October 2023 decisions expected from the FDA

Your monthly synopsis of new drugs expected to hit the market

September 8, 2023
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 October 2023:

10/09/2023: BIIB800/BAT1806 (tocilizumab)
The United States (US) Food and Drug Administration (FDA) is reviewing Biogen’s BIIB800 (BAT1806), a proposed biosimilar referencing Genentech’s IV formulation of Actemra® (tocilizumab), an interleukin-6 receptor-directed monoclonal antibody. Actemra is indicated for the treatment of rheumatoid arthritis (RA), giant cell arteritis (GCA), systemic sclerosis-associated interstitial lung disease (SSc-ILD), polyarticular and systemic juvenile idiopathic arthritis (pJIA, sJIA), cytokine release syndrome (CRS), and Coronavirus disease-19 (COVID-19). BIIB800 demonstrated similar efficacy, safety, and immunogenicity as Actemra IV in a Phase 3 trial with 621 patients with RA.1 BIIB800 will be the first Actemra biosimilar available in the US if approved and may launch in late 2023 depending on patent litigation.

10/11/2023: AVT04 (ustekinumab)
Alvotech’s AVT04 has been accepted for review by the FDA as a biosimilar candidate to Janssen’s interleukin-12 and-23 antagonist Stelara® (ustekinumab) for subcutaneous (SC) injection. Stelara is indicated for the treatment of select adults and pediatric patients (≥ 6 years of age) with plaque psoriasis (PSO), psoriatic arthritis (PsA) and in adults with Crohn’s disease (CD) or ulcerative colitis (UC) when given via IV or SC injection. In a Phase 3 clinical trial in 581 adults with PSO, the improvement in the Psoriasis Area and Severity Index (PASI) was similar between patients who received Stelara throughout the study and those who switched to AVT04 after 16 weeks. The safety profile and pharmacokinetic were also similar between the groups.2 If approved, AVT04 will be the first FDA-approved biosimilar to Stelara and may be available in the U.S. no later than February 21, 2025, based on a settlement agreement.3

10/17/2023: Maxigesic® IV (ibuprofen/acetaminophen)
Upon resubmission of their 505(b)(2) new drug application (NDA), the FDA is reviewing Hyoris Pharmaceuticals’ non-steroidal anti-inflammatory drug (NSAID) product Maxigesic IV. It contains ibuprofen 300 mg and acetaminophen 1,000 mg and is seeking approval for treatment of post-surgical pain. In an open label, single-arm, Phase 3 trial (NCT04005755), Maxigesic IV was administered IV over 15 minutes every six hours for two to five days in 232 adults following non-laparoscopic general, plastic, or orthopedic surgery.4 The study demonstrated that the combination of ibuprofen and acetaminophen produced greater pain relief and faster onset of action compared to either component used alone.5 If approved, Maxigesic IV will provide a non-opioid option for relief of post-surgical pain in a surgical facility setting.

10/17/2023: Xphozah® (tenapanor)
Ardelyx is seeking FDA approval of Xphozah (tenapanor) for the control of serum phosphate in adults with chronic kidney disease (CKD) who are on dialysis and have an inadequate response or intolerance to phosphate binder therapy. Xphozah inhibits the sodium hydrogen exchanger 3 (NHE3). The oral agent works locally in the gastrointestinal (GI) tract to reduce sodium and phosphate absorption. FDA submission was supported by three Phase 3 clinical trials that evaluated tenapanor in doses up to 30 mg twice daily in patients on maintenance dialysis. In the AMPLIFY trial (n=236), Xphozah plus a phosphate binder resulted in a significantly greater mean change in serum phosphorus concentration from baseline to week 4 compared with a phosphate binder alone (LS mean difference -0.65; p<0.001).6 The BLOCK (n=219) and PHREEDOM (n=564) trials demonstrated significantly larger mean changes in serum phosphate level with Xphozah compared to placebo during the 4-week and 12-week withdrawal periods of each trial, respectively (BLOCK: difference, -0.72 mg/dL [p=0.003]; PHREEDOM intent-to-treat: difference, -0.7 mg/dL [p=0.002]).7,8 The PHREEDOM study also included a sevelamer control arm; serious adverse events were reported less often with Xphozah than with sevelamer and a post hoc analysis reported that efficacy was similar between the two products. Diarrhea was the most common treatment-emergent adverse event (TEAE) reported with Xphozah. If approved, Xphozah will be the first non-phosphate binder medication for the treatment of hyperphosphatemia in CKD patients. Since Xphozah is minimally absorbed in the GI tract, systemic accumulation of cations is not anticipated, which is a potential concern with the available phosphate binders (e.g., Auryxia®, Fosrenol®, Renvela®, Phoslyra®, Renagel®, Velphoro®). Tenapanor 50mg tablets are currently available as brand Ibsrela®, also by Ardelyx, for the treatment of irritable bowel syndrome with constipation (IBS-C) in adults.

