NDA Submitted for Potential Severe Jaundice Therapy

Mallinckrodt plc has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA), as per an official announcement from the company yesterday.

The NDA is for its recently acquired developmental product stannsoporfin which, if approved, will become the first and only pharmacologic option in the United States indicated for the treatment of neonates at risk for developing severe hyperbilirubinemia, or severe jaundice.

Hyperbilirubinemia is a condition in which there is an excess of bilirubin in the blood. When red blood cells break down, bilruibin forms, which can cause a buildup of the substance in the blood and other tissues. Common symptoms of the condition include yellow staining of the skin and the whites of the eyes (sclerae). The discoloration extends to other areas of the body, and may also turn bodily fluids, including urine, darker than normal.

The combined potential patient treatments required annually in the U.S. for severe jaundice is estimated between 70,000 and 125,000.

"We are pleased to complete this important milestone for stannsoporfin," said Steven Romano, M.D., Chief Scientific Officer and Executive Vice President of Mallinckrodt in a press release. "This brings us one step closer to addressing an unmet need for therapies to treat thousands of infants at risk for severe jaundice, and we look forward to working closely with the FDA toward the goal of obtaining approval of stannsoporfin to treat this population."

Stannsoporfin is a heme oxygenase inhibitor, and its safety and efficacy have not yet been established by the FDA. If approved, it is expected to be used for late-preterm and full-term infants at risk of developing complications associated with severe jaundice.

In December 2016, stannsoporfin was granted Fast Track designation by the FDA, a process designed to facilitate development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. In August, Mallinckrodt entered into an agreement under which it acquired InfaCare, a privately held specialty pharmaceutical company, and with that, acquired stannsoporfin.

A Phase 2 study evaluating the safety and efficacy of 2 doses of stannsoporfin in combination with phototherapy in neonates with hyperbilirubinemia enrolled 91 participants and was completed in July 2017.

For more from the FDA, including applications, designations and approvals, follow Rare Disease Report on Facebook and Twitter.