Abstract
In this period, there was U.S. federal activity on guidance for compounding sterile products, broadening enrollment in clinical trials, unapproved stem cell clinics, generic pharmaceuticals, therapeutic use of cannabinoids, and the use of federal resources for fetal cell research. Many firms had progress in the development of novel therapeutics for eye disease, including new targets in glaucoma and wet age-related macular degeneration (AMD) therapy, another trial of low-dose atropine to prevent pediatric myopia, and a treatment for persistent corneal epithelial defects.
Ophthalmic Pharmaceuticals and Biologics
Acucela received orphan designation from the European Medicines Agency (EMA) for its emixustat for the treatment of Stargardt disease (June 2019).
Aerpio commenced enrollment in a study of its AKB-9778 for topical treatment of glaucoma. This is a phase 1 study in normal volunteers (June 2019).
Aldreyra announced results from its SOLACE phase 3 trial of reproxalap in the treatment of anterior uveitis (June 2019).
Alimera Sciences received recommendation for reimbursement from the U.K.'s National Institute for Health and Care Excellence (NICE) for its Iluvien® (fluocinolone acetonide intravitreal implant) for the prevention of relapse in recurrent noninfectious uveitis affecting the posterior segment (June 2019).
Allegro announced results for a phase 2 study of its Luminate™ (risuteganib) in the treatment of patients with intermediate dry AMD (June 2019).
EyeGate has been cleared by the U.S. FDA to initiate its photorefractive keratectomy pivotal study of its crosslinked thiolated carboxymethyl hyaluronic acid, a modified form of the natural polymer hyaluronic acid (June 2019). *
Eyenovia commenced enrollment in its phase 3 CHAPERONE study of atropine (“MicroPine”) for the prevention of pediatric myopia (June 2019).
Noveome Biotherapeutics commenced dosing in its phase 2 open label, multicenter clinical trial evaluating topical ocular delivery of ST266 for the treatment of persistent corneal epithelial defects (June 2019).
Ocular Therapeutix received an additional indication from the U.S. FDA for its Dextenxa™ intracannicular dexamethasone—the treatment of ocular inflammation after ophthalmic surgery (June 2019).
Oxurion announced results for its THR-317 for the treatment of diabetic macular edema (June 2019).
Premark Pharma licensed worldwide rights to PMP2207 for ophthalmic indications, and seeking the indication of blepharitis (June 2019).
Ribomic announced top-line results from its SUSHI study, a phase 1/2a single ascending dose clinical study of RBM-007, anti-FGF2 aptamer, in 9 subjects with wet AMD (June 2019).
Santen presented results of its 2 real-world evidence studies, VISIONARY and PERSPECTIVE of topical preservative-free tafluprost (0.0015%)/timolol (0.5%) fixed-dose combination and cyclosporine, respectively (June 2019).
Gene Therapy
RegenxBio completed dosing in all 5 cohorts in the phase 1/2a clinical trial of RGX-314 for the treatment of wet AMD (May 2019).
Medical Devices
Sight Sciences commenced enrollment in OLYMPIA, a U.S. study of the safety and efficacy of its TearCare® (which delivers regulated localized heat to the eyelids) for patients with dry eye disease (June 2019).
Regulatory, Government, and Pharmaceutical Industry
AbbVie, Inc., agreed to buy Allergan PLC (June 2019).
The U.S. FDA:
○ Held a public meeting to discuss therapeutic uses of tetrahydrocannabinol and cannabidiol (June 2019).
○ Released a draft guidance on “Enhancing the Diversity of Clinical Trial Populations” in which they suggest that sponsors should consider loosening their exclusion criteria to allow more diverse patient populations—especially when recruiting for later stage trials (June 2019). †
○ Suspended clinical trials of fecal matter transplants due to safety reasons (June 2019).
Seven former FDA commissioners, hailing from both sides of the political aisle and spanning many administrations, recently recommended that the “FDA should be an independent federal agency reporting to the President.” 1 Adashi et al. explored the utility, desirability, and feasibility of restructuring the FDA charter with political insulation and administrative streamlining (May 2019). 2
U.S. Congressman David N. Cicilline (D-Rhode Island, RI-01) sponsored HR 965, the “Creating and Restoring Equal Access to Equivalent Samples” bill to prohibit pharmaceutical and biologic companies from engaging in anticompetitive conduct that blocks lower cost generic drugs from entering the market. An identical version of the bill was introduced in the Senate by Senators Patrick Leahy (D-Vermont), S.340. (February 2019).
A U.S. federal judge ruled in favor of the FDA in a lawsuit against a Florida-based stem cell company whose treatments have blinded at least 4 patients (June 2019).
U.S. National Institutes of Health (NIH) director Francis Collins, MD stated he will only accept speaking invitations from panels from which are inclusive of scientists of all backgrounds (June 2019).
The U.S. executive branch halted fetal tissue research by government scientists and those seeking funding from the U.S. NIH (June 2019). 3
The United States Pharmacopeia (USP) released new and revised standards to help ensure the quality of compounded medicine relevant to ophthalmic medications, including chapters on compounding nonsterile medicines (USP <795> and <797>, June 2019).
Footnotes
Author Disclosure Statement
The author consults for numerous ophthalmic pharmaceutical and medical device firms.