Abstract
In this period, advances were reported in gene therapy and genome editing trials for achromatopsia and Leber's congenital amaurosis. In drug delivery, novel intraocular systems are being evaluated for the therapy of glaucoma and retinal disease. In dry eye, several novel therapeutics are in various stages of clinical development. A medication was approved in Europe for the treatment of retinopathy of prematurity. Among much governmental/regulatory activity, an action that might be of interest to the ocular community is by the FDA, who is concerned with the safety of systemic exposure after the use of topical nonprescription sunscreens.
Ophthalmic Pharmaceuticals and Biologics
Aerie commenced dosing patients with wet age-related macular degeneration (AMD) or diabetic macular edema (DME) with an intravitreal delivery system of AR-13503 (August 2019).
Aldreyra completed enrollment in its SOLACE phase 3 trial of its topical reproxalap in the treatment of noninfectious anterior uveitis (May 2019).
Alimera received approval from the Australian Department of Health for its Iluvein® (fluocinolone acetonide intravitreal implant) for the treatment of DME (August 2019).
Alkahest, Inc., announced results from two phase 2 studies of AKST4290 (formerly ALK4290), an orally administered CCR3 inhibitor that blocks the action of eotaxin for the treatment of wet AMD (August 2019).
Kala received a complete response letter from the FDA denying approval for the new drug application for its KPI-121 (loteprednol etabonate) 0.25% for the treatment of dry eye disease (August 2019).
Kodiak announced interim results from an ongoing phase 1b study of its KSI-301, an anti-vasoactive endothelial growth factor (VEGF) antibody biopolymer conjugate for the treatment of wet AMD (July 2019).
Oxurion reported results on THR-317, a humanized antibody against placental growth factor, in a study of combination use with ranibizumab in patients with DME (August 2019).
Oyster point commenced enrollment in a phase 3 study (ONSET-2) of OC-01, a nasal spray for the treatment of dry eye disease (July 2019).
Polyactiva completed enrollment in a phase 1 study of its intraocular drug delivery system of latanoprost (July 2019).
Regeneron received FDA approval for Eylea® (aflibercept) in a prefilled syringe (August 2019).
Roche received approval for treatment of retinopathy of prematurity for Lucentis® (ranibizumab) from the European Medicines Association Committee for Medicinal Products for Human Use (August 2019).
SilkTech Biopharmaceuticals completed enrollment in a phase 2b clinical trial of SDP-4 for the treatment of dry eye disease (August 2019).
Gene Therapy
Allergan and Editas Medicine initiated an open-label phase 1/2 clinical trial of AGN-151587 (Brilliance study), an experimental CRISPR-based genome editing medicine for the treatment of Leber congenital amaurosis 10 (July 2019).
Applied Genetic Technologies Corporation completed enrollment in the third group of a dose escalation portion of a phase 1/2 trial of its subretinal injection gene therapy achromatopsia treatment candidate (July 2019).
CRISPR Therapeutics treated the first patient with a CRISPR gene editing therapy (sickle cell anemia, July 2019).
Novartis and the FDA announced that there was a data accuracy issue with nonclinical data provided in support of its recently approved gene therapy for spinal muscular atrophy, Zolgensma™ (onasemnogene abeparvovec-xioi, August 2019).
Regulatory, Government, and Pharmaceutical Industry
European Biopharmaceutical Enterprises and the European Federation of Pharmaceutical Industries and Associations issued a document on the industry perspective of clinical requirements for prefilled single-use integral drug-device combination products (July 2019).
Samsung Bioepis received US FDA approval for its Hadima™ (adalimumab-bwwd), a biosimilar referencing HUMIRA® (adalimumab), for the treatment of rheumatoid arthritis and other indications (July 2019).
Sipp and Sleeboom-Faulkner presented considerations for downgrading of regulation in regenerative medicine (August 2019). 1
The Canadian government announced final regulations to reduce patented drug prices (August 2019).
The US FDA is issuing a proposed rule for nonprescription over-the-counter sunscreen drug products related to a concern over systemic exposure after topical use.2,3
Footnotes
Author Disclosure Statement
The author consults for numerous ophthalmic pharmaceutical and medical device firms.