The U.S. FDA approves a patent for an "orphan drug" from the CIMA of the University of Navarra
Digna Biotech and the ISDIN laboratory will co-develop a cream for treating two indications of sclerodermy, a skin disease
The Food and Drive Administration of the U.S.A. has given the designation of "orphan drug" to a drug based on one of the 18 patents obtained by the Centre for Applied Medical Research (CIMA) of the University of Navarra. This is the peptide p144, which belongs to Digna Biotech, the company which developed at the preclinical, clinical and commercial stages the intellectual property (patents) belonging to the CIMA.
The FDA provides the designation of orphan drugs to those products which demonstrate a therapeutic potential for the treatment of rare or uncommon diseases, such as in the case of sclerodermy, a disease of the skin.
This approval as an orphan drug will provide advantages for the preclinical and clinical development of p144: free assessment by the FDA for the design of the clinical phase, and economic aid for performing the necessary clinical trials; exemption from registration fees and advising during the preparation of the registration dossier; and commercial exclusivity for 7 years once the drug has been approved by the American agency.
Digna Biotech has signed an agreement with the pharmaceutical laboratory ISDIN, a leader in the Spanish dermatology market. Thanks to this alliance, ISDIN will finance 50% of the development costs for p144 in Europe and 100% in the U.S., will own the register of the pharmaceutical product throughout the world, and will have exclusive worldwide rights for commercialization for the treatment of local or systemic sclerodermy, once the health authorities have approved its use. ISDIN will also have the first option of producing products for all indications related to the skin (keloids, burns, skin cancer, etc.) which Digna Biotech develops based on p144.
This is one of the first designations as an orphan drug which the FDA has conceded to a molecule which was patented and developed entirely in Spain. The approval obtained in the U.S. is in addition to that obtained from the European Agency for the Evaluation of Medicinal Products (EMEA) in 2005. Up to now, p144 has shown its effectiveness in the animal model, and has passed satisfactorily the first toxicological analyses. It is hoped that the product can begin the clinical phase at the end of this year, and that it will obtain authorization for commercialization in 2009. Both companies believe that the sales potential for this medicine should not be less than 200 million euros per year by 2014.
Digna Biotech received financing from the Center for the Development of Industrial Technology (CDTI), as a result of obtaining a Concerted Industrial Research Project grant, which finances projects presented by businesses in collaboration with universities or research and innovation centers.
Sclerodermy is a chronic skin disease which is characterized by fibrosis in the skin, blood vessels and internal organs such as the lungs. It is believed that an excess of TGF-Beta 1 (Transformational Growth Factor) may be one of the key factors in the development of this pathology. At the current time, there are no therapies which can cure the disease, only symptomatic treatments of dubious effectiveness.
The peptide p144 is an inhibitor of TGF- β1. It is believed that TGF- β1 inhibitors can play a key role in the inhibition of the fibrosis, and therefore in reducing the severity of the sclerodermy. p144 is being developed in cream form for the treatment of the cutaneous lesions caused by systemic and localized sclerodermy. Preclinical data indicate that p144 will be effective in treating this disease, and toxicological studies are currently underway. Once these studies are concluded, the clinical phase should begin at the end of 2006.