4D110 has just received Orphan Drug Designation for the treatment of Choroideremia (CHM). The phase 1 trial of the drug is expected to start in 2019.
What is Chorioderemia?
Chorioderemia is a progressive disease that eventually causes blindness. The condition starts with a loss of night vision. Then patients begin to lose their peripheral vision and finally their visual acuity. It’s caused by a mutation in the REP-1 gene. 4D110 works to deliver healthy expressions of the gene to the retina. The drug is administered with a shot directly into the eye. Unlike other types of gene therapy being investigated to treat this condition, 4D110 could be used when the disease is in any stage. Additionally, it works in the entire retina, as opposed to just one part of it like some treatments.
4D Molecular Therapeutics
4D Molecular Therapeutics is the company conducting the analysis of this drug. They have created an ongoing natural history study which currently has over 50 patients enrolled. This study will hopefully accelerate enrollment for the clinical trial when the time comes. Additionally, it will help researchers by providing a comparison of patient characteristics before and after receiving the treatment.
What Does the Designation Mean?
Orphan Drug Designation for 4D110 will aid 4D Molecular Therapeutics in their process of trying to obtain FDA approval. It exempts 4D Molecular Therapeutics from the FDA application fees, it gives them marketing exclusivity for the drug (following approval), and exempts the company from tax credits for clinical trials.
This designation is progress toward getting Choroideremia patients better treatment!
You can read more about this new treatment and its progression toward clinical trials here.