Approved Therapy Recorlev Now Wins FDA Orphan-drug Exclusivity

US patent on treatment for endogenous Cushing’s good until 2040

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by Steve Bryson, PhD |

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The U.S. Food and Drug Administration (FDA) has granted Xeris Pharmaceuticals orphan-drug exclusivity for Recorlev (levoketoconazole), its approved treatment for adults with endogenous Cushing’s syndrome.

Orphan-drug exclusivity provides the company — which recently acquired the therapy’s developer, Strongbridge Biopharma — seven years of marketing exclusivity for Recorlev in the U.S.

The therapy previously was awarded orphan drug status by the FDA, a designation given to medications that aim to treat rare medical conditions, such as Cushing’s. During development, that status typically provides tax credits for clinical trials and an exemption from user fees.

This new entitlement is in addition to the patent held by Xeris in the U.S., which covers Recorlev and its medicinal use, and extends at least until March 2040.

“We are excited to receive this important orphan-drug exclusivity approval for Recorlev on a new therapeutic option that can address symptoms while treating the root cause of the disease for this underserved Cushing’s patient community,” Paul R. Edick, Xeris’ chairman and CEO, said in a press release.

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Long-term Recorlev Found Safe, Effective for Endogenous Cushing’s

Orphan-drug exclusivity good for 7 years

Recorlev was approved by the FDA in late 2021 for the treatment of endogenous Cushing’s syndrome, and launched by Xeris early last year. It is available to patients in the U.S. through the specialty pharmacy PANTHERx Rare.

To help patients access Recorlev, the company established Xeris CareConnection. The program, for patients and their caregivers, provides financial assistance, one-on-one support, and educational resources, as well as help with access and reimbursement for healthcare professionals.

Endogenous Cushing’s syndrome is marked by the excess production of the stress hormone cortisol in the body, which leads to symptoms like weight gain, increased body fat, and skin problems. Over time, other complications may arise, such as high blood pressure and cholesterol levels, diabetes, and mood problems like depression and anxiety.

“Cushing’s syndrome is a rare disease that can be physically and emotionally devastating to the patient,” Edick said. “Most patients endure years of symptoms prior to obtaining a diagnosis and are then faced with limited effective treatment options.”

Cushing’s disease is a subtype of the syndrome in which a tumor in the brain’s pituitary gland results in the excessive production of cortisol. Surgical removal of the pituitary tumor is the first-line treatment for Cushing’s disease patients, but for some, the procedure is ineffective at lowering cortisol levels or is not an option.

Recorlev, which is designed to block cortisol production in the body, is indicated for people with Cushing’s syndrome for whom surgery is not an option or has not been effective.

The recommended starting dose is 150 mg twice daily, with the option to increase up to a maximum dose of 600 mg twice daily, or 1,200 mg total.

We are excited to receive this important orphan-drug exclusivity approval for Recorlev on a new therapeutic option that can address symptoms while treating the root cause of the disease for this underserved Cushing’s patient community.

The therapy’s approval was mainly supported by data from two Phase 3 clinical trials: SONICS (NCT01838551) and LOGICS (NCT03277690), which jointly involved a total of 166 patients.

SONICS included 94 adults with endogenous Cushing’s syndrome, whose 24-hour urine free cortisol levels were at least 1.5 times higher than normal, and who were unable to undergo radiation therapy or surgery.

Two-year SONICS data, recently published, demonstrated that long-term Recorlev treatment safely led to sustained reductions in cortisol levels and lessened disease signs and symptoms. The data showed the treatment eased depression and enhanced patient quality of life.

LOGICS was a placebo-controlled withdrawal and rescue study that assessed the safety, efficacy, and pharmacological properties of Recorlev in 79 endogenous Cushing’s syndrome patients. Eligible participants were those who had participated in SONICS or had never received Recorlev.

Complete trial data showed Recorlev outperformed the placebo at normalizing urine cortisol levels. In fact, more than 95% of participants who first received Recorlev and then switched to a placebo saw their cortisol levels rise above normal. Treatment also lowered cholesterol levels and body fat compared with the placebo.

“The approval of Recorlev was based upon data from two positive Phase 3 studies that evaluated a combined study population of 166 patients and was shown to be effective for reducing and normalizing cortisol,” Edick said.

Safety data from an ongoing Phase 3 extension study called OPTICS (NCT03621280) also supported the therapy’s approval. Slated to wrap up in June of this year, the trial is investigating the long-term effects of Recorlev in those who completed one or both previous studies, for up to three years.

“Recorlev is an important and welcome therapeutic option for clinicians to help manage patients with endogenous Cushing’s syndrome, a severe, potentially life-threatening rare disease,” Edick said.