According to a story from BioSpace, the gene therapy company Reflection Biotechnologies Ltd. recently announced that the US Food and Drug Administration (FDA) has given Orphan Drug designation to its investigational gene therapy RBIO-101. This therapy is in development for the treatment of Bietti’s crystalline dystrophy. Reflection Biotechnologies is focused on the creation of new therapies for rare diseases.
About Bietti’s Crystalline Dystrophy
Bietti’s crystalline dystrophy is a rare genetic eye disease which appears to be more common people of Asian descent. The disease is linked to mutations that affect the CYP4V2 gene. Symptoms of Bietti’s crystalline dystrophy include the formation of crystals in the cornea, crystals in lymphocytes (a type of white blood cell), reflective, yellow deposits that appear on the retina, and the progressive atrophy choroid, choroicapillaries, and retina. This results in restriction of the visual field and night blindness. Vision problems usually become noticeable in the teenage years or early twenties. Disease progression does not always occur at the same rate in each eye. This is a very rare disease that has not received much attention or extensive research. As a result, there are currently no available treatments for the disease. To learn more about Bietti’s crystalline dystrophy, click here.
About Orphan Drug Designation
Orphan Drug designation is typically reserved for therapies that are meant to treat rare diseases, which is legally defined as any condition which affects less than 200,000 people in the US. In addition, the therapy must either display the potential to offer significant advantages over currently available treatments in either effectiveness or safety. Alternatively, drugs that could potentially satisfy a currently unmet medical need (Bietti’s crystalline dystrophy would certainly apply) also may be eligible. The designation confers certain benefits to the recipient company, such as the waiving of certain fees, tax breaks, and a seven year period of market exclusivity if the drug gains approval.
This news is a significant milestone for patients with the disease. Currently, dealing with Bietti’s crystalline dystrophy meant having to accept a severe decline in vision ability over the course of one’s life; most patients are legally blind by the time they reach age forty or fifty. RBIO-101 could give these patients a new reason to hope.