ProQR Therapeutics announced that its sepofarsen drug candidate, also known as QR-110, has received access to a program for promising medicines, according to a press release.
Sepofarsen was granted access into the European Medicines Agency’s Priority Medicines (PRIME) program, which offers a pathway of accelerated evaluation and approval for medicines that may provide a therapeutic advantage over existing treatments or that are for indications that currently have no treatment options, according to the release. Sepofarsen is under development as a treatment for Leber’s congenital amaurosis 10.
“There are thousands of rare inherited diseases that currently have no cure or treatment options. Our goal at ProQR is to directly correct the cause of genetic conditions, including LCA10, using our RNA editing platform,” ProQR CEO Daniel A. de Boer, told Healio.com/OSN. “There are many possible advantages and benefits of targeting the RNA over other editing platforms, including gene replacement and gene editing. RNA editing drugs don’t permanently alter a person's genetic code, reducing the chance of permanent off-target effects, and their administration (intravitreal) is far less cumbersome than other types of therapy that involve complex sub-retinal surgeries.”
de Boer said the PRIME eligibility designation of sepofarsen means that it has the potential to reach patients sooner.
According to the release, as of June, only 20% of ophthalmology applications have been granted PRIME access.
“There is currently no treatment for the majority of patients with LCA10, and we are aiming to bring a safe and effective treatment to patients using our RNA editing therapy in order to improve their quality of life,” de Boer said. “We believe that our RNA editing therapies have the potential to directly benefit patients and their quality of life.” – by Earl Holland Jr.
Editor’s note: This article has been updated to include quotes from ProQR CEO Daniel A. de Boer.