One of the 18 patents submitted by the Centre for the Study of Applied Medicine (CIMA) at the University of Navarra, has been approved as an "orphan drug" by the European Medicines Agency (EMEA). The biotechnology company Digna Biotech can now proceed to the clinical trial phase of peptide p144 in the treatment of localized and systemic sclerodermia.

The function of Digna Biotech is the development of the intellectual property of CIMA through the pre-clinical, clinical and commercial phases.

The approval, granted by the Committee for Orphan Medicinal Products (COMP), facilitiates the development of the patent that Digna Biotech will now undertake. The company will have open access to the European Medicines Agency"s scientific resources and advisory services, receive a reduction in the fees charged as part of the commercial authorisation process, and will enjoy 10 years exclusive market availability when the developed product has been granted the Agency"s formal approval.

Dr. Pablo Ortiz, the managing director of Digna Biotech, explained that the approval of the patent "will speed up the clinical development of the project and reduce our costs. We are particularly happy with the decision because, for the first time in Spain, the EMEA has approved an orphan drug for two forms of the same disease, a fact which underscores the consistency and quality of the data presented by CIMA to the Agency and the importance of this patent for society in general".

Dr. Juan Ruiz, the medical director of Digna Biotech, commented on the committment of those involved in the project "to improve the treatment of both localized and systemic sclerodermia. Although the incidence of sclerodermia is not very high, it is a chronic condition and can greatly diminish the sufferer´s quality of life". Dr. Ruiz also thanked the Carlos III Institute, the Universidad Autonoma in Barcelona and Idifarma, a pharmaceutical company in Navarra, for their work and cooperation in the successful EMEA application.

Digna Biotech is already at work on the application that will be submitted to the Food and Drug Administration (FDA) in order to obtain orphan drug approval in the United States. The company is also in discussions with other companies in the biotechnology sector who may be interested in participating in the development of the product or its launch on the market.

Sclerodermia and p144: treatment in a topical cream

Sclerodermia is a chronic disease, characterised by fibrosis of the skin, blood vessels and internal organs such as the lungs. It is thought that one of the key factors in the onset of the disease may be an excess of TGF Beta-1 (Transforming Growth Factor Beta-1). There is no known cure for sclerodermia, and the efficacy of existing treatments is uncertain.

Sclerodermia is a rare disease: it affects between 37,000 and 72,000 people in the European Union; that is, the incidence of the condition is between 0.82 and 1.58 cases in 10,000 people. The fact that sclerodermia is not a very common disease has made research into new and better forms of treatment more difficult.

Peptide p144 is a TGF Beta-1 inhibitor. It is now thought that TGF Beta-1 inhibitors have a key role to play in fighting fibrosis, and will thus improve the condition of those who suffer from sclerordermia. Digna Biotech is developing p144 in the form of a topical cream in order to treat the cutaneous lesions that appear in both localized and systemic sclerodermia. The findings from pre-clinical tests suggest that p144 cream is an effective form of treatment, and toxicological studies have been set up as part of the preparation for the clinical trial phase which is due to begin during the first half of 2006.

At the same time, Digna Biotech is also examining other potential uses of p144, in the treatment of pulmonary fibrosis, cheloid scars, fibrosis caused by the introduction of a foreign body, and macular degeneration of the retina.