Gene therapy company 4D Molecular Therapeutics (4DMT) announced that the FDA has granted orphan drug designation to 4D-110 for the treatment of choroideremia.
Choroideremia (CHM) is an X-linked monogenic recessive disease (caused by mutation of the REP-1 gene) starting with reduced night vision and progressive loss of peripheral vision and eventually leading to loss of visual acuity and blindness.
“Our product for CHM patients, 4D-110, will be administered by intravitreal injection, and is designed to result in widespread expression of the REP-1 protein within cells in the retina,” Peter Francis, SVP Clinical, Translational R&D Program Leader, Retina Therapeutic Area, said in a company news release. “In contrast to other AAV gene therapy agents for CHM patients, which are administered by subretinal surgical injection, 4D-110 is designed to treat the entire retina, and to treat patients with all stages of the disease.”
The company has enrolled more than 50 CHM patients in an ongoing natural history study. This study is expected to support the rapid enrollment of the company’s planned clinical studies and to provide baseline characteristics for pre- and post-treatment analysis. 4DMT expects to initiate the phase 1 trial in 2019.
The FDA’s Office of Orphan Drug Products grants orphan drug designation to support the development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the United States. Orphan drug designation provides to 4D Molecular Therapeutics certain benefits, including market exclusivity upon regulatory approval, if received, exemption of FDA application fees and tax credits for qualified clinical trials.