EMEA Grants Cellerix, S.L. Orphan Drug Status To CX501 For The Treatment Of Epidermolysis Bullosa

Published: Jun 14, 2006

Madrid, June 2006 - Cellerix is developing specialised cell-based therapies for the treatment of perianal fistula (Cx401) and Epidermolysis bullosa (Cx501). Its Cx501 product has been granted orphan drug status by the Committee of Orphan Medicinal Products (COMP) from the European Medicines Agency (EMEA). Cx501 is a novel bilayered engineered skin composed of keratinocytes from the patient and fibroblasts from a donor embedded in a plasma matrix. This product allows for the treatment of an illness which severely restricts the quality of patients, and for which there is currently no adequate cure.

Cellerix was founded in 2004 with the objective of exploring the therapeutic potential offered by the latest developments related to the use of adult stem cells for the regeneration and repair of tissues. Together with Cellerix’s Cx401 product for fistula treatment in Crohn’s and non-Crohn’s patients, Cx501 is thus far the second cell-therapy medicine to have been granted Orphan Status in Europe.

Epidermolysis bullosa (EB) is a rare, genetically inherited disease, in which the patient is unable to produce collagen VII, one of the structural proteins of the skin. As a result, the skin of these patients is extremely fragile and develops blisters after minimal friction. There is no current treatment for these patients and their life quality and expectancy is severely compromised. Depending on the type of EB, the disease may also affect other tissues in the body. EB patients are at a high risk of developing infections, and skin cancer at a relatively early age.

Cx501 is an artificial skin graft obtained from bringing together keratinocytes from the patient and skin fibroblasts from a healthy donor. Both components are embedded in a plasma matrix which provides the adequate environment for natural healing to occur. These healthy fibroblasts provide the patient with the structural protein that is lacking in the skin of EB patients. Cx501 has demonstrated promising results in a pilot assay and is currently entering phase II.

Eduardo Bravo, CEO of Cellerix, explains “this second Orphan designation for one of our products positions the company as a reference in cell-based therapeutic treatments in Europe. Cellerix will continue making strides in the clinical development of cell-based therapeutic indications for improving the lives of sufferers with these rare and debilitating illnesses.“.

Orphan Drugs

Orphan drug status is awarded to medicinal products aimed at preventing or treating life-threatening, or very serious conditions that are rare (less than 5 sufferers per 10,000). Up until May 2006, only 300 medicinal products have received Orphan drug status by the EMEA.

This designation allows Cellerix to benefit from the incentives provided to these types of medicinal products, which amongst others, include market exclusivity for 10 years, fee reductions, and access to EU-funded research, as well as facilitating a faster time to market.

Cell-based medicinal products

In the different tissues that make up the human body, so called stem cells can be found which are capable of transforming themselves into a wide variety of different human tissues. At present, it is possible to isolate and multiply these cells for subsequent transplantation into the body in places where tissue regeneration is needed. It allows cells to be used as “real living medicines”, paving the way for new therapeutic applications.

Cellerix SL

Cellerix is a Spanish biotechnology company actively developing and producing advanced therapies based on the use of stem cells of adult origin. The company has a developing product pipeline, and has just completed Phase II trials in 50 patients for the treatment of complex perianal fistulas.

Cellerix’s strategy is to target niche indications for which there is a high unmet medical need. The company is led by a team with strong expertise in clinical trials and regulatory issues related to cell therapy products.

For more information, please contact: Vicente Díaz Communication Manager Tel. +34 (91) 804 92 64 ext. 209 e-mail: vdiaz@genetrix.es

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