Specialty Pharmacy Pipeline Update: Asembia 2025 Highlights
Shifting Trends in Specialty Drug Development
Dr Brad McElya opened the session by highlighting the rapid evolution of the specialty pharmaceutical market, fueled by innovation and a growing demand for advanced therapies. A retrospective review of Center for Drug Evaluation and Research's (CDER’s) Novel Drug Approvals from 2015 to 2024 demonstrated a consistently strong output, with 2024 witnessing the approval of significant products such as Alhemo, Alyftrek, Crenessity, Ebglyss, Hympavzi, Iqirvo, Kisunla, Livdelzi, Nemluvio, Rezdiffra, and Tryvio.
Key therapeutic trends shaping the specialty pipeline include:
- Expansion in rare and orphan diseases
- Breakthroughs in oncology
- Growth of cell and gene therapies
- Innovation in Metabolic dysfunction–associated steatohepatitis (MASH)
- Advances in immunology, neurology, diabetes, obesity management, and biosimilars
Looking ahead to 2025, strategic investment and cutting-edge research are expected to drive further pipeline growth.
Promising Near-Term Pipeline Candidates by Therapeutic Area
Cardiology and Renal Diseases
- Aficamten (Cytokinetics): Oral cardiac myosin inhibitor for obstructive hypertrophic cardiomyopathy (HOCM). Orphan and breakthrough therapy designations; PDUFA date: September 26, 2025.
- Inaxaplin (Vertex): Oral apolipoprotein L1 (APOL1) inhibitor for chronic kidney disease (CKD); breakthrough therapy designation, with an anticipated launch at the end of 2025.
- Atrasentan (Novartis): Oral endothelin A receptor antagonist targeting immunoglobulin A (IgA) nephropathy to preserve kidney function; Now approved as of April 2025.
Pulmonary and Inflammatory Diseases
- Brensocatib (AstraZeneca/Insmed): Oral dipeptidyl peptidase-1 (DPP-1) inhibitor for non-cystic fibrosis bronchiectasis; breakthrough therapy and priority review status, PDUFA date: August 12, 2025.
- BMS-986278 (Bristol Myers Squibb): Oral lysophosphatidic acid receptor 1 (LPA1) antagonist for idiopathic pulmonary fibrosis (IPF); breakthrough therapy designation, PDUFA