The European Commission, acting on the positive recommendation from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP), has granted orphan drug designation to Soligenix, Inc's synthetic hypericin (the active pharmaceutical ingredient in SGX301) for the treatment of cutaneous T-cell lymphoma (CTCL), a rare disease and a class of non-Hodgkin's lymphoma, a type of cancer of the white blood cells that are an integral part of the immune system.

SGX301 has previously been granted both orphan drug and fast track designations from the US Food and Drug Administration (FDA) for the first-line treatment of CTCL.

Soligenix is currently working with leading CTCL centers, as well as with the National Organisation for Rare Disorders (NORD) and the Cutaneous Lymphoma Foundation (CLF) to begin a 120 subject pivotal phase 3 clinical trial with SGX301 in the second half of 2015. SGX301 is a novel, first-in-class, photodynamic therapy utilising safe, visible light for activation. Synthetic hypericin is a potent photosensitizer which is topically applied to skin lesions and activated by visible fluorescent light. This treatment approach avoids the risk of secondary malignancies (including melanoma) inherent with the frequently employed DNA-damaging chemotherapeutic drugs and other photodynamic therapies that are dependent on ultraviolet exposure. Topical hypericin has demonstrated safety in a phase 1 clinical study in healthy volunteers. In a phase 2, double-blind, placebo-controlled clinical study in CTCL patients, the drug was safe and well tolerated, with 58.3 per cent of the CTCL patients responding to SGX301 treatment compared to only 8.3 per cent receiving placebo (p = 0.04).

The European Commission grants orphan designations for medicines that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the EU and where no satisfactory treatment is available. In addition to a 10-year period of marketing exclusivity in the EU after product approval, orphan drug designation provides incentives for companies seeking protocol assistance from the EMA during the product development phase, and direct access to the centralised authorisation procedure.

"We are extremely pleased to have received European orphan drug designation for the SGX301 programme," stated Christopher J. Schaber, PhD, president & chief executive officer of Soligenix, Inc. "This EU orphan designation, combined with our US orphan and fast track designations, positions SGX301 for an accelerated global regulatory product development pathway to address the significant unmet need that exists for this important indication. We look forward to aggressively advancing our pivotal phase 3 study of SGX301 in CTCL."

CTCL is a class of non-Hodgkin's lymphoma (NHL), a type of cancer of the white blood cells that are an integral part of the immune system.  Unlike most NHLs which generally involve B-cell lymphocytes (involved in producing antibodies), CTCL is caused by an expansion of malignant T-cell lymphocytes (involved in cell-mediated immunity) normally programmed to migrate to the skin. These malignant cells migrate to the skin, causing various lesions to appear that may change shape as the disease progresses, typically beginning as a rash and eventually forming plaques and tumors. Mortality is related to the stage of CTCL, with median survival generally ranging from about 12 years in the early stages to only 2.5 years when the disease has advanced. There is currently no cure for CTCL.

CTCL constitutes a rare group of NHLs, occurring in about 4 per cent of the approximate 500,000 individuals living with the disease. It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of CTCL that it affects over 20,000 individuals in the US, with approximately 2,800 new cases seen annually.

SGX301 is a novel first-in-class photodynamic therapy utilizing safe visible light for activation. The active ingredient in SGX301 is synthetic hypericin, a potent photosensitizer which is topically applied to skin lesions and then activated by fluorescent light 16 to 24 hours later. Combined with photoactivation, hypericin has demonstrated significant anti-proliferative effects on activated normal human lymphoid cells and inhibited growth of malignant T-cells isolated from CTCL patients. In a published phase 2 clinical study in CTCL, patients experienced a statistically significant (p = 0.04) improvement with topical hypericin treatment whereas the placebo was ineffective: 58.3 per cent compared to 8.3 per cent, respectively. SGX301 has received orphan drug and fast track designations from the FDA.

Soligenix is a late-stage biopharmaceutical company developing products that address unmet medical needs in the areas of inflammation, oncology and biodefense.