Q1 2026 Preview: 5 FDA Decisions to Watch in Specialty Care

Over the past several years, specialty medicine has entered a period of sustained acceleration, characterized by an expanding volume of late-stage clinical data, an increasing number of regulatory submissions, and a growing diversity of therapeutic mechanisms reaching the US Food and Drug Administration (FDA).

Advances that once unfolded gradually are now arriving in rapid succession, requiring clinicians, researchers, and health system leaders to reassess standards of care across multiple disease areas continuously. For many in the medical community, keeping pace with this evolution has become an ongoing challenge, as meaningful regulatory decisions and practice-shaping data emerge with increasing frequency.

This momentum is expected to carry forward into early 2026, with the first quarter shaping up to be particularly active across several specialty fields. Multiple FDA decisions anticipated during this period have the potential to introduce first-in-class therapies, expand treatment options for conditions with long-standing unmet need, and further refine regulatory expectations around clinically meaningful endpoints. In this article, we highlight 5 FDA decisions to watch in the coming months, each representing a potential inflection point in its respective disease area. Together, these decisions, listed below in chronological order, underscore both the opportunities and the complexity facing specialty care as therapeutic innovation continues to accelerate.

5 FDA Decisions to Watch in Q1 2026

1. Sparsentan

Company: Travere Therapeutics
PDUFA date: January 13, 2026
Indication: Focal Segmental Glomerulosclerosis
Summary: A decision on sparsentan (Filspari) is expected from the US FDA by January 13, 2026. Sparsentan, a dual endothelin A receptor and angiotensin II type 1 receptor antagonist from Travere Therapeutics, was assessed in the phase 3 DUPLEX Study and phase 2 DUET Study, which demonstrated rapid, sustained reductions in proteinuria compared with maximally dosed irbesartan. The FDA accepted Travere’s supplemental New Drug Application for traditional approval in May 2025, initially indicating plans for an advisory committee meeting, which the agency later determined was no longer necessary as of September 10, 2025. If approved, sparsentan would become the first FDA-approved therapy specifically indicated for focal segmental glomerulosclerosis (FSGS).

Why It Matters: FSGS remains a leading cause of kidney failure with no approved disease-specific therapies, leaving clinicians reliant on off-label and often poorly tolerated options. Approval of sparsentan would establish proteinuria reduction as a validated regulatory endpoint in FSGS and provide a non-immunosuppressive, oral treatment option for both adult and pediatric patients at risk of progression.

Expert Perspective: Understanding Sparsentan in Pediatric FSGS, With Michelle Rheault, MD

2. Sublingual Epinephrine Film (Anaphlym)

Company: Aquestive Therapeutics
PDUFA date: January 31, 2026
Indication: Type 1 Allergic Reactions
Summary: A decision on epinephrine sublingual film (Anaphylm) is expected from the US FDA by January 31, 2026. Epinephrine sublingual film, a polymer matrix–based epinephrine prodrug delivered via sublingual film from Aquestive Therapeutics, was assessed in an Oral Allergy Syndrome challenge study and supporting adult and pediatric pharmacokinetic and safety studies, which demonstrated rapid symptom resolution beginning within 2 minutes of administration and pharmacokinetic profiles comparable to traditional epinephrine exposure. The FDA accepted the New Drug Application on June 16, 2025, following positive pre-NDA feedback in December 2024 and completion of a pediatric study announced in April 2025, and assigned a PDUFA target action date of January 31, 2026.

Why It Matters: Anaphylaxis remains a life-threatening condition in which delayed or avoided epinephrine administration is a major contributor to morbidity and mortality, often driven by fear of needles and poor device adherence. Approval of epinephrine sublingual film could alter emergency allergy care by introducing the first orally delivered, device-free epinephrine option, potentially improving real-world access, carriage, and timely treatment across pediatric and adult populations.
Expert Perspective: Sublingual Epinephrine Film Well Tolerated in Children, With David Golden, MD

3. Navepegritide (TransCon CNP)

Company: Ascendis Pharma
PDUFA date: February 28, 2026
Indication: Achondroplasia
Summary: A decision on TransCon CNP (navepegritide) is expected from the US FDA by February 28, 2026, following a 3-month PDUFA extension announced November 25, 2025, due to a major amendment submitted on November 5, 2025, related to post-marketing requirements. TransCon CNP, a once-weekly investigational prodrug of C-type natriuretic peptide (CNP) from Ascendis Pharma, was assessed in the pivotal phase 3 ApproaCH trial and additional randomized, placebo-controlled studies, which demonstrated increased growth velocity alongside improvements in muscle strength, leg bowing, and health-related burden with a safety profile comparable to placebo. The New Drug Application was originally submitted in 2025, and Ascendis is also planning submission of a European Marketing Authorisation Application in Q3 2025. If approved, TransCon CNP would expand the therapeutic landscape for pediatric achondroplasia with a long-acting, systemic CNP replacement strategy.

