Recently, biopharmaceutical company Strongbridge Biopharma plc (“Strongbridge”) shared that the company submitted a New Drug Application (NDA) to the FDA. The NDA is centered around RECORLEV (levoketoconazole), a treatment option for patients with endogenous Cushing syndrome (sometimes known as endogenous Cushing‘s syndrome). Currently, the standard-of-care includes surgery, medications, or even radiation treatment. However, many patients still struggle to manage their symptoms. Thus, RECORLEV offers a possible opportunity to fill this unmet need.
So what is RECORLEV? RECORLEV is an investigational cortisol synthesis inhibitor which received Orphan Drug status in both Europe and the United States. It is a pure 2S,4R enantiomer of ketoconazole. The NDA was supported by safety and efficacy data sourced from two Phase 3 clinical trials evaluating RECORLEV for endogenous Cushing syndrome.
In the Phase 3 SONICS trial, 94 patients enrolled. The trial worked in three phases: dose titration, maintenance, and extended evaluation. Within the first phase, patients received 150mg RECORLEV 2x daily and titrated by 150mg until a therapeutic dose was determined. In the secondary phase, the dose was kept the same for 6 months before evaluating the response rate. Finally, patients remained on RECORLEV for an additional 6-month period to analyze safety, efficacy, and tolerability. Ultimately, the SONICS trial showed that RECORLEV normalized cortisol levels. See the full results from the SONICS trial here.
In the Phase 3 LOGICS trial, 79 patients enrolled. Of these 79, 7 were previously treated during the SONICS trial, while 72 had not received prior RECORLEV therapy. Once again, RECORLEV was shown to normalize cortisol and reduce negative symptoms. To see the full results of the LOGICS trial, click here.
Altogether, this data was submitted alongside the NDA. The FDA describes the New Drug Application as:
the vehicle through which drug sponsors formally propose that the FDA approve a new pharmaceutical for sale and marketing in the U.S.
Also known as hypercortisolism, Cushing syndrome (not to be confused with Cushing disease, one of its subtypes), is an endocrine disorder which causes excessive cortisol in the blood. Using too many oral corticosteroids may cause Cushing syndrome. However, the endogenous Cushing syndrome means it has a known cause, typically the body’s overproduction of the hormone. In many cases, this is caused by a benign pituitary tumor. Generally, Cushing syndrome affects those between ages 30 and 50. It is also more common in females than males. Without treatment, many people with Cushing syndrome may only live for 5 years.
- A fatty hump between the shoulders, in your midsection, around your face, or on the upper back
- Pink or purple stretch marks
- Unintended weight gain/obesity
- Increased skin pigmentation
- Thin, easily bruised skin
- High blood sugar and blood pressure
- Changes in mood, such as anxiety, depression, and irritability
- Muscle weakness
- Bone loss
- Extra facial/body hair (for females)
- Absent or irregular menstruation (for females)
- Erectile dysfunction (for males)
- Low libido
Learn more about Cushing syndrome.