Eyes on New Product Development

Abstract

In this reporting period, 4 new therapies (2 to treat glaucoma, 1 to treat allergic conjunctivitis, and 1 to treat postoperative inflammation) were resubmitted to the U.S. Food and Drug Administration (FDA) for marketing approval. The potential approval of these agents was held up for manufacturing reasons.¹ A new gene therapy for an inherited retinal degeneration entered clinical trials as well as a novel therapy for dry eye. Furthermore, a novel cannabinoid is being evaluated for topical treatment of glaucoma, which may improve on the low therapeutic index for this class of compounds,² and a business arrangement was made to explore clustered regularly interspaced short palindromic repeats (CRISPR) technology in ophthalmology. Growing concerns about how pharmaceutical products are developed, regulated, and priced in the United States have lead to several actions by the pharmaceutical industry and government. A regulatory pathway used for “repurposed” drugs, perceived by many to have a faster review time, may have actually taken longer.

Ophthalmic Pharmaceuticals and Biologics

• Aerie Pharmaceuticals resubmitted its new drug application (NDA) for Rhopressa™ (netarsudil) to the U.S. FDA after notification by its contract drug product manufacturer that the vendor's Tampa, Florida, facility is now ready for preapproval inspection by the FDA (March 2017).

• Allergan and Editas Medicine entered into a strategic R&D alliance to discover and develop CRISPR genome editing medicines for eye diseases (March 2017).

• Nemus Bioscience is working with Nanomerics Ltd. to develop a topical ocular formulation of tetrahydrocannabinol-valine-hemisuccinate, a prodrug of tetrahydrocannabinol (NB1111) for the treatment of glaucoma (March 2017).

• Nicox resubmitted an NDA for AC-170 (Zerviate^®, certizine) to the FDA for the treatment of ocular itching associated with allergic conjunctivitis (March 2017).

• NightstaRx Ltd. commenced enrolling and dosing subjects in a phase 1/2 clinical trial for patients with X-linked retinitis pigmentosa. The therapy is a codon-optimized copy of the retinitis pigmentosa GTPase regulator using an adeno-associated viral vector (March 2017).

• Ocular Therapeutix resubmission of its NDA for Dextenza™, an intracanicular dexamethasone insert for treatment of postoperative inflammation, was accepted for review by the FDA for review (February 2017).

• TearSolutions, Inc. submitted an investigational new drug exemption to the FDA for lacritin for the treatment of dry eye disease (February 2017).

• Tyrogenex completed patient enrollment in the phase 2 wet AMD in Previously treated Eylea patients with X-82 (APEX) study of its X82 (vorolanib) for the oral treatment of wet ager-elated macular degeneration (March 2017).

• Valeant Pharmaceuticals subsidiary, Bausch + Lomb, and Nicox S.A. announced the resubmission of an NDA to the FDA seeking approval for latanoprostene bunod ophthalmic solution, 0.024% (February 2017).

Regulatory, Government, and Pharmaceutical Industry

• Amgen is making an offer of refunds to patients using its new drug for the treatment of hyperlipidemia, Repatha^® (evolocumab), if they suffer a heart attack or stroke (March 2017).

• Some patients are complaining about the secrecy of payments from payers (typically insurance companies) to pharmacies (called “clawbacks”) on prescription pharmaceuticals (February 2017).

• The U.S. Government Accounting Office, acting on a request from Senators Orrin Hatch (R-Utah), Chuck Grassley (R-Iowa), and Tom Cotton (R-Ark.), will investigate potential abuses of the Orphan Drug Act (March 2017).

• The United States and the European Union (EU) completed an exchange of letters to amend the Pharmaceutical Annex to the 1998 U.S.–EU Mutual Recognition Agreement. Under this agreement, United States and EU regulators will be able to utilize each other's good manufacturing practice inspections of pharmaceutical manufacturing facilities (March 2017).

• Tufts Center for the Study of Drug Development released a report in which it found that NDAs for already approved molecules with formulations and/or routes and indications (505(b)(2)) took an average of about 5 months longer than those for new molecular entities for FDA review. Note that 505(b)(2) applications represent nearly two-thirds of all NDAs approved during 2009–2015 (March 2017).

Footnotes

Author Disclosure Statement

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

References

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G.D

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Novack

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