10/20/2023: IDP-126 (clindamycin 1.2%/adapalene 0.15%/benzoyl peroxide 3.1% gel)
The FDA is reviewing Bausch Health’s IDP-126 (clindamycin/adapalene/benzoyl peroxide) for the treatment of acne vulgaris. If approved, it will be the first fixed-dose, triple combination product─containing an antibiotic, retinoid, and bacteride─ available for this indication. Two Phase 3 clinical trials evaluated once daily topical application of IDP-126 in patients ≥ 9 years of age.9,10,11 In both trials, all co-primary efficacy endpoints were met, including changes from baseline in inflammatory lesion count, non-inflammatory lesion count, and percentage of patients achieving treatment success (defined as a 2 grade reduction of the Evaluator Global Severity Score [EGSS] with a final score of indicating clear or almost clear skin). TEAEs reported were mild to moderate in severity.

10/21/2023: etrasimod
Etrasimod, a selective sphingosine-1-phosphate (S1P) receptor modulator, by Pfizer is being reviewed by the FDA for the treatment of moderate to severe ulcerative colitis (UC). Etrasimod 2mg orally once daily was evaluated in the Phase 3 ELEVATE-UC-12 (n=354) and ELEVATE-UC-52 (n=433) trials in patients ≥ 16 years of age with moderate to severe UC and prior treatment with a biologic agent or Janus kinase (JAK) inhibitor.12 Both studies reported, at week 12, significantly more patients treated with etrasimod achieved clinical remission of UC than those who received placebo (ELEVATE-UC-12: 25% versus 15%, p=0.026; ELEVATE-UC-52: 27% versus 7%, p=0.0001). If approved, etrasimod will be the second S1P receptor modulator to treat UC available in the US. Unlike Zeposia® (ozanimod), etrasimod was not associated with serious bradycardia or atrioventricular block in clinical trials, and dose titration was not required.

10/22/2023: CSF-1 (pilocarpine hydrochloride [HCl] 0.4%)
The FDA has accepted the Oasis Pharmaceuticals’ 505(b)(2) new drug application (NDA) for CSF-1 to improve near vision for individuals with presbyopia. The corrective eye drops contain low-dose, preservative-free, pilocarpine hydrochloride (0.4%) and improves near visual acuity by pupil modulation (pinhole effect) and increased depth of field. Two Phase 3 clinical trials, NEAR-1 and NEAR-2, evaluated CSF-1, administered as 1 drop in each eye twice daily, in > 600 adults (45 to 64 years of age) with presbyopia.13,14,15 Both studies met the primary endpoints on Day 8, including achieving a ≥ 3-line gain in distance-corrected near visual acuity (DCNVA) and no loss of ≥ 1-line in distance visual acuity. The most common TEAE reported with CSF-1 was headache and instillation site pain. If approved, CSF-1 will be the second pilocarpine ophthalmic formulation for the treatment of presbyopia in adults. It could compete with the higher-strength Vuity® (pilocarpine HCl 1.25%) by Allergan, that contains benzalkonium chloride as a preservative and is administered once-daily.

10/22/2023: CT-0P13 SC (infliximab, subcutaneous)
The FDA is reviewing Celltrion’s subcutaneous (SC) formulation of infliximab for the treatment of inflammatory bowel disease (IBD). The Biologics License Application (BLA) for the tumor necrosis factor (TNF) blocker is based on data from the Phase 3 LIBERTY-UC (n=438) and LIBERTY-CD (n=343) trials in adults with ulcerative colitis and Crohn’s disease, respectively. Both studies administered CT-P13 SC as maintenance therapy after induction therapy with IV infliximab and demonstrated superiority of CT-P13 SC over placebo in producing clinical remission at Week 54 in patients with UC (43.2% versus 20.8%, respectively; p<0.0001) and CD (62.3% versus 32.1%, respectively; p<0.0001).16 If approved, the CT-P13 SC will provide a convenient maintenance option for patients with UC and CD compared to IV infliximab (Remicade™, IV biosimilars).

10/26/2023: vamorolone
Santhera Pharmaceuticals’ vamorolone is undergoing FDA review for the treatment of Duchenne muscular dystrophy, a rare X-linked neuromuscular disorder affecting boys. Vamorolone is an oral dissociative steroid that binds to the glucocorticosteroid receptor. In the Phase 2b VISION-DMD trial, vamorolone 6mg/kg per day led to a significant difference in the primary endpoint of time to stand (TTSTAND) velocity compared to placebo (LS mean, 0.05 versus -0.01 m/s, respectively; p=0.002) at 24 weeks.17 If approved, vamorolone could be available in the U.S. in the fourth quarter of 2024.

4Q 2023: tislelizumab
Tislelizumab is a humanized immunoglobulin G4 (IgG4) anti-programmed cell death (PD)-1 monoclonal antibody designed to minimize binding to macrophage Fc gamma receptors (FcγRs) and potentially reduce anti-PD-1 resistance. The Phase 3 RATIONALE 302 trial (n=512) compared second-line treatment of tislelizumab with investigator’s choice chemotherapy (e.g., paclitaxel, docetaxel, irinotecan) in patients with advanced or metastatic ESCC.18,19 At data cut-off, the median follow-up was 8.5 months in the tislelizumab group and 5.8 months in the chemotherapy group. Data revealed that the primary endpoint of overall survival (OS) was significantly longer with tislelizumab compared to chemotherapy (median OS, 8.6 versus 6.3 months, respectively; hazard ratio, 0.7; p=0.0001). If approved tislelizumab will be the third PD-1 inhibitor option for second-line therapy for ESCC. Tislelizumab differs from other PD-1 inhibitors by its lack of FcγR binding; however, it remains to be seen whether this confers clinical benefit over the available PD-1 inhibitors. Based on non-comparative data, tislelizumab appears to demonstrate similar efficacy (based on OS) to Opdivo® (nivolumab) and Keytruda® (pembrolizumab) for the proposed indication.

Contact your Prime representative for more information or with any questions you have about drugs in the pipeline.

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://ard.bmj.com/content/annrheumdis/81/Suppl_1/388.2.full.pdf.
  2. https://pubmed.ncbi.nlm.nih.gov/37435850/
  3. https://www.tevapharm.com/news-and-media/latest-news/alvotech-and-teva-secure-u.s.-license-date-for-avt04-a-proposed-biosimilar-to-stelara/
  4. https://www.clinicaltrials.gov/study/NCT04005755?tab=table
  5. Press release. https://www.globenewswire.com/news-release/2023/05/02/2658739/0/en/Hyloris-announces-potential-registration-date-for-Maxigesic-IV-in-the-US.html
  6. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8259655/
  7. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6442342/
  8. https://journals.lww.com/kidney360/fulltext/2021/10000/safety_and_efficacy_of_tenapanor_for_long_term.9.aspx
  9. https://www.accesswire.com/viewarticle.aspx?id=752070&lang=en
  10. https://www.clinicaltrials.gov/study/NCT04214652
  11. https://www.clinicaltrials.gov/study/NCT04214639
  12. https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(23)00061-2/fulltext
  13. https://www.prnewswire.com/news-releases/fda-accepts-orasis-pharmaceuticals-new-drug-application-for-csf-1-for-the-treatment-of-presbyopia-301750185.html
  14. https://www.clinicaltrials.gov/study/NCT04599933
  15. https://www.clinicaltrials.gov/study/NCT04599972?tab=table
  16. https://www.businesswire.com/news/home/20221221005226/en/Celltrion-USA-announces-submission-of-the-Biologics-License-Application-BLA-of-novel-subcutaneous-formulation-of-CT-P13-to-U.S.-Food-and-Drug-Administration
  17. https://jamanetwork.com/journals/jamaneurology/fullarticle/2795868?utm_campaign=articlePDF&utm_medium=articlePDFlink&utm_source=articlePDF&utm_content=jamaneurol.2022.2480
  18. https://meetings.asco.org/abstracts-presentations/195922
  19. https://www.businesswire.com/news/home/20210604005076/en/

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