US FDA says no to orphan status for DUSA Pharma's skin cancer drug Levulan

USA-based DUSA Pharmaceuticals received a disappointment last week, when the Food and Drug Administration notified the company that it has not granted Orphan Drug Designation for the use of Levulan (aminolevulinic acid HCl) Photodynamic Therapy (PDT) for the prevention of cutaneous squamous cell carcinomas (SCCs) in patients who have a proven history of multiple localized cutaneous SCCs over a 12 month period. The news saw the firm's stock tumble 12.7% to $2.20.

The FDA acknowledged that cutaneous SCC is a serious problem in patients at high risk for developing SCCs, such as solid organ transplant recipients (SOTRs), and that aminolevulinic acid would be a potential preventative therapy in these patients. However, the agency also stated that it believes there are other factors which place patients at high risk of developing SCCs that should be included in determining the target population. To be eligible for Orphan Drug Designation, the drug must target a disease that affects fewer than 200,000 patients in the USA.

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