Recordati Rare Diseases Will Market Signifor, Signifor LAR in U.S.
The medications will be marketed through Recordati Rare Diseases’ newly established endocrinology franchise, the company said. During the transfer of marketing rights, both medications will continue to be available for patients and healthcare members with no disruption, via a direct distribution model with a specialty pharmacy.
The company provides a copay assistance program to help eligible patients with their insurance copayments or co-insurance, and a patient assistance program to aid eligible uninsured or underinsured patients receive Signifor and Signifor LAR.
Recordati acquired worldwide rights to Signifor and Signifor LAR from Novartis in 2019. The treatments contain the active ingredient pasireotide, which acts in receptors in the pituitary glands and lowers the production of the adrenocorticotrophic hormone (ACTH) – a hormone that is produced in excess by a pituitary tumor in people with Cushing’s disease.
The two medications are approved for people with Cushing’s disease for whom surgery has failed or is not an option. Signifor is given as a twice-daily, under-the-skin injection, and Signifor LAR is a long-acting formulation that is injected into the muscle every four weeks.
Signifor was approved by the U.S. Food and Drug Administration in 2012, based on positive results from the Phase 3 B2305 trial (NCT00434148) showing that the medicine was effective at lowering cortisol levels.
The trial tested two Signifor doses (600 micrograms and 900 micrograms), both given under the skin and twice daily, in 162 patients. The main objective was to assess the proportion of patients whose cortisol levels returned to normal after six months without increasing the dose – a goal that was met by 15% of those receiving 600 mcg, and 26% of those on 900 mcg dose.
Treatment with Signifor also lessened the signs and symptoms of the disease after six and 12 months, including decreases in blood pressure, fat molecules, weight, and improvements in health-related quality of life. In some patients, fat deposits were also smaller.
Findings also showed reductions in tumor volume, particularly among those receiving the higher dose, who experienced an average 44% decrease in tumor volume after six months.
The treatment continued to benefit patients in the long term, reducing their cortisol levels even further after they entered an extension trial that followed the 12-month treatment period in the initial trial.
As with Signifor, the long-acting formulation also improved other symptoms of the disease, including high blood pressure and body weight, and reduced pituitary tumors in a large proportion of patients.