The U.S. Food and Drug Administration (FDA) has designated one of the 18 patents of the University of Navarra's research center Médica Aplicada (CIMA) as an orphan drug. This is the p144 peptide from Digna Biotech, business which is developing at the preclinical, clinical and commercial levels the intellectual property (patents) for CIMA.

The FDA grants orphan drug designation to products that demonstrate potential therapeutic value in the treatment of rare or uncommon diseases, such as in this case scleroderma, a skin disease.

The approval entails advantages for the preclinical and clinical development of p144: free advice from the FDA for the design of the clinical phase and financial aid economic to carry out the necessary clinical trials; exemption of the fees of the registration and advice in the preparation of the registration dossier; and commercial exclusivity for 7 years, once C the product has been approved by the US agency.

Sales forecast: 200 million euros/year in 2014

Digna Biotech has just signed an agreement agreement with the pharmaceutical laboratory ISDIN, leader in the Spanish dermatological market. Thanks to this alliance, ISDIN will finance 50% of the cost of development of p144 in Europe and 100% in the USA, will assume the registration of the pharmaceutical specialization program worldwide and will own the worldwide and exclusive licence for its commercialization in local or systemic scleroderma, once C by the health authorities. ISDIN will have a first choice in all skin-related indications (keloids, burns, skin cancer, etc.) that Digna Biotech obtains for its p144.

This is one of the first orphan drug designations granted by the FDA to a patented molecule developed entirely in Spain. The approval obtained now in the U.S. joins the one already obtained in 2005 from the European Medicines Agency (EMEA). So far, p144 has demonstrated its efficacy in the animal model and has successfully passed the first toxicological tests. The product is expected to enter the clinical phase by the end of this year and to achieve marketing authorization in 2009. Both companies believe that the potential of this product should not be less than 200 million euros/year from 2014 onwards.

Digna Biotech counts for this project with the financing of the Center for Industrial Technological development (CDTI), thanks to the obtaining of a project of research Industrial Concerted that finances projects presented by companies in partnership with universities and / or centers of research and innovation.

Scleroderma and p144

Scleroderma is a chronic disease characterized by fibrosis of the skin, blood vessels and internal organs such as the lung. It is believed that the excess of TGF-Beta 1 (Transforming Growth Factor) may be one of the factors core topic in the development of this pathology. For now, there is no therapy to cure the disease, only symptomatic treatments of dubious efficacy.

The p144 peptide is an inhibitor of TGF- β1. It is believed that TGF- β1 inhibitors may develop a role core topic in inhibiting fibrosis and thus improving the progression of scleroderma. The p144 is being developed in cream form for the treatment of systemic and localized scleroderma skin lesion. There are preclinical data that support its efficacy in this pathology, and the toxicological programs of study are being carried out to initiate the clinical phase at the end of 2006.