FDA grants orphan drug status to experimental Cushing’s therapy
Asedebart is being tested in a Phase 2 trial for Cushing’s disease
Written by |
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Lundbeck’s asedebart, a designation that may provide incentives for the development of this experimental antibody for endogenous Cushing’s syndrome. A Phase 2 trial is currently underway in people with Cushing’s disease, one form of the syndrome.
“This milestone reflects the strength of the science behind asedebart’s development to date and supports our ambition to advance innovative medicines in areas where patients continue to face significant unmet need,” Tarek Samad, PhD, Lundbeck’s executive vice president, said in a company press release.
FDA designation may provide development incentives
The designation may provide incentives such as tax credits for qualified clinical testing, exemption from certain FDA application fees, and, if approved, potential seven years of market exclusivity for the designated indication. Samad, who is also Lundbeck’s head of research and development, said the designation “is an important step for asedebart and for Lundbeck’s growing commitment to rare neuroendocrine disorders.”
The ongoing Phase 2 clinical study, called BalanCeD (NCT06471829), is still recruiting adults with a diagnosis of Cushing’s disease at more than a dozen locations worldwide. Preliminary data from the first part showed that seven of eight participants who completed intravenous dose adjustment achieved normal urinary free cortisol levels. Asedebart was also generally well tolerated.
Symptoms of endogenous Cushing’s syndrome occur when the body produces an excess of the hormone cortisol. Some forms are driven by excess adrenocorticotropic hormone (ACTH), which signals the adrenal glands above the kidneys to produce cortisol. The excess ACTH may come from a pituitary tumor, in which case the condition is called Cushing’s disease. Less commonly, it comes from a tumor elsewhere in the body.
Current treatments can reduce cortisol levels, but long-term disease control can be difficult. For ACTH-dependent Cushing’s syndrome, surgery to remove the source of excess ACTH is the preferred first-line treatment when feasible, but some patients cannot undergo surgery or do not achieve lasting remission, meaning the disease does not remain under control.
Asedebart, also known as Lu AG13909, is an antibody designed to bind ACTH and prevent it from activating the melanocortin 2 receptor in the adrenal glands, blocking ACTH signaling that stimulates adrenal hormone production. The therapy is being developed to reduce excess hormone production in Cushing’s disease and other ACTH-dependent disorders.
Phase 2 trial is testing asedebart in Cushing’s disease
Lundbeck is sponsoring BalanCeD to evaluate asedebart’s effects, safety, and pharmacokinetics — how the treatment moves into, through, and out of the body. The study aims to enroll up to 18 patients, ages 18 to 70, who have Cushing’s disease with evidence of excess ACTH from a pituitary tumor.
The study has three parts. In Part A, patients receive asedebart through an intravenous (into-the-vein) infusion, with the dose adjusted based on their cortisol levels. They then switch to a subcutaneous (under-the-skin) injection. In Part B, researchers further adjust the subcutaneous dose to normalize cortisol levels. Patients then enter an extension in which asedebart is tested for long-term safety and efficacy.
Earlier this year, asedebart also received orphan drug designation in Japan for the treatment of Cushing’s disease and congenital adrenal hyperplasia, which also involves abnormally high ACTH levels, and in the European Union for the treatment of endogenous Cushing’s syndrome.
Leave a comment
Fill in the required fields to post. Your email address will not be published.