The pharmaceutical company Protalix Biotherapeutics, Inc. announced that a drug currently in development called PRX-102 has been granted orphan drug status by the European Commission. The treatment is being developed to treat Fabry disease, a very rare inherited genetic disorder.
In Fabry disease, a genetic mutation causes a deficiency of an enzyme called alpha galactosidase A. The deficiency causes a buildup of the a glycolipid called Gb3 within various areas of the body, such as the blood vessels, tissues, and other organs. The buildup eventually keeps the systems from functioning properly. People with the disease begin to experience symptoms in early childhood; these symptoms can include pain, sometimes localized and sometimes the entire body. Other symptoms include reduced kidney function (and eventually kidney failure, which is responsible for many deaths), increased risk of heart disease, high blood pressure, skin papules, inability to sweat, and the clouding of the corneas. To learn more about this disease, click here.
Currently, there are only a few treatments available for Fabry disease. While they can be effective in inhibiting the progression of the disease, and can sometimes even reverse some of the symptoms, the treatments are extremely costly at around $200,000 per year. PRX-102 is being tested in combination with the earlier treatments. If the orphan drug status remains until the drug’s approval, PRX-102 will have nearly ten years of exclusivity. The orphan drug status provides tax reductions and gives the company rights to be the sole developer of the drug.
The orphan drug status is intended to be an incentive for companies to research and develop treatments for rare diseases. In the past, these treatments would have had little marketable value for the company simply because the conditions that would be treated are so rare, and therefore demand for the treatments would be low. This also explains the extreme expense of treatments for rare diseases like Fabry.