January 2024 decisions expected from the FDA

December 15, 2023

Your monthly synopsis of new drugs expected to hit the market

FDA Decisions Expected

At Prime Therapeutics (Prime), we’ve positioned ourselves to best prepare our clients to manage new drugs. 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). 

Drug pipeline for January 2024

January 2024: STS101 (dihydroergotamine [DHE] nasal powder) 

The United States (U.S.) Food and Drug Administration (FDA) is reviewing the 505(b)(2) new drug application (NDA) for Shin Nippon Biomedical Laboratories’ STS101, a 5-HT1D serotonin receptor agonist for the acute treatment of migraine. Valeant’s D.H.E 45 was used as the reference product. The open-label, phase 3 ASCEND trial reported that 21.9% of patients were free from migraine pain and 32.8% were free from their most bothersome symptom (MBS) (photophobia, phonophobia, nausea) at 2 to 48 hours after a single 5.2 mg dose of STS101.¹ In the phase 3, double-blind EMERGE and SUMMIT trials, the percentages of patients who were free from migraine pain or MBS were not statistically significant when compared to placebo at 2 hours post dose which was the primary endpoint. Statistical significance was reached at all time points 3 hours or later after the dose.²,³ The most common side effects reported were nasal discomfort and dysgeusia. If approved, STS101 will add another intranasal DHE option to the multiple generics and brand name products available to treat acute migraine.

January-February 2024: TAK-721 (budesonide oral suspension)

The FDA is reviewing the 505(b)(2) NDA for Takeda’s TAK-721 for the short-term treatment of eosinophilic esophagitis (EoE). TAK-721 is a muco-adherent topically active viscous formulation. The double-blind, phase 3 ORBIT-1 trial enrolled patients 11 to 55 years of age with EoE and dysphagia. It reported that twice daily doses of TAK-721 led to a significant increase the rate of histologic response (≤ 6 eosinophils/high-powered field) compared to placebo (53.1% versus 1%, respectively; p<0.001) after 12 weeks.4 There was also a significant difference in the proportion of patients who experienced ≥ 30 reduction in dysphagia (52.6% versus 39.1%, respectively; p=0.024). Oral and esophageal candidiasis was reported in < 5% of patients in either group. A withdrawal study that included patients from ORBIT-1 reported, over 36 weeks, among patients who were full responders to induction therapy, histologic and dysphagia symptom relapses were reported more often in those who stopped TAK-721 compared to those who continued TAK-721 treatment (histologic, 43.5% versus 24% [p=0.131]; dysphagia, 50% versus 16.7% [p=0.038], respectively).5 The FDA has granted TAK-721 Breakthrough Therapy and Orphan Drug designations for EoE. If approved, TAK-721 will be the first corticosteroid indicated for EoE in the U.S.

01/03/2024: cosibelimab

Checkpoint Therapeutics is awaiting FDA approval of cosibelimab for the treatment of metastatic cutaneous squamous cell carcinoma (cSCC) for locally advanced cSCC in patients who are not candidates for curative surgery or radiation. Cosibelimab is an anti-programmed death ligand-1 (PD-L1) antibody. FDA submission is based on data from an open-label, phase 1 clinical trial (NCT03212404) that reported an objective response rate (ORR) of 47.4% at a median follow-up of 15.4 months (range, 0.4 to 40.5 months).6 Treatment-related adverse effects (TEAEs) were experienced by 70.5% of patients; 9% had a grade 3 or higher effects. Most common TEAEs were fatigue, rash, and anemia. If approved, cosibelimab will compete with the programmed death receptor-1 (PD-1)-blocking antibodies Keytruda® (pembrolizumab) and Libtayo® (cemiplimab-rwlc); both agents were approved for cSCC based on phase 2 data that reported ORRs of 34% and 47%, respectively.

01/05/2024: berdazimer

Berdazimer is a topical nitric oxide-releasing antiviral by Ligand Pharmaceuticals. It is being reviewed by the FDA for the treatment of molluscum contagiosum (MC). The phase 3, double-blind B-SIMPLE4 trial revealed that significantly more immunocompetent patients ≥ 6 months of age treated with berdazimer achieved the primary endpoint of complete clearance of all MC lesions at week 12 compared to patients who received vehicle (43.5% versus 24.6%, respectively; p<0.001).7 A significant difference between the groups was seen as early as week 4. However, berdazimer did not demonstrate statistical significance in the rate of complete clearance of treatable MC lesions at week 12 in the phase 3, double-blind, vehicle-controlled B-SIMPLE1 (p=0.375) and B-SIMPLE2 (p=0.062) trials. Berdazimer was well-tolerated in clinical trials.8 If approved, It will be the first agent indicated for topical agent for MC that is administration by the patient or caregiver and may be a more convenient alternative to Verrica’s healthcare provider-administered topical Ycanth™ (cantharidin).

01/12/2024: zolbetuximab

The FDA granted a Priority Review for Astellas’ zolbetuximab for the 1st-line treatment of locally advanced unresectable or metastatic gastric or gastroesophageal junction (G/GEJ) adenocarcinoma that is HER2-negative and Claudin-18 isoform 2 (CLDN18.2)-positive. Zolbetuximab is an intravenously (IV)-administered CLDN18.2-directed antibody. The double-blind, phase 3 SPOTLIGHT trial reported a longer median progression-free survival (PFS) when zolbetuximab was added to mFOLFOX6 (folinic acid + 5-fluorouracil + oxaliplatin) chemotherapy compared to chemotherapy alone (10.61 versus 8.67 months, respectively; p=0.0066).9 The ORR was similar between groups. The double-blind, phase 3 GLOW trial reported a significantly longer median PFS when zolbetuximab was added to CAPOX (oral capecitabine + oxaliplatin) chemotherapy compared to chemotherapy alone (8.21 versus 6.8 months, respectively; p=0.0007).10 Overall survival, a key secondary endpoint, was higher in the zolbetuximab treated group (median, 14.29 versus 12.16 months; Hazard Ratio=0.771; 95% confidence interval [CI] 0.615 to 0.965; p=0.0118). The most common TEAEs were nausea, vomiting, and decreased appetite were reported with and without the addition of zolbetuximab. If approved, the Orphan Drug will be the first agent to target CLDN18.2 in patients with HER2-negative, CLDN18.2-positive G/GEJ.

01/13/2024: GC5107B (immune globulin intravenous [IVIG], human, 10% liquid)

GC Biopharma is awaiting approval for GC5107B for patients with primary humoral immunodeficiency (PI). In a phase 3 clinical trial, GC5107B met the primary efficacy endpoint of acute serious bacterial infections, reporting a frequency of 0.02 over 12 months, which meets the FDA guidance of ≤ 1 per person-year.11 The study met its primary safety endpoint of < 40% of infusions with ≥ 1 infusion-related adverse events. If approved, GC5107B will provide another IVIG option for patients with PI.

01/26/2024: DPI-386 (scopolamine gel)

The FDA is reviewing intranasally administered scopolamine gel by Repurposed Therapeutics and Defender Pharmaceuticals for the prevention of motion sickness. In the DPI-386-MS-33 phase 3 clinical trial, DPI-386 led to a significantly lower incidence of vomiting or the need for rescue medication (e.g., antihistamine) (p<0.0001) and the incidence of moderate to severe nausea (p<0.0001) compared to placebo in participants exposed to motion on an ocean-going vessel.12 DPI-386 demonstrated rapid absorption in a pharmacokinetics study. The FDA has granted priority review for the anticholinergic agent, at the request of the U.S. Navy. Scopolamine is currently available as oral tablets and transdermal system for the prevention of motion sickness.

01/31/2024: NVK-002 (atropine 0.01%)

The FDA is reviewing Nevakar/Vyluma’s 505(b)(2) application for NVK002, a low dose atropine 0.01% ophthalmic solution, for the treatment for myopia in children. The phase 3 clinical trial, CHAMP, did not meet the primary endpoint of the portion of participants eyes responding (having < 0.5 D myopia progression from baseline) to atropine 0.02% compared to placebo at 36 months among children 6 to 10 years of age. The study did meet the secondary endpoint of response to atropine 0.01% at 36 months (% responder eyes, 28.5% versus 17.5%, respectively; p=0.03) in children 6 to 10 years of age.13 In addition, significantly more children in the atropine 0.1% group experienced slowed mean spherical equivalent refractive error (SER) progression and slowed axial elongation (p< 0.001 for both). If approved, the anticholinergic agent will be the first medication in the U.S. for myopia.


1. ASCEND: https://www.clinicaltrials.gov/study/NCT04406649?tab=results

2. EMERGE: https://investors.satsumarx.com/press-releases?item=50

3. SUMMIT: https://www.globenewswire.com/news-release/2023/05/18/2672216/0/en/Satsuma-Pharmaceuticals-Announces-FDA-Acceptance-of-505-b-2-NDA-for-STS101-a-Novel-and-Investigational-Dihydroergotamine-DHE-Nasal-Powder-Product-for-the-Acute-Treatment-of-Migrain.html

4. ORBIT-1: https://www.takeda.com/en-us/newsroom/news-releases/2019/first-ever-u.s.-pivotal-phase-3-clinical-study-in-eosinophilic-esophagitis-eoe-completes

5. Withdrawal study: https://www.cghjournal.org/action/showPdf?pii=S1542-3565%2821%2900684-4

6. NCT03212404: https://jitc.bmj.com/content/11/10/e007637

7. B-SIMPLE4: https://jamanetwork.com/journals/jamadermatology/fullarticle/2794188

8. Press release: https://novan.com/novan-submits-new-drug-application-to-the-u-s-fda-for-berdazimer-gel-10-3-sb206-for-the-treatment-of-molluscum-contagiosum

9. SPOTLIGHT: https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(23)00620-7/fulltext

10. GLOW: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10427418/

11. Press release: https://www.businesswire.com/news/home/20210225006332/en/GC-Pharma-Submits-Biologics-License-Application-to-US-FDA-for-Immune-Globulin-GC5107

12. Press release: https://www.globenewswire.com/news-release/2023/06/05/2682084/0/en/Defender-Pharmaceuticals-Reports-Positive-Results-from-Pivotal-Phase-III-Clinical-Trial-of-Intranasal-Scopolamine-for-the-Prevention-of-Nausea-and-Vomiting-Induced-by-Motion.html

13. https://jamanetwork.com/journals/jathx maophthalmology/article-abstract/2805504

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