Eyes on New Product Development

Abstract

In this reporting period, the U.S. Food and Drug Administration (FDA) approved the first gene therapy in the United States. The indication was a form of acute lymphoblastic leukemia and opens the door to potential treatments for ocular diseases. As well, the “FDA Reauthorization Act of 2017” (FDARA) was signed into law, extending the program wherein sponsors pay user fees to support review of new product applications. The FDA also has begun enforcement against “stem cell clinics,” which FDA finds to be marketed against current statute and regulation. Regulatory activities worldwide reflect novel medicines (e.g., use of diagnostic in combination with therapy) and issues surrounding compounding pharmacies as well as political conflict. Products to treat dry eye disease are continuing to be evaluated from early to late stage development. In retina, a wide range of products are being evaluated for improved treatment of wet age-related macular degeneration (AMD) as well as gene and immunotherapy for retinal degenerative diseases, including a direct cortical stimulator for blind patients. Treatment of inflammation and glaucoma and novel drug delivery approaches are in clinical trials.

Ophthalmic Pharmaceuticals and Biologics

• Aerie Pharmaceuticals entered into a collaborative research, development, and license agreement with the Dutch firm Royal DSM for its bioerodible polymer implant technology, initially for retinal diseases (July 2017).

• Allegro Ophthalmics announced results of the phase 2b DEL MAR study of Luminate™, a integrin-peptide therapy, for diabetic macular edema (August 2017).

• Apellis Pharmaceuticals announced results of its phase 2 trial of its complement C3 inhibitor, APL-2, in patients with geographic atrophy (August 2017).

• Applied Genetic Technologies Corporation filed an investigational new drug application with the U.S. FDA for a phase 1/2 clinical trial of its gene therapy candidate for X-linked retinitis pigmentosa (August 2017).

• Clearside Biomedical completed patient enrollment in the pivotal PEACHTREE clinical trial of its suprachoroidal administration of CLS-TA (triamcinolone acetonide) for the treatment of macular edema associated with noninfectious uveitis (August 2017).

• EyeGate Pharmaceuticals commenced enrollment in its phase 2b clinical trial of its EGP-437 product (dexamethasone) for the treatment of pain and inflammation in patients undergoing cataract surgery (August 2017).

• GenSight Biologics completed enrollment in RESCUE, a phase 3 clinical trial of its GS010 gene therapy for the treatment of Leber's Hereditary Optic Neuropathy (August 2017).

• Nemus Bioscience continues its preclinical development of NB2222, a cannabinoid for ophthalmic diseases (August 2017).

• Ocugen received FDA orphan drug designation for OCU300 (brimonidine) to treat ocular graft-versus-host disease (August 2017).

• Ophthotech announced results from a phase 3 clinical trial investigating the concomitant use of Fovista^® (pegpleranib) anti-PDGF therapy with Eylea^® (aflibercept) or Avastin^® (bevacizumab) for the treatment of wet AMD (August 2017).

• RegeneRx completed enrollment in its phase 3 “ARISE-2” clinical trial testing RGN-259 preservative-free eye drops for dry eye syndrome (July 2017).

• Santen initiated a phase 2a clinical study of the efficacy and safety of intravitreal injections of DE-122 for the treatment of wet AMD (July 2017).

• Shire submitted a marketing authorization application (MAA) for lifitegrast (Xiidra™) to the United Kingdom (August 2017).

• Spark Therapeutics submitted a MAA to the European Medicines Agency (EMA) for Luxturna™ (voretigene neparvovec) for Leber congenital amaurosis or retinitis pigmentosa caused by biallelic RPE65 gene mutations (August 2017).

• Tear Solutions commenced enrollment for its trial of Lacripep™ (lacritin) in patients with dry eye disease associated with Primary Sjögren's Syndrome (July 2017).

• Valeant's latanoprostene bunod received a Complete Response Letter from the FDA for its New Drug Application for treatment of glaucoma (August 2017).

Ophthalmic Devices

• Second Sight Medical Products is starting a human feasibility clinical study of the Orion cortical visual prosthesis system for blind patients, bypassing the retina and optic nerve and stimulating the visual cortex (August 2017).

Regulatory, Government, and Pharmaceutical Industry

• Allergan reached a settlement with Famy Care Limited regarding litigation concerning its U.S. patents for Restasis^® (cyclosporine ophthalmic emulsion, August 2017).

• Cempra, Inc. and Melinta Therapeutics entered into an agreement under which Melinta will merge with a subsidiary of Cempra. The firms research and develop novel anti-infective therapies (August 2017).

• Janssen, a division of Johnson & Johnson, stated that its anesthetic, etomidate, now off-patent and manufactured by several firms, should not be used for lethal injection in convicted prisoners (August 2017).

• Johnson & Johnson Vision will acquire TearScience through Abbott Medical Optics, its surgical vision operating company (August 2017).

• Novartis received approval by the FDA for its Kymriah^® (tisagenlecleucel), a gene therapy for forms of acute lymphoblastic leukemia. This is the first gene therapy approved in the United States. In noting the milestone approval, FDA Commissioner Scott Gottlieb, MD, said that this approval involved a cross-agency approach, and was given only 7 months after submission of the marketing application (August 2017).

• The “FDARA” (S 934) was signed into law. This extends by 5 years the Prescription Drug User Fee Act. This acts deals primarily with user fees paid by sponsors, but also includes directives on more rapid review of generic drugs (August 2017).

• The Drug Controller General of India requested local pharmaceutical firms to prepare an emergency plan on dealing with any blockade on shipments from China of active pharmaceutical ingredients, or raw materials used to make medicines, in the event of border tensions escalating (August 2017). The Indian health ministry plans to waive clinical trial requirements for select essential drugs approved in other major markets in an effort to make innovative medicines more readily available (August 2017).

• The EMA is working on guidelines to help drug firms to develop companion diagnostics in parallel with medicines according to a concept article (August 2017).

• The FDA conduced enforcement actions against clinical marketing of stem cells as therapeutics. FDA Commissioner Scott Gottlieb, MD, stated that FDA “will not allow deceitful actors to take advantage of vulnerable patients by purporting to have treatments or cures for serious diseases without any proof that they actually work” (August 2017).

• The FDA issued an alert on adverse events reported for a compounding pharmacy, off-label (July 2017).

Footnotes

Author Disclosure Statement

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