Why It Matters: Achondroplasia is a multisystem genetic condition associated with lifelong skeletal, neurologic, respiratory, and functional complications, and current treatment options remain limited. Approval of TransCon CNP could represent a shift toward disease-modifying therapy that addresses not only linear growth but also broader functional and quality-of-life outcomes in affected children.
Expert Perspective: The Latest on Navepegritide for Children with Achondroplasia, with Carlos Bacino, MD

4. Deucravacitinib

Company: Bristol Myers Squibb
PDUFA date: March 6, 2026
Indication: Psoriatic Arthritis
Summary: A decision on deucravacitinib (Sotyktu) is expected from the US FDA by March 6, 2026. Deucravacitinib, a selective oral tyrosine kinase 2 (TYK2) inhibitor from Bristol Myers Squibb, was assessed in the phase 3 POETYK PsA-1 and POETYK PsA-2 trials, which demonstrated statistically significant improvements in ACR20 response at week 16 versus placebo, along with favorable skin, functional, and quality-of-life outcomes. The FDA accepted the supplemental New Drug Application in July 2025. If approved, deucravacitinib would become the first TYK2 inhibitor indicated for psoriatic arthritis, expanding on its September 2022 FDA approval for moderate-to-severe plaque psoriasis.

Why It Matters: Psoriatic arthritis remains a heterogeneous, progressive disease with persistent unmet need for effective oral therapies that balance efficacy with long-term safety. Approval of deucravacitinib could introduce a differentiated oral mechanism distinct from JAK inhibition, offering clinicians an earlier-line systemic option with a safety profile that may be better suited for long-term disease management.
Expert Perspective: Deucravacitinib, Zasocitinib Validate TYK2 Inhibition in PsA, With Philip Mease, MD

5. Linerixibat

Company: GSK
PDUFA date: March 24, 2026
Indication: Cholestatic Pruritus in Primary Biliary Cholangitis
Summary: A decision on linerixibat is expected from the US FDA by March 24, 2026. Linerixibat, an ileal bile acid transporter (IBAT) inhibitor from GSK, was assessed in the phase 3 GLISTEN trial, demonstrating clinically meaningful reductions in moderate to severe cholestatic pruritus and associated quality-of-life burden in patients with primary biliary cholangitis (PBC). The FDA accepted the New Drug Application in June 2025, positioning linerixibat as a potential symptom-directed therapy amid a rapidly evolving PBC treatment landscape following the 2024–2025 approvals of seladelpar and elafibranor. If approved, linerixibat would be the first therapy specifically indicated for PBC-associated pruritus in the United States, according to GSK.

Why It Matters: Despite recent disease-modifying approvals, up to half of patients with PBC continue to experience moderate to severe pruritus, a symptom strongly linked to impaired sleep, mental health, and overall quality of life. A targeted antipruritic option such as linerixibat could address a critical residual unmet need that remains largely refractory to current PBC therapies.
Expert Perspective: GLISTEN: Linerixibat Improves Biomarkers and Itch in Patients With PBC, With Andreas Kremer, MD, PhD, MHBA

References:

1. Travere Therapeutics. Travere Therapeutics Provides Update on FDA Advisory Committee Meeting for FILSPARI® (sparsentan) in FSGS. September 10, 2025. Accessed September 10, 2025. https://ir.travere.com/press-releases/news-details/2025/Travere-Therapeutics-Provides-Update-on-FDA-Advisory-Committee-Meeting-for-FILSPARI-sparsentan-in-FSGS/default.aspx

2. Aquestive Therapeutics. Aquestive Therapeutics Announces FDA Acceptance of New Drug Application and PDUFA Date for Anaphylm™ for the Treatment of Severe Allergic Reactions. June 16, 2025. Accessed June 19, 2025. https://investors.aquestive.com/news-releases/news-release-details/aquestive-therapeutics-announces-fda-acceptance-new-drug-0

3. Ascendis Pharma. Ascendis Announces Extension of FDA Review Period for TransCon CNP (navepegritide) for Children with Achondroplasia | Ascendis Pharma. Ascendispharma.com. Published November 25, 2025. Accessed December 24, 2025. https://investors.ascendispharma.com/news-releases/news-release-details/ascendis-announces-extension-fda-review-period-transcon-cnp

4. Bristol Myers Squibb’s Supplemental New Drug Application (sNDA) for Sotyktu (deucravacitinib) for the Treatment of Adults with Active Psoriatic Arthritis Accepted for Review Across Four Regions Globally. News release. Bristol Myers Squibb. July 21, 2025. https://news.bms.com/news/details/2025/Bristol-Myers-Squibbs-Supplemental-New-Drug-Application-sNDA-for-Sotyktu-deucravacitinib-for-the-Treatment-of-Adults-with-Active-Psoriatic-Arthritis-Accepted-for-Review-Across-Four-Regions-Globally/default.aspx

5. GSK. Linerixibat New Drug Application (NDA) accepted for review by the US FDA for cholestatic pruritus in patients with primary biliary cholangitis (PBC) | GSK. Gsk.com. Published June 2, 2025. Accessed December 24, 2025. https://www.gsk.com/en-gb/media/press-releases/linerixibat-new-drug-application-nda-accepted-for-review-by-the-us-fda