Eyes on New Product Development

Abstract

In this reporting period, there was exceptional activity. Several new products were approved or cleared by the U.S. Food and Drug Administration (FDA), including a new topical antihistamine, a device that increases tear flow by electrical stimulation, and the first treatment for giant cell arteritis. In glaucoma, phase 3 trial results were presented for a new fixed-dose combination product, and a firm started to develop a novel pharmacological therapy. Treatment of inflammation in both anterior and posterior segments continues to be a target, from new preclinical firms to submission of a New Drug Application (NDA) to the U.S. FDA for an intracameral corticosteroid. In retina, results were presented for a study in macular telangiectasia type 2, a novel biologic in phase 3 for neovascular age-related macular degeneration (wet AMD), and studies are underway for a host of systemic and ocular treatments for diabetic and degenerative eye disease, including gene therapy. Development of novel antibiotics has long been a concern of physicians, developers, and regulators. Thus the recent FDA approval of a new systemic fluoroquinolone to treat dermatological disease is of interest. In the area of personalized medicine, an expanded approval for an oncology therapy was based upon a biomarker, rather than the typical approval based on the organ with the primary cancer. Government agencies and legislators continue to be concerned with drug prices. The U.S. FDA is trying to encourage development of generic drugs for off-patent products (including some ophthalmics) as well as to speed up review of orphan designation requests. Other state and federal legislatures have proposed various laws to restrict price increases as well as to preserve compounding pharmacies, a potential surrogate for off-label use.

Ophthalmic Pharmaceuticals and Biologics

• Aerie announced results for its second phase 3 study of a fixed combination of netarsudil and latanoprost for the treatment of open-angle glaucoma and ocular hypertension (May 2017).

• Aerpio started dosing in the TIME-2b phase 2b study of its AKB-9778 subcutaneous treatment for diabetic retinopathy (June 2017).

• Aldeyra announced results from a phase2b trial of its ADX-102 treatment for allergic conjunctivitis (June 2017).

• Allergan received a positive recommendation from the National Institute for Health and Care Excellence (NICE) in the United Kingdom for Ozurdex^® (intravitreal dexamethasone) for the treatment of noninfectious posterior uveitis (June 2017).

• Applied Genetic Technologies Corporation announced results from a phase 1/2 trial of its adeno-associated virus-based gene therapy for X-linked retinoschisis (June 2017).

• EyeGate is continuing to develop its EGP-437 (dexamethasone by iontophoretic application) for the treatment of postoperative ocular inflammation and pain in ocular surgery patients (May 2017).

• Genentech received FDA approval for an additional indication for Actemra^® (tocilizumab), given by subcutaneous injection. This is the United States first approval for any treatment for giant cell arteritis (May 2017).

• GenSight Biologics reported results of a phase 1/2 trial of its intravitreal GS010, an AAV2 containing the human wild-type ND4 gene, for the treatment of Leber's hereditary optic neuropathy (June 2017).

• Icon Bioscience submitted an NDA to the U.S. FDA for its intracameral dexamethasone (IBI-10090, Dexycu™) for the treatment of postoperative inflammation. The FDA has accepted this NDA for review (June 2017).

• Neurotech announced results of its intravitreal NT-501 (ciliary neurotrophic factor) from a phase 2 trial in patients with macular telangiectasia type 2 (June 2017).

• Nicox received U.S. FDA approval for Zerviate™ (cetirizine) for the topical treatment of allergic conjunctivitis (May 2017).

• Novartis released results of its HAWK study of RTH258 (brolucizumab), an intravitreal treatment for wet AMD (June 2017).

• pSivida announced results from a phase 3 study of its Durasert™ (fluocinolone acetonide intravitreal implant) for the treatment of posterior segment uveitis. They also announced submission of a market authorization application to the European Medicines Agency (EMA) (June 2017).

• ReNeuron is in a phase 1/2 clinical trial in the United States for a cryopreserved formulation of human retinal progenitor cells for patients with retinitis pigmentosa (June 2017).

• Tisbury Pharmaceuticals is developing a novel therapy for the treatment of glaucoma (June 2017).

Medical Devices

• Allergan received clearance from the U.S. FDA for its TrueTear™ Intranasal Tear Neurostimulator to increase tear production (April 2017).

• Implandata received market certification (CE) in Europe for its EyeMate™ IOP monitoring system (May 2017).

Regulatory, Government, and Pharmaceutical Industry

• Apellis received orphan drug designation from the European Medicines Agency for APL-2, a complement inhibitor, for the treatment of paroxysmal nocturnal hemoglobinuria (May 2017).

• Melinta Therapeutics received U.S. FDA approval for delafloxacin, by either oral or intravenous routes, for the treatment of acute bacterial skin and skin structure infections (June 2017).

• Ocugen is developing drug candidates for treatment of inflammatory, degenerative, and neovascular diseases of the eye, which activated novel biologic pathways (June 2017).

• The American Psychological Association (APA) is enforcing the Digital Millennium Copyright Act against authors who post their final, formatted publications in APA journals on their academic web sites (June 2017).

• The coowner of a pharmacy deemed responsible for the deaths of 76 people in a national meningitis outbreak because of contamination was sentenced to prison for conspiracy and fraud charges (June 2017).

• The EMA expanded its 2015 guidance with regard to publication of submitted data to include modifications of indications as well as pediatric study extensions (April 2017).

• The International Committee of Medical Journal Editors proposed a statement to be required as of July 1, 2018, on all articles with explicit data sharing plans (June 2017).¹

• The U.S. FDA provided a list of ∼180 approved medications, which are off patent and have no generic approvals (June 2017). The FDA also announced a plan to accelerate review of requests for orphan drug designation (June 2017). They requested Endo Pharmaceuticals to remove its Opana ER^® (oxymorphone) from the market over concerns about the product's links to injection drug abuse (June 2017). Finally, FDA approved Keytruda^® (pembrolizumab) for cancer treatment based upon a biomarker (microsatellite instability-high or mismatch repair deficient). According to Richard Pazdur, M.D. of FDA's Office of Hematology and Oncology Products, this is the first approval “…based on a tumor's biomarker without regard to the tumor's original location” (May 2017).

• The U.S. House of Representatives is considering the “Preserving Patient Access to Compounded Medications Act of 2017” (HR 2871), sponsored by representatives Morgan Griffith (R-VA) and Henry Cuellar (D-TX) to protect “named patient” compounding pharmacy use (June 2017).

• In state legislature news, the Maryland State General Assembly passed a bill that would give the state attorney general powers to sue drug manufacturers that dramatically raise prices on off-patent or generic drugs (House Bill 631, April 2017). The California State Senate is considering SB 17 sponsored by Ed Hernandez (D-West Covina), which would increase the regulation of pharmaceuticals in the state, including creation of a new board and advanced notice of price increases (June 2017).

Footnotes

Author Disclosure Statement

The author consults for numerous ophthalmic pharmaceutical and medical device firms.

References

Taichman

D.B.

, Sahni

, Pinborg

, et al. Data sharing statements for clinical trials: a requirement of the International Committee of Medical Journal Editors. PLoS Med. 14:e1002315, 2017.