Reflection Biotechnologies Limited ("ReflectionBio") announced on 8/28/18 that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ReflectionBio's RBIO-101 program (AAV.CYP4V2), an AAV-based gene therapy product for treating Bietti's Crystalline Dystrophy (BCD).
"Receiving orphan drug designation from the FDA is a milestone. It validates our R&D progress and provides us with various incentives in further developing BCD gene therapy. This brings hope to BCD patients and their families because BCD is a devastating blinding disease for which currently there is no approved treatment. BCD is estimated to affect more than 100,000 patients worldwide. As the next step, we plan to advance BCD gene therapy into human clinical trial," said Richard R. Yang, Founder and CEO of ReflectionBio.
“As a rare disease patient, I am proud to be driving R&D efforts for BCD gene therapy together with our collaborator, Dr. Stephen H. Tsang of Columbia University. The RBIO-101 preclinical data from Dr. Tsang’s lab support the clinical translation for patients with BCD. I am grateful for Dr. Tsang, his team and Columbia University for partnering with us, the patients, in fighting rare diseases,” Yang added.
“One out of every 10 people lives with at least one of more than 7,000 rare diseases. Many rare diseases cause premature death or life-long disabilities. Sadly, more than 90% of rare diseases do not have any approved treatment available,” Yang said.
BCD is a rare retinal degeneration. BCD was first identified by Italian ophthalmologist Dr. G.B. Bietti in 1937. In BCD, progressive atrophy and degeneration of the retinal pigment epithelium (RPE) lead to symptoms similar to those of other forms of retinal degeneration, including retinitis pigmentosa (RP). Yellow-white crystals are seen in the retina of BCD patients, which may disappear in advanced stages. Onset usually occurs between early teenage years and the third decade of life, leading to legal blindness by the 3rd to 6th decade of life. Since it is inherited in an autosomal recessive pattern, some BCD patients may have no family history. Based on the BCD incidence rate (1:67,000)1, BCD is estimated to affect more than 100,000 patients globally. BCD has been reported in patients from Asia, Europe, North and South America, the Middle East, and Africa. It is more common in East Asia, especially among Chinese and Japanese populations. Researchers suggested that BCD may be underdiagnosed because its symptoms are similar to those of other eye disorders that progressively damage the retina.
