The PIM designation is the first step towards inclusion in the Early Access to Medicines Scheme (EAMS). Launched in April 2014, EAMS offers severely ill patients with life-threatening and seriously debilitating conditions the lifeline of trying ground-breaking new medicines much earlier than they would normally be accessible.
PIM status, the first phase of EAMS, which is awarded following an assessment of early nonclinical and clinical data by the MHRA, has been created as an early signal to companies that the development plan is appropriate and indicates that a product could be a candidate for the second phase of the EAMS scheme, once further development work has been conducted.
In this second phase, the product is made available to UK patients before a marketing authorization is approved. This early boost to a drug’s potential is expected to be beneficial to companies, especially small and medium-sized enterprises.
The criteria products must meet to obtain the PIM designation are:
- Criterion 1 – The condition should be life-threatening or seriously debilitating with a high unmet medical need (i.e., there is no method of treatment, diagnosis or prevention available or existing methods have serious limitations).
- Criterion 2 – The medicinal product is likely to offer major advantage over methods currently used in the UK.
- Criterion 3 – The potential adverse effects of the medicinal product are likely to be outweighed by the benefits, allowing for the reasonable expectation of a positive benefit risk balance. A positive benefit risk balance should be based on preliminary scientific evidence, as justified by the applicant, that the safety profile of the medicinal product is likely to be manageable and acceptable in relation to the estimated benefits.
Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix, said: “We are excited that the MHRA agrees that SGX301 meets the specified criteria for PIM designation.
“This is now the second of our programs to receive this important designation, with the first being SGX942 for the treatment of severe oral mucositis in head and neck cancer patients receiving chemoradiation therapy. We look forward to working with the MHRA to advance both programs and leverage the potential benefits of the EAMS scheme to make these important products available to patients and physicians facing the challenges of CTCL and severe oral mucositis.”
About Cutaneous T-Cell Lymphoma
Cutaneous T-cell lymphoma (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% 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.
About SGX301
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 that is topically applied to skin lesions and then activated by fluorescent light 16 to 24 hours later. 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.
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% compared to 8.3%, respectively. SGX301 has received orphan drug and fast track designations from the US Food and Drug Administration (FDA), as well as orphan designation from the European Medicines Agency (EMA).