FDA grants 12-year exclusivity to RUCONEST (C1 esterase inhibitor [recombinant])

Salix Pharmaceuticals and Pharming Group NV (EURONEXT: PHARM) announced today that the U.S. Food and Drug Administration (FDA) has granted 12 years of exclusivity to RUCONEST^® (C1 esterase inhibitor [recombinant]) 50 IU/kg. The determination of exclusivity ensures that FDA will not approve before July 16, 2026 any applications for biosimilars of RUCONEST— i.e. applications for recombinant C1 esterase inhibitors referencing RUCONEST submitted under section 351(k) of the Public Health Service Act under the framework established by the Biologics Price Competition and Innovation Act of 2009.

RUCONEST was approved by the FDA on July 16, 2014, for the treatment of acute angioedema attacks in adult and adolescent patients with hereditary angioedema (HAE). Effectiveness was not established in HAE patients with laryngeal attacks.

A rare condition occurring in about 1 in 10,000 to 1 in 50,000 people worldwide, HAE is potentially life-threatening and stems from a genetic defect that impacts the production of C1-INH protein. This can lead to a biochemical imbalance that causes swelling in various parts of the body, including the hands, feet and face. Swelling in the intestinal wall can also cause severe abdominal pain, nausea and vomiting. Due to its rarity, many patients often remain undiagnosed for years.

RUCONEST is contraindicated in patients with a history of allergy to rabbits, or rabbit-derived products, and patients with a history of life-threatening immediate hypersensitivity reactions to C1 esterase preparations, including anaphylaxis.

Under the Biologics Price Competition and Innovation Act of 2009, exclusivity for licensed biologics—like RUCONEST—can be granted for a 12-year period from the date of first licensure of the product.

"We are pleased the anticipated exclusivity for RUCONEST has been formally granted," said Deb Jorn, Executive Vice President/Company Group Chairman, Valeant Pharmaceuticals. "The response to RUCONEST has been positive since its launch in November 2014 and we look forward to continued growth."

Sijmen de Vries, Pharming's CEO, commented: "Pharming strived to make RUCONEST available to the HAE patient community in the US, because we were aware of the great value and benefits that RUCONEST could provide to patients. That the FDA granted 12 year exclusivity for RUCONEST reinforces this long-standing commitment and we are excited to continue to work closely with Salix to ensure patients in the US have access to RUCONEST."

SOURCE Salix Pharmaceuticals