5 FDA Decisions to Watch for Through June 2023

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From a novel topical gene therapy to a multi-pathway targeting HF drug, the second quarter of 2023 is laden with interesting regulatory decisions.

The second quarter of 2023 is shaping up to a formative one for the pharmacotherapeutic fields of chronic disease; among the dozens of US Food and Drug Administration (FDA) PDUFA decision dates on the docket through June are a handful of first-in-class treatments, novel agents, and familiar drugs with refined indication capabilities.

Here are 5 PDUFA dates to look out for through the next 3 months.

SER-109 (Seres Therapeutics) for Recurrent Clostridioides difficile: April 26

A candidate among the emerging live microbiome therapeutic class for C difficile, the oral drug SER-109 has been previously supported by pivotal data from the phase 3 ECOSPOR IV trial wherein just 6.5% of treated patients reported 1 C difficile recurrence at 8 weeks, and 9.7% reported ≥2 recurrences.1

In a 2021 interview with HCPLive, Paul Feuerstadt, MD, of the Yale School of Medicine, stressed that reduced recurrence and sustained treatment response are key components he and colleagues are looking for from the first live microbiome therapy candidates for C difficile.

“This shouldn't be an infection that we treat 3 and 4 times in one individual, that should be an initial, if you recur, we're going to shut it down,” he said. “When patients come into my office, I call myself the closer. But really, I have the tools and I'm comfortable using the tools to shut down that infection and minimize future occurrences.”2

B-VEC (Krystal Biotech) for Dystrophic Epidermolysis Bullosa (DEB): May 19

The topically-applied gene therapy was originally granted a January 5 PDUFA date, but was since delayed to May 19 due to Krystal Biotech’s submission of updated manufacturing information relevant to the agent.3

The unique drug candidate for the rare skin condition caught attention at the American Academy of Dermatology (AAD) 2022 Annual Meeting, when investigators presented phase 3 data showing a significantly greater rate of patients treated with B-VEC achieved complete wound healing versus placebo at both 3 and 6 months (P <.005).

Primary investigator Peter Marinkovich, MD, associate professor of dermatology at Stanford University School of Medicine, told HCPLive at the time that B-VEC may be managed by dermatologists despite its unique gene therapy background, due to the simplicity of its topical application.

“You use it at an outpatient basis, you can just use it in the clinic to treat these patients’ wounds,” Marinkovich said. “I think it’s really a cutting-edge type of therapy that, if everything goes well, may be approved in a year or so.”4

Sotagliflozin (Lexicon Pharmaceuticals) for Heart Failure (HF): May 23

The FDA accepted a New Drug Application (NDA) for the dual SGLT-1 and 2 inhibitor last July, on the backing of the randomized, double-blind SOLOIST-WHF trial data showing sotagliflozin plus standard care was associated with a significant reduction in major adverse cardiovascular events (MACE) among treated patients with type 2 diabetes who had been hospitalized recently due to worsening heart failure.5

Deepak Bhatt, MD, MPH, director of Mount Sinai Heart and Dr. Valentin Fuster Professor of Cardiovascular Medicine at Mount Sinai Icahn School of Medicine, told HCPLive during the American College of Cardiology (ACC) 2022 Scientific Sessions that the unique capability of sotagliflozin to inhibit both SGLT-1 and 2 pathways implicates benefit in both glucose absorption and reduced cardiovascular event risk—a key opportunity for patients with comorbid cardiovascular, diabetologic and renal disease risks.

“So, in addition to the benefits in the kidney with respect to glucose uptake, there’s also benefits in the gut with the SGLT-1 inhibition added to SGLT-2,” Bhatt said.6

Mavacamten (BMS) for NYHA Class II-III Obstructive Hypertrophic Cardiomyopathy (oHCM): June 16

Originally approved to treat symptomatic oHCM in April 2022, the first-in-class myosin inhibitor is up for an expanded indication to reduce patient need for septal reduction therapy (SRT)—a regimen that often requires open-heart or septal ablation procedures.7

Findings from the VALOR-HCM trial, presented at ACC 2022, evidenced the reduced eligibility of SRT among patients with oHCM who received mavacamten over 16 weeks.8

Aflibercept 8 mg (Regeneron) for Wet Age-Related Macular Degeneration (AMD) and Diabetic Macular Edema (DME): June 27

The FDA accepted a Biologics License Application (BLA) for aflibercept 8 mg in February, on the basis of supporting data from the PULSATE and PHOTON trials for the respective indications.9 A majority of patients in the 8 mg aflibercept treatment arms (12- and 16-week dosing) of each trial achieved the primary endpoint of noninferior vision gains versus standard-dose aflibercept injection (8-week dosing).

Flexible dosing regimen with the intravitreal injection therapy is a highly sought-after standard with anti-VEGF therapies—a topline drug class for chronic macular disease, but also one prone to patient inadherence and anxiety surrounding frequent administrations.

Research from Theodore Leng, MD, MS, Associate Professor of Ophthalmology at Stanford University, and colleagues in 2021 showed that patients with wet AMD are prone to anti-VEGF treatment discontinuation or loss in follow-up anywhere up to 6 years into their regimen.

“We definitely want to dig deeper into the treatment gaps we’ve also noticed—where people are stopping treatment and about 40% of patients are discontinuing treatment in less than 2 years, with an average time of about 90 weeks of stopping treatment,” Leng said at the time.10


  1. Inman S. Oral SER-109 Effectively Prevents C Difficile Recurrences in Open-Label Study. HCPLive. Published October 26, 2022. Accessed April 7, 2023.
  2. Walter K. A Race to the Top for The New C Difficile Treatment Class. HCPLive. Published December 10, 2021. Accessed April 7, 2023.
  3. Smith T. FDA Extends PDUFA Date for B-VEC Gene Therapy for Dystrophic Epidermolysis Bullosa. HCPLive. Published January 12, 2023. Accessed April 7, 2023.
  4. Kunzmann K. Topical Gene Therapy B-VEC Reaches Phase 3 Efficacy Endpoints for DEB Disease Reversal. HCPLive. Published March 26, 2022. Accessed April 7, 2023.
  5. Lexion Pharmaceuticals. Lexicon Announces FDA Acceptance of New Drug Application for Sotagliflozin to Treat Heart Failure. News release. Published July 27, 2022. Accessed April 7, 2023.
  6. Kunzmann K. Sotagliflozin Reduces MACE Risk in Diabetic, Kidney Disease Patients in SCORED Trial. HCPLive. Published April 4, 2022. Accessed April 7, 2023.
  7. Bristol Myers Squibb. U.S. Food and Drug Administration (FDA) Accepts Supplemental New Drug Application for CAMZYOS® (mavacamten) in Symptomatic Obstructive Hypertrophic Cardiomyopathy to Reduce the Need for Septal Reduction Therapy. News release. Published October 21, 2022. Accessed April 7, 2023.
  8. Iapoce C. Mavacamten Reduces Eligibility for SRT in Obstructive Hypertrophic Cardiomyopathy. HCPLive. Published April 2, 2022. Accessed April 7, 2023.
  9. Iapoce C. FDA Accepts BLA for Aflibercept 8mg for Priority Review. HCPLive. Published February 24, 2023. Accessed April 7, 2023.
  10. Kunzmann K. Long-Term nAMD Patient Buy-In Is Still Lacking for Anti-VEGF Therapy. HCPLive. Published October 21, 2021. Accessed April 7, 2023.