Pharming’s Prodarsan obtains orphan drug status

The Netherlands-based biotechnology company Pharming Group has reported that its wholly owned subsidiary DNage has received a notice from the FDA that its product Prodarsan has obtained an orphan drug designation for the treatment of Cockayne syndrome.

Prodarsan is a combination of two small molecules, formulated for oral use, which are believed to reduce the accumulation of DNA-damage, the underlying biochemical cause of Cockayne syndrome. Cockayne syndrome, the initial target of Prodarsan, is a rare genetic disease in which children suffer from accelerated (or premature) aging, while developing severe aging diseases.

DNage has demonstrated that Prodarsan has significant beneficial effects in animal models for Cockayne syndrome. Furthermore, it has shown that the product was safe and well-tolerated in a study in human volunteers. In consultation with the FDA, the company is now preparing to submit an investigational new drug application that will allow the initiation of additional clinical studies in patients.

The FDA’s orphan drug designation is reserved for new therapies being developed to treat diseases or conditions that affect fewer than 200,000 people in the US. The orphan drug designation provides for an accelerated review process, tax benefits, exemption from user fees and a seven-year period of market exclusivity in the US after product approval.

The designation is awarded based on an application describing the disease, the product and an extensive outline of the proposed development plan. The application for Prodarsan was filed by Clinquest, DNage’s agent in the US.