Q2 2025: 10 FDA Decisions to Watch

The first quarter of 2024 set a high bar for drug approvals, with the US Food and Drug Administration (FDA) greenlighting several groundbreaking therapies. Suzetrigine (Journavx), a nonopioid pain reliever, broke a decades-long drought in FDA approvals for this critical area of unmet need. Meanwhile, semaglutide (Ozempic) expanded its reach, earning an indication for chronic kidney disease in type 2 diabetes, and esketamine (Spravato) made history as the first monotherapy for treatment-resistant depression.

As the medical community prepares to enter Q2, all eyes are on the FDA’s next moves. With major regulatory decisions expected across multiple therapeutic areas, the coming months could bring new options for patients and shift treatment paradigms once again. Here is HCPLive’s curated list of 10 decisions to watch for Q2 2025.

Q2 2025 FDA Decisions to Watch

1. Prademagene zamikeracel (pz-cel)

Indication: Recessive Dystrophic Epidermolysis Bullosa
Projected Action Date: April 29, 2025
Background Info:
On October 29, 2024, Abeona Therapeutics announced the resubmission of the Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) for recessive dystrophic epidermolysis bullosa (RDEB). On November 13, 2024, the FDA accepted the BLA and set a target action date of April 29, 2025.

The resubmission follows an earlier complete response letter requesting additional chemistry, manufacturing, and controls data, which Abeona addressed in alignment with the FDA. According to clinical trial data, pz-cel met primary efficacy endpoints in the phase 3 VIITAL study, demonstrating significant wound healing and pain reduction. If approved, pz-cel would become the second gene therapy for RDEB.

2. Sebetralstat

Indication: Hereditary Angioedema
Projected Action Date: June 17, 2025
Background Info:
On September 2024, KalVista Pharmaceuticals announced the FDA’s acceptance of its New Drug Application (NDA) for sebetralstat for the on-demand treatment of hereditary angioedema (HAE) attacks in patients aged 12 and older. The FDA set a PDUFA target action date of June 17, 2025.

Data from the phase 3 KONFIDENT trials showed that oral sebetralstat provided rapid symptom relief, with a median time to initial relief of 1.79 hours. According to researchers, the therapy could significantly reduce treatment burden by offering an oral alternative to existing injectable options. If approved, sebetralstat would become the first oral on-demand treatment for HAE, potentially transforming disease management.

3. Dupilumab

Indication: Bullous Pemphigoid
Projected Action Date: June 20, 2025
Background Info:
In February 2025, Regeneron and Sanofi announced the FDA’s acceptance of their sBLA for dupilumab (Dupixent) for bullous pemphigoid for priority review, assigning a PDUFA date of June 20, 2025.

New data from the LIBERTY-BP ADEPT trial, presented at the 2025 AAD Annual Meeting, showed dupilumab significantly improved disease remission, reduced severity, and lowered corticosteroid exposure. The trial found that 20% of patients on dupilumab achieved sustained remission versus 4% on placebo, with notable reductions in itch and flare-ups. According to investigators, dupilumab could become the first targeted biologic for bullous pemphigoid, addressing a major unmet need. If approved, this would mark another expansion for dupilumab, which is already approved for multiple inflammatory conditions.

4. Clesrovimab

Indication: Respiratory Syncytial Virus
Projected Action Date: June 10, 2025
Background Info:
On December 17, 2024, Merck announced the FDA’s acceptance of the BLA for clesrovimab, a long-acting monoclonal antibody for preventing respiratory syncytial virus (RSV) in infants. The FDA set a PDUFA date of June 10, 2025.

The application is supported by data from the Phase 2b/3 CLEVER trial, which showed clesrovimab reduced RSV-associated medically attended lower respiratory infections through day 150 after a single dose. According to Merck, clesrovimab is designed for all infants regardless of weight, offering season-long protection. If approved, it would be available for ordering by July 2025, ahead of the 2025 RSV season.

5. Lenacapavir

Indication: HIV Prevention
Projected Action Date: June 19, 2025
Background Info:
On February 18, 2025, Gilead announced the FDA accepted its NDA submissions for lenacapavir (Sunlenca) as a twice-yearly PrEP for HIV prevention. The FDA has assigned a priority review with a target action date of June 19, 2025.

Based on data from the phase 3 PURPOSE 1 and PURPOSE 2 trials, lenacapavir demonstrated near-total risk reduction in preventing HIV infection. According to Gilead, if approved, it would be the first and only twice-yearly PrEP option. The company has also submitted applications to the European Medicines Agency to expand global access, including in low-income regions.

6. Reproxalap

Indication: Dry Eye Disease
Projected Action Date: April 02, 2025
Background Info:
On October 3, 2024, Aldeyra Therapeutics announced the FDA accepted its resubmitted NDA for reproxalap, a topical ocular treatment for dry eye disease (DED).

Based on positive Phase 3 trial data requested by the FDA, the resubmission demonstrated acute and chronic improvements in dry eye symptoms and ocular redness. According to Aldeyra, reproxalap could become the first chronic-use DED therapy with proven rapid symptom relief.

7. Ketamine

Indication: Multiple Indications
Projected Action Date: June 04, 2025
Background Info:

On March 11, 2025, PharmaTher announced a new FDA goal date of June 4, 2025, for ketamine (Ketarx) in anesthesia, sedation, pain, mental health, and neurological indications. Based on an Amendment Acknowledgment Letter following a resubmission to address minor deficiencies, the FDA did not request new clinical trials.

According to PharmaTher, this approval could help resolve the ongoing ketamine shortage, which has been on the FDA’s drug shortage list since 2018. The shortage has led to increased use of compounded ketamine products, raising safety concerns. If approved, PharmaTher expects a transition period like that of semaglutide and other GLP-1 RAs when they were removed from the shortage list.

8. Elamipretide

Indication: Barth Syndrome
Projected Action Date: April 29, 2025
Background Info:
On January 23, 2025, Stealth BioTherapeutics announced the FDA extended the PDUFA action date for elamipretide, a mitochondria-targeted therapy for Barth syndrome, to April 29, 2025. According to the company, the FDA required more time to review additional data submitted after a positive advisory committee meeting but had not identified safety concerns or requested new studies.

According to Stealth, elamipretide could become the first FDA-approved therapy for Barth syndrome, a rare and life-threatening genetic disorder. The drug holds multiple FDA designations, including Orphan Drug and Priority Review.

9. Roflumilast Foam 0.3%

Indication: Head and Scalp Psoriasis
Projected Action Date: May 22, 2025
Background Info:
On September 24, 2024, Arcutis Biotherapeutics announced the FDA accepted its sNDA for roflumilast (Zoryve) foam 0.3% to treat scalp and body psoriasis in patients 12 and older. The FDA has set a PDUFA target action date of May 22, 2025.

Based on data from the phase 3 ARRECTOR trial, the foam demonstrated significant improvements in psoriasis severity, itch reduction, and rapid relief compared to a vehicle. According to Arcutis, the unique formulation of roflumilast foam 0.3%’s offers a potential new option for patients struggling with hard-to-treat scalp psoriasis. If approved, it would be the company’s fifth topical roflumilast submission in under 3 years.

10. Dupilumab

Indication: Chronic Spontaneous Urticaria
Projected Action Date: April 18, 2025
Background Info:
On November 15, 2024, Regeneron Pharmaceuticals and Sanofi announced the FDA accepted their resubmitted sBLA for dupilumab to treat chronic spontaneous urticaria (CSU) in patients 12 and older, with a target action date of April 18, 2025.

Based on data from the phase 3 LIBERTY-CUPID clinical program, the resubmission includes findings from 3 studies demonstrating significant reductions in itch and hives. According to Sanofi, dupilumab offers a potential new option for patients whose CSU is inadequately managed with antihistamines. The drug is already approved for CSU in the UAE and Japan.

References:

1. Smith T. FDA accepts resubmission of PZ-cel Gene Therapy Bla for epidermolysis bullosa. HCP Live. November 13, 2024. Accessed March 26, 2025. https://www.hcplive.com/view/fda-accepts-resubmission-pz-cel-gene-therapy-bla-epidermolysis-bullosa.

2. KalVista Pharmaceuticals. Kalvista announces FDA acceptance of New Drug Application for SEBETRALSTAT for oral on-demand treatment of hereditary angioedema. KalVista Pharmaceuticals. September 3, 2024. Accessed March 26, 2025. https://ir.kalvista.com/news-releases/news-release-details/kalvista-announces-fda-acceptance-new-drug-application.

3. Campbell P. Dupilumab proves benefit in bullous pemphigoid ahead of June 2025 FDA decision. HCP Live. March 8, 2025. Accessed March 26, 2025. https://www.hcplive.com/view/dupilumab-proves-benefit-in-bullous-pemphigoid-ahead-of-june-2025-fda-decision.

4. Parkinson J. FDA accepts bla for Merck’s monoclonal antibody for infant protection against RSV. ContagionLive. December 17, 2024. Accessed March 26, 2025. https://www.contagionlive.com/view/fda-accepts-bla-for-merck-s-monoclonal-antibody-for-infant-protection-against-rsv.

5. Parkinson J. FDA sets Pdufa date for Lenacapavir for HIV Prevention. ContagionLive. February 18, 2025. Accessed March 26, 2025. https://www.contagionlive.com/view/fda-sets-pdufa-date-for-lenacapavir-for-hiv-prevention.

6. Iapoce C. Aldeyra resubmits new drug application for REPROXALAP for dry eye disease. HCP Live. October 3, 2024. Accessed March 26, 2025. https://www.hcplive.com/view/aldeyra-resubmits-new-drug-application-reproxalap-dry-eye-disease.

7. Derman C. Pharmather receives new early June FDA goal date for ketamine. HCP Live. March 11, 2025. Accessed March 26, 2025. https://www.hcplive.com/view/pharmather-receives-new-early-june-fda-goal-date-for-ketamine.

8. Stealth Bio TherapeuticsInc. Stealth Biotherapeutics announces PDUFA action date extension for Elamipretide to treat patients with barth syndrome. Stealth BioTherapeutics Announces PDUFA Action Date Extension for Elamipretide to Treat Patients with Barth Syndrome. January 23, 2025. Accessed March 26, 2025. https://www.prnewswire.com/news-releases/stealth-biotherapeutics-announces-pdufa-action-date-extension-for-elamipretide-to-treat-patients-with-barth-syndrome-302358052.html.

9. Iapoce C. FDA accepts sNDA for roflumilast foam as scalp, body psoriasis treatment. HCP Live. September 24, 2024. Accessed March 26, 2025. https://www.hcplive.com/view/fda-accepts-snda-roflumilast-foam-scalp-body-psoriasis-treatment.

10. Smith T. FDA accepts resubmitted sbla for dupilumab treatment of chronic spontaneous urticaria. HCP Live. November 15, 2024. Accessed March 26, 2025. https://www.hcplive.com/view/fda-accepts-resubmitted-sbla-dupilumab-treatment-chronic-spontaneous-urticaria.