April 2020 decisions expected from the FDA

Your snapshot of new drugs expecting FDA decisions in April 2020

March 15, 2020
At Prime Therapeutics (Prime), we’ve positioned ourselves to best prepare our clients to manage the new drugs that come on the market. Our clinical and trade relations teams keep a keen eye on drugs likely to be approved by the U.S. Food and Drug Administration (FDA).

4/18/20: UGN-101 (mitomycin gel for instillation)
The U.S. Food and Drug Administration (FDA) gave UroGen Pharma’s UGN-101 (mitomycin gel for instillation) Orphan Drug Designation. It would be the first drug approved and the first non-surgical option for the treatment of low-grade upper tract urothelial cancer (LG UTUC). Six weekly instillations of UGN-101, administered via a standard catheter, allows urinary tract tissue longer exposure to mitomycin, thereby enabling the treatment of tumors by non-surgical means.

4/26/2020: opicapone
The FDA is reviewing Neurocrine Biosciences’ opicapone as a combination treatment of levodopa/carbidopa to treat patients with Parkinson’s disease experiencing OFF episodes. Opicapone is a once-daily, selective catechol-o-methyltransferase (COMT) inhibitor used as adjunct therapy to levodopa/DOPA decarboxylase inhibitors in Parkinson’s patients. Neurocrine Biosciences’ is submitting for approval based on two Phase 3 trials that enrolled more than 900 Parkinson’s patients. The studies demonstrated that opicapone given as an add-on to levodopa resulted in longer periods without dyskinesia or involuntary movements compared to placebo by up to two hours.1 Opicapone must be taken at bedtime and at least one hour before or after levodopa combinations. Similar products include Bausch’s Tasmar® (tolcapone) and Novartis’ Comtan® (entacapone).

4/27/2020: Trevyent™ (treprostinil for infusion with PatchPump device)
SteadyMed’s Trevyent is being reviewed by the FDA as a drug-device combination infusion system that delivers the vasodilatory prostacyclin analog subcutaneously or intravenously over 48 hours using SteadyMed’s PatchPump pre-programmed single-use disposable infusion platform for treatment of pulmonary arterial hypertension (PAH). Treprostinil, a prostacyclin PH-targeted therapy that helps relax the pulmonary arteries, is already FDA-approved. Trevyent PatchPump system is pre-filled and pre-programmed to deliver a steady flow over two days. In January 2016, Trevyent was granted Orphan Drug Designation. In August 2017 the FDA issued a Refusal to File letter which stated application was incomplete and asked for additional information on specifications and performance of the PatchPump device. SteadyMed conducted a 60-patient study, testing the PatchPump device containing a placebo, to be used for 48 hours to evaluate the essential safety and performance functions. The results indicated that the PatchPump device performed as intended.2 Similar products include United Therapeutics Corporation’s Remodulin® (treprostinil) and generics in oral and inhaled forms.

4/30/2020: Contepo™ (fosfomycin for injection)
Nabriva Therapeutics’ Contepo is being reviewed by the FDA for complicated urinary tract infections (cUTI). Contepo is an intravenous (IV) antibiotic with broad spectrum activity against Gram-negative and Gram-positive organisms. Nabriva Therapeutics is applying for approval using the 505(b)(2) pathway with Zambon’s Monurol® (fosfomycin) as its reference drug. Nabriva Therapeutics’ also conducted Zeus Study, which found Contepo to be statistically non-inferior to piperacillin/tazobactam in patients with cUTI including pyelonephritis.3 In April 2019, the FDA issued a Complete Response Letter due to issues related to facility inspections and manufacturing deficiencies. In December 2019, Nabriva Therapeutics resubmitted their New Drug Application for Contepo. Similar products include piperacillin/tazobactam.

4/30/2020: isatuximab
The FDA is reviewing Sanofi’s isatuximab for treatment of relapsed/refractory multiple myeloma (RRMM) in combination with pomalidomide and dexamethasone. Isatuximab is an IV monoclonal antibody targeting a specific epitope on the CD38 receptor. Isatuximab was granted Orphan Designation by the FDA. Isatuximab’s application is based on Phase 3 Icaria-mm trial results that demonstrated isatuximab added to pomalidomide and dexamethasone showed statistically significant improvements compared to pomalidomide and dexamethasone (pom-dex) alone (overall response rate 60% vs. 35%). Icaria-mm also demonstrated isatuximab combination had a longer duration of response compared to pom-dex alone (11.53 months vs. 6.47 months).4 Sanofi is currently conducting multiple ongoing Phase 3 clinical trials of isatuximab in combination with current standard treatments for relapsed/refractory or newly-diagnosed multiple myeloma, hematologic malignancies and solid tumors. Similar products include Janssen’s Darzalex® (daratumumab).

While the information in this newsletter is from sources we believe to be reliable, we do not warrant that the information in this document is free from error. Use it only as a guide. Statements regarding drugs or manufacturers are not intended as promotion; those statements should not be used to make assumptions about formulary status. Each trademarked drug name is the property of its respective owner.


1. https://www.prnewswire.com/news-releases/neurocrine-biosciences-announces-fda-acceptance-of-new-drug-application-for-opicapone-as-an-adjunctive-treatment-for-patients-with-parkinsons-disease-300882953.html

2. https://www.globenewswire.com/news-release/2017/04/05/954740/0/en/SteadyMed-Completes-Clinical-Validation-Study-of-Trevyent.html

3. https://www.biospace.com/article/releases/nabriva-therapeutics-resubmits-new-drug-application-for-intravenous-contepo-fosfomycin-for-injection/

4. http://www.news.sanofi.us/press-releases?item=137220

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