EMA grants Sucampo orphan drug designation for unoprostone isopropyl to treat retinitis pigmentosa

May 29 2013

Sucampo Pharmaceuticals, Inc. ("Sucampo") (NASDAQ: SCMP), a global biopharmaceutical company, today announced that it has received orphan drug designation in the European Union for unoprostone isopropyl for the treatment of retinitis pigmentosa (RP). RP is a genetic disease characterized by progressive, irreversible vision loss and decreasing visual acuity, and there are no drugs or therapeutic procedures currently approved for the treatment of RP today.

“We are very pleased to receive orphan drug designation by the EMA for unoprostone isopropyl for the treatment of RP”

To qualify for orphan designation by the European Medicines Agency (EMA), a medicine must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating; the prevalence of the condition in the EU must not be more than 5 in 10,000; and no satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorized, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition¹.

"We are very pleased to receive orphan drug designation by the EMA for unoprostone isopropyl for the treatment of RP," said Ryuji Ueno, M.D., Ph.D., Ph.D., Chairman, Chief Executive Officer, and Chief Scientific Officer of Sucampo. "RP is a significant burden to the patients and caregivers whose lives are affected by the disease. The granting of this orphan drug designation is yet another important milestone for unoprostone isopropyl as we continue in our development of the product to address the unmet needs of patients afflicted with this disease."

As previously announced, in February of this year R-Tech Ueno, Ltd., Sucampo's development partner, signed an agreement for unoprostone isopropyl with the Japan Science and Technology Agency in which the Japanese government shall provide the majority of funding for phase 3 clinical development costs for unoprostone isopropyl for retinitis pigmentosa (RP). In the first quarter, R-Tech announced the enrollment of the first patient in this program. Sucampo is co-developing unoprostone isopropyl with R-Tech and may file for EMA and U.S. Food and Drug Administration (FDA) approval of the product for RP in the future assuming successful trials. A form of unoprostone isopropyl is currently approved in the United States. In addition, the FDA has granted orphan drug designation to unoprostone isopropyl for treatment of RP.

RP causes the degeneration of photoreceptor cells in the retina. Photoreceptor cells capture and process light, helping us to see. As these cells degenerate and die, patients experience progressive vision loss². Blindness from all causes is among the most significant injuries to a patient's qualify of life and is a major driver of patient-based cost of care and lifestyle maintenance.