'Orphan medication' in Europe

October 21, 2005

The European Agency for the Evaluation of Medicinal Products (EMEA) has designated, as an orphan medicine, one of the 18 patents of the Centro de Investigacion Medica Aplicada (CIMA) at the University of Navarre. This approval, achieved by the Digna Biotech biotechnological company, will facilitate the clinical development of its p144 peptide in the treatment of two symptoms of skin disease: local and systemic scleroderma.

The aim of Digna Biotech (www.dignabiotech.com) is the development of the intellectual property CIMA at the University of Navarre on pre-clinical, clinical and commercial planes.

This designation of p144 will mean a series of advantages for the clinical development that Digna Biotech is to undertake: free access to scientific advice from EMEA during the clinical phase of the design process, reduction in the taxes payable for the permission to market the product and, once authorised by the Agency, to maintain market exclusivity for a period of ten years.

According to Dr. Pablo Ortiz, Director General of Digna Biotech, this approval by EMEA will speed up the clinical development and reduce costs of the product. It is the first time in the Spain that EMEA has conceded the simultaneous designation as an orphan medicine of a product involving two distinct symptoms, thereby endorsing the consistency of the data presented to the Agency and the interest of this new product for the public at large.

Also, Dr. Juan Ruiz, medical director of Digna Biotech, in reference to the commitment to improve current treatment of local and systemic scleroderma, pointed out that they are chronic conditions which, while not having a very high prevalence amongst the public, they can considerably diminish the quality of life of the patient.

Scleroderma and p144 in cream form

Scleroderma is a chronic illness that is characterised by fibrosis in the skin, blood vessels and internal organs such as the lungs. It is believed that excess of TGF Beta 1 (Transformant Growth Factor ¦Â) may be one of the key factors in the development of this pathology. Currently there is no treatment for its cure, only symptomatic treatments of doubtful efficacy.

Scleroderma is a rare or uncommon illness: it is estimated that it may affect between 37,000 and 72,000 persons within the EU, a prevalence of 0.82 to 1.58 cases per 10,000 persons. This low incidence further impedes research into new treatments.

p144 is an inhibitor of TGF Beta 1. It is currently considered that inhibitors of TGF Beta 1 may play a key role against fibrosis and, thereby, in improving sclerodermic conditions.

This is why Digna Biotech is developing p144 in a cream form for the treatment of cutaneous lesions resulting from local and systemic scleroderma.

Simultaneously, Digna Biotech is investigating other symptoms potentially treatable with p144, such as pulmonary fibrosis, keloid scars, fibrosis arising from the implantation of a foreign body and the macular degeneration of the retina.
-end-


Elhuyar Fundazioa

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