Here's a look at some of the latest news regarding ophthalmic drugs and therapies from the past week.
- Dompé announced the Committee for Human Medicinal Products (CHMP) of EMA (European Medicines Agency) has released a positive opinion, recommending the marketing authorization for Oxervate® (cenegermin eye drops), for the treatment of adult patients with moderate or severe neurotrophic keratitis. If confirmed by EMA’s Committee for Orphan Medicinal Products (COMP) and approved by the European Commission, Oxervate® would reportedly be the first biotechnology orphan drug in the world authorized for this indication.
- ProQR Therapeutics N.V. has received Fast Track designation from the U.S. FDA for QR-110 -- the lead molecule in its ophthalmology pipeline being developed for the treatment of patients with Leber’s Congenital Amaurosis Type 10 (LCA 10). QR-110 is reportedly a novel investigational RNA oligonucleotide targeting LCA 10 due to the p.Cys998X mutation, which is one of the most prevalent forms of gene-related blindness in children.
- Spark Therapeutics announced the completion of the rolling submission of a Biologics License Application (BLA) with the U.S. FDA for voretigene neparvovec — an investigational, one-time gene therapy for the treatment of patients with vision loss due to confirmed biallelic RPE65 mutation-associated retinal disease. The BLA submission reportedly includes data from three clinical trials that enrolled 41 participants with RPE65-mediated IRD, including the first randomized, controlled Phase 3 trial for a gene therapy for a genetic disease.
- ProtoKinetix has completed the first year of retinal replacement therapy trials on animals using its family of hyper-stable, potent glycopeptides (AAGP™) , and the results are reportedly encouraging enough to proceed to a second phase of testing.
- Quethera recently presented strong efficacy data at the 2017 Association for Research in Vision and Ophthalmology (ARVO) conference showing its gene therapy QTA020V resulted in a 61% reduction in retinal ganglion cell (RGC) loss in a widely used experimental model of glaucoma.
- REGENXBIO Inc. announced the first patient was dosed in a phase I clinical trial evaluating RGX-314 for patients with wet age-related macular degeneration (wet AMD). This multi-center, open-label, multiple-cohort, dose-escalation clinical trial will reportedly assess the safety and tolerability of RGX-314 as a one-time therapy for patients with previously treated wet AMD.
- Lamellar Biomedical has recruited the first patients for a clinical study assessing LAMELLEYE for the treatment of moderate to severe Dry Eye Disease (DED). The new study will reportedly assess the clinical utility of LAMELLEYE and compare its performance with Optive Plus, a leading over-the-counter eye-drop for patients with DED.
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