful that this therapy could become a new option for patients by the first quarter of 2022.
Cushing’s syndrome is a rare condition which is much more prevalent in women than men and in individuals from 30-50.
It is an endocrine disease which is caused by a chronic elevated cortisol exposure which typically results from a tumor located on the pituitary gland. This benign tumor causes the body to overproduce cortisol, which influences the body both externally and internally.
Females may experience the following symptoms-
- Issues with the menstrual cycle
- Excess production of androgens, including testosterone
- Oily skin
Internal complications of the condition include-
- High blood sugar
- High blood pressure
- Fragile tissues (such as bone, muscle, skin, and blood vessels)
- High cholesterol
RECORLEV is an adrenal steroidogenesis inhibitor which works to suppress and inhibit serum cortisol. The therapy has already received Orphan Drug Designation from the EMA and FDA.
The two studies which led to RECORLEV’s NDA review were Phase 3 investigations called SONICS and LOGICS.
This study was an open-label and multicenter investigation including 94 patients from Europe, North America, as well as the Middle East. There were 3 treatment phases.
- Dose Titration- All participants began with 150mg of the therapy twice each day. The does was then titrated at 150mg increments. When mUFC was normalized, the titration was stopped.
- Maintenance- For 6 months, patients were monitored at the same dose. At the end of the phase, mUFC RR was measured.
- Extended Evaluation- All patients continued the therapy for another 6 months in order to understand the safety, tolerability, and efficacy over a longer time period.
The primary endpoint and secondary endpoint of this trial were met. mUFC was normalized at 6 months.
This study was a placebo-controlled, double-blind, multinational trial. 79 patients were included in the open-label titration-maintenance phase. Of these, 7 were previously treated in the SONICS study. At baseline, the median mUFC was 3.5 times the upper limit of normal.
44 patients were randomized to receive the treatment or placebo for 8 weeks. Following this period, all patients were given the therapy again. Of those in the treatment group, 11 patients had been enrolled in the SONICS study.
This study met both its primary and secondary endpoints.
The OPTICS study will now allow patients to continue to receive the therapy open-label and allow researchers to continue to evaluate its long-term effects and benefits.
You can read more about this study and the NDA here.