Abstract
In this review period, the U.S. Food and Drug Administration (FDA) provided further details on some aspects of the recently passed Prescription Drug User Fee Act (PDUFA) VII law, and the U.S. Congress continues to be concerned with drug prices—in this case, evaluating the impact of Pharmacy Benefit Managers (PBMs). There was news from 3 novel pharmacological treatments for dry eye disease (DED)—ranging from early- to late-stage development. Results from pharmacotherapies were presented for 3 different novel indications—Demodex blepharitis, meibomian gland disease (MGD), and myopia. In retina, a wide range of novel therapies are in development.
Ophthalmic Pharmaceuticals and Biologics
Allysta completed enrollment in its Phase 2b/3 trial (OASIS-1) of ALY688 Ophthalmic Solution, a novel anti-inflammatory peptide, for the treatment of DED (November 2022).
Apellis announced 24-month Phase 3 from its DERBY and OAKS trials of intravitreal pegcetacoplan for geographic atrophy, which was submitted to FDA to support its New Drug Application (NDA) under review, moving the PDUFA target action date to February 2023 (November 2022).
Azura announced 3-month efficacy and safety results from its Phase 2b study of AZR—MD—001 0.5% in MGD (November 2022).
Neurotech Pharmaceuticals announced results in 2 replicative Phase 3 clinical trials with their investigational encapsulated cell therapy of Ciliary Neurotropic Factor for the treatment of Macular telangiectasia type 2 (November 2022).
Nicox announced results from its Mont Blanc Phase 3 study of NCX 470 0.1% versus latanoprost 0.005% for the treatment of patients with open-angle glaucoma or ocular hypertension (October 2022).
Novaliq announced that the U.S. FDA accepted for review its NDA for CyclASol (cyclosporine ophthalmic solution), for the treatment for the signs and symptoms of DED (October 2022).
Oculis announced results of an active-controlled multicenter randomized parallel-group Phase 2 clinical trial assessing the effect of topical licaminlimab (OCS-02) in patients with acute anterior uveitis (November 2022). 1
Ocuterra announced results from a Phase1b study of its OTT166, a topical integrin inhibitor, for the treatment of diabetic eye disease (October 2022). 2
Okyo Pharma filed an Investigational New Drug application with the U.S. FDA for its OK—101, a lipid conjugated chemerin peptide agonist of the ChemR23 G-protein coupled receptor, to treat DED (November 2022).
Outlook Therapeutics announced that the FDA has accepted a Biologics License Application for its ONS-5010/Lytenava (bevacizumab-vikg), for the treatment of wet age-related macular degeneration (October 2022).
Salvat submitted an NDA to the U.S. FDA for a topical ocular nanoemulsion formulation of clobetasol, for inflammation and pain in patients undergoing anterior segment surgery (October 2022).
Tarsus Pharmaceuticals announced that the FDA accepted the company's NDA for TP-03 (lotilaner) for the treatment of Demodex blepharitis (November 2022).
Vyluma announced results from its Phase 3 Childhood Atropine for Myopia Progression clinical study of chronic use of topical atropine (October 2022).
Regulatory, Government, and Pharmaceutical Industry
In an editorial in Science entitled “Do no harm,” editor in chief Holden Thorp, PhD, wrote that “until the scientific community deals with misinformation from within, it cannot expect to deal with it from without.” 3
Neurology published the results from an under-enrolled double-masked placebo control randomized trial of systemic prednisolone in children with Bell's Palsy. The journal also posted a commentary discussing the reasons for under-enrollment, including inclusion/exclusion criteria being too restrictive, and not doing an up-front assessment. An ethical issue was raised: “…Study participants assume risk by enrolling in a clinical trial. This risk is taken in vain if the data are discarded.”4,5
Ocuphire announced a license agreement with FamyGen Life Sciences for its Nyxol™ (phentolamine ophthalmic solution) for the treatment of anterior segment indications (November 2022).
Viatris signed an agreement to acquire Oyster Point Pharma and Famy Life Sciences (November 2022).
The FDA:
Provided further details on activities related to the PDUFA VII law, including:
Updating the meeting guidance to reflect a new “Type D,” faster meeting for clarification of selected issues
Issued a program relating to Advancing Real-World Evidence related to PDUFA VII (October 2022). 6
Issued guidances on: In vivo bioequivalence for generic topical dermatologic corticosteroids, requi-rements for postapproval manufacturing changes, manufacturing guidance for manufacturing quality for investigational products that address unmet medical needs in the treatment of serious or life-threatening conditions, multiple endpoints in clinical trials, sameness evaluations for generic products, and expanded access for investigational products (October to November 2022).
Issued a draft proposal to migrate naloxone nasal spray products from prescription to over the counter for treatment of opioid overdose (November 2022).
Proposed updates to a ruling regarding limitations to interstate shipping of compounded medications (October 2022). 7
The Centers for Device and Radiological Health held meeting of the Ophthalmic Devices Panel to discuss the general classification recommendation for ophthalmic dispensers (November 2022).
The International Conference on Harmonisation issued 2 guidances: M10 “Bioanalytical Method Validation and Study Sample Analysis” and S1B(R1) “Addendum to S1B Testing for Carcinogenicity of Pharmaceuticals” (November 2022).
The U.S. Congress is concerned about the role of PBMs in the pricing of medications in the United States and the impact on patient out-of-pocket payments. This is especially of concern for premium-priced biologics, some of which may be going off-patent in the near future, and insulin. In a recent report covering the period 2014–2018, the share of insulin expenditures retained by PBMs increased by 154.6% (from $5.64 to $14.36), the share retained by pharmacies increased by 228.8% (from $6.21 to $20.42), and the share retained by wholesalers increased by 74.7% (from $4.63 to $8.09, November 2022). 8
Footnotes
Author Disclosure Statement
The author consults for numerous ophthalmic, pharmaceutical, and medical device firms. A complete listing of all of G.D.N.'s consulting affiliations can be found online.
Funding Information
No funding was received for this article.