• Category: Pipeline
Dompé receives FDA priority voucher for NAION intranasal therapy
Pipeline

Dompé receives FDA priority voucher for NAION intranasal therapy

New expedited pilot program cuts the standard application review time for investigational candidates from 10 to 12 months to just 1 to 2 months.
Nanoscope's optogenetic therapy demonstrates visual improvements for Stargardt
Pipeline

Nanoscope's optogenetic therapy demonstrates visual improvements for Stargardt

Findings from phase 2 STARLIGHT trial support the safety and efficacy of MCO-010 in Stargardt disease.
FDA grants IDE approval for Avisi's glaucoma treatment device
Pipeline

FDA grants IDE approval for Avisi's glaucoma treatment device

IDE approval enables initiation of the open-label SAPPHIRE to evaluate the VisiPlate, an aqueous shunt designed to lower IOP among OAG patients.
FDA grants Fast Track designation to Epion's EpiSmart epithelium-on CXL system
Pipeline

FDA grants Fast Track designation to Epion's EpiSmart epithelium-on CXL system

Status supports an expedited pathway toward a potential regulatory submission and approval for the minimally-invasive keratoconus treatment.
SightGlass unveils 18-month data on DOT spectacle lenses for myopia
Pipeline

SightGlass unveils 18-month data on DOT spectacle lenses for myopia

Latest findings follow 12-month data reported at ARVO; results show over 50% of pediatric patients had no clinically meaningful disease progression.
Oculis advances neuro-ophthalmic asset after positive FDA feedback
Pipeline

Oculis advances neuro-ophthalmic asset after positive FDA feedback

Privosegtor (OCS-05) gets the green light to be studied in registrational trials beginning this quarter for acute optic neuritis and NAION in 2026.
FDA grants Orphan Drug designation to AAVantgarde's Stargardt gene therapy
Pipeline

FDA grants Orphan Drug designation to AAVantgarde's Stargardt gene therapy

Regulatory milestone is second designation for AAVB-039; includes tax credits for qualified clinical testing and, if approved, seven years of U.S. market exclusivity.
Opus Genetics reports positive pediatric data from LCA5 gene therapy study
Pipeline

Opus Genetics reports positive pediatric data from LCA5 gene therapy study

Ongoing phase 1/2 trial on OPGx-LCA5 indicates large gains in cone-mediated vision, with durable responses observed out to 18 months among adult patients.
FDA grants Breakthrough Therapy Designation to Nacuity's RP tablets
Pipeline

FDA grants Breakthrough Therapy Designation to Nacuity's RP tablets

Third designation for NPI-001 follows recent positive 24-month data showing a 50% photoreceptor reduction after 2 years of daily dosing.
Kala's phase 2b PCED trial fails to meet primary goal
Pipeline

Kala's phase 2b PCED trial fails to meet primary goal

Company plans to cease development of lead asset KPI-012 as the CHASE study also falls short of achieving statistical significance for secondary endpoints.
FDA grants priority review for Chiesi's LHON therapeutic
Pipeline

FDA grants priority review for Chiesi's LHON therapeutic

Approved by the EMA as Raxone, idebenone could become the first and only clinically proven treatment to address the underlying cause of LHON.
Opus Genetics doses first patient in LYNX-3 phentolamine 0.75% study
Pipeline

Opus Genetics doses first patient in LYNX-3 phentolamine 0.75% study

Third phase 3 trial is evaluating the PF-based eye drop for chronic night driving impairment in keratorefractive patients with reduced mesopic vision.
FDA grants RMAT designation to Nanoscope for Stargardt gene therapy
Pipeline

FDA grants RMAT designation to Nanoscope for Stargardt gene therapy

MCO-010 optogenetic therapeutic has potential to become first-ever treatment for Stargardt; phase 3 trial expected to kick off in Q4 2025.
Nicox reports positive phase 3 data on NCX 470 glaucoma eye drop
Pipeline

Nicox reports positive phase 3 data on NCX 470 glaucoma eye drop

Topline results from the Denali trial support a future NDA submission for the nitric oxide-donating bimatoprost formulation to lower IOP.
Outlook Therapeutics receives second FDA CRL over wet AMD BLA
Pipeline

Outlook Therapeutics receives second FDA CRL over wet AMD BLA

Agency rejects submission of bevacizumab-vikg for a second time, citing a single issue with the application: a lack of effectiveness data.
FDA extends review period for EYLEA HD expanded labeling
Pipeline

FDA extends review period for EYLEA HD expanded labeling

Delay follows a recent site inspection; proposed expansion includes a new indication, updated dosings for current indications, and a pre-filled syringe version.
FDA grants RMAT designation to VeonGen's Stargardt therapy
Pipeline

FDA grants RMAT designation to VeonGen's Stargardt therapy

Status marks third designation for the VG801, intended to treat patients with biallelic
jCyte doses first patients in RP allogeneic cell therapy trial
Pipeline

jCyte doses first patients in RP allogeneic cell therapy trial

Phase 2b JC02-88 trial to evaluate a single IVT injection of jCell (famzeretcel), an agnostic cell therapy with the potential to restore vision in all RP genetic subtypes.
Avista and Forge Biologics partner to advance XLRS gene therapy
Pipeline

Avista and Forge Biologics partner to advance XLRS gene therapy

Collaboration leverages Forge’s in-house AAV development and manufacturing services for AVST-101, an AAV-based IVT gene therapy.
FDA green lights Myra Vision's IDE study for glaucoma
Pipeline

FDA green lights Myra Vision's IDE study for glaucoma

IDE approval enables company to initiate the open-label ADAPT study on its proprietary Calibreye TGT Surgical System.