According to an article from kait8.com, the pharmaceutical company Polaryx announced that its experimental drug PLX-200 has received Orphan Drug Designation from the European Medicines Agency (EMA) for the treatment of late infantile neuronal ceroid lipofuscinosis (NCL) as well as other forms of NCL, which is also known as Batten disease.
Batten disease is a disease of the nervous system that appears in early childhood and is ultimately fatal. Symptoms normally appear between 5 and 10 years of age. Batten disease is linked to mutations in the CLN3 gene. Early symptoms include problems with vision or seizures. There may also be fairly subtle changes in personality, clumsiness, slow learning, repetitive speech, and spine curvature. Ultimately, as the disease progresses, patients suffer severe mental impairment, loss of sight, motor skills, and speech, and more severe seizures. There is currently no cure or treatment that can prevent disease progression and death, although one available treatment, Brineura, can slow the loss of walking ability in a specific form of the condition. To learn more about Batten disease, click here.
Both the EMA and the US Food and Drug Administration (FDA) have their own Orphan Drug Designation (ODD) specifications in place. PLX-200 was granted the designation by the FDA last year. In Europe, a drug qualifies for ODD if it is intended for a severe disease, such as one that is known to be life-threatening and/or could lead to long-term debilitation. ODD is meant for rare diseases specifically, however, so the condition may not affect more than 5 in 10,000 people. In addition, the treatment must treat a currently unmet medical need or represent a major improvement in treatment for those affected by the disease. The EMA’s ODD also offers some fee reductions and a ten year period of market exclusivity if the drug gains approval.
