Italian drugmaker Recordati is acquiring worldwide rights to Novartis’ rare endocrine disease drugs Signifor, its long acting sister drug Signifor LAR, and osilodrostat, a follow-up treatment in late stage development, for $390 million in cash upfront.

Signifor and Signifor LAR are marketed for the treatment of Cushing’s disease and acromegaly in adult patients for whom surgery is not an option or for whom surgery has failed. Worldwide sales of Signifor were $72 million in 2018. Osilodrostat is in development to treat endogenous Cushing’s syndrome, and marketing authorization applications have been filed in the European Union and in the United States.

Under the terms of their agreement, Recordati is eligible for additional milestone payments contingent upon the approval and market access of osilodrostat, as well as royalties on sales of the new product. Novartis will supply these products to Recordati during a transitional period.

“These important additions to our product portfolio of treatments for severe rare diseases represent a key and historical milestone for Recordati,” said Andrea Recordati, CEO.

Cushing’s disease, a form of Cushing’s syndrome, is a severe endocrine disease caused by an enlargement in the pituitary gland that results in over-production of cortisol by the adrenal glands. It usually affects adults between the ages of 20 and 50 years, and leads to upper body obesity, fatigue, muscle weakness, high blood pressure, backache, high blood sugar, easy bruising, and bluish-red stretch marks on the skin. Among the causes are long-term use of corticosteroid medications, tumors in the pituitary gland, and adrenal adenomas. Treatment depends on the underlying cause, but may include decreasing the dosage of corticosteroids or surgery to remove tumors.

Acromegaly is a rare, slowly progressive, acquired disorder that affects adults. It occurs when the pituitary gland produces too much growth hormone, and is most commonly caused by a pituitary adenoma. Symptoms include abnormal enlargement in bones of the hands, arms, feet, legs, jaws, and in the front of the skull. It may also cause thickening of the soft tissues of the body, including the heart, lips, and tongue. When the disease affects children prior to the end of puberty, excess growth hormone can lead to accelerated growth and tall stature, known as gigantism. If untreated, acromegaly can potentially cause serious illness and life-threatening complications.

Signifor contains the active substance pasireotide, a somatostatin analogue. The human body naturally produces somatostatin, which blocks the production and release of certain hormones, including ACTH. Pasireotide works in a very similar way to somatostatin, blocking the production of ACTH, and helping to control the over-production of cortisol to improve the symptoms of Cushing’s disease. Signifor is an injectable that must be administered by a physician.

Novartis had hoped that Signifor would add to sales of its acromegaly treatment Sandostatin, which has been off-patent for many years but still brings in about $1.9 billion in annual sales. But that didn’t happen.

Osilodrostat is an orally administered steroidogenesis inhibitor of 11Beta-hydroxylase, an enzyme which catalyzes the final step of cortisol synthesis in the adrenal cortex. This new drug for endogenous Cushing’s syndrome is expected to represent an effective new treatment option for patients.

Photo: Andrea Recordati, CEO of Recordati

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