Endogena’s retinitis pigmentosa treatment granted Orphan Drug Designation by FDA
pharmafile | May 12, 2021 | News story | Research and Development | DFA, Endogena, Orphan Drug Designation
Endogena Therapeutics Inc has been granted Orphan Drug Designation for its EA-2353 ophthalmic suspension targeting retinitis pigmentosa by the FDA.
Retinitis pigmentosa is a rare eye condition that causes slow and progressive loss of vision and is the leading cause of inherited blindness. Clinical trials are anticipated to start by the end of the year.
The treatment works by selective regulation of the endogenous adult stem and progenitor cells for controlled tissue repair.
The concept is driven by cutting-edge knowledge of molecular pathways that regulate retinal stem cells, which generate photoreceptors during development and are dormant in the adult mammalian eye.
Orphan status provides benefits including market exclusivity once approved, exemption of FDA application fees and tax credits for qualified clinical trials.
The FDA’s Office of Orphan Drug Products provides orphan status to drugs and biologics which are defined as those intended for the treatment, prevention, or diagnosis of a rare disease or condition, which is one that affects less than 200,000 people in the US or meets cost recovery provisions of the act.
CEO of Endogena, Matthias Steger said: “Receiving orphan drug designation for EA-2353 is an important milestone to emerge from our innovative endogenous regenerative platform and confirms its potential for a medical treatment paradigm shift for degenerative diseases, in this case for retinitis pigmentosa patients who so desperately need it.
“We have completed our IND enabling studies and, subject to IND approval, we anticipate enrolling our first patients into the clinical trial by the end of this year.”
Mark Pennesi, PhD, the Kenneth C Swan Associate Professor of Ophthalmology at the Casey Eye Institute in Portland, who will lead the Phase I/IIa studies, said: “I’m eager to begin clinical proof-of-concept studies to establish whether Endogena’s approach could offer a new treatment paradigm in retinitis pigmentosa.
“This novel method of tissue repair holds great promise for patients with this devastating degenerative eye condition.”
Lilly Subbotin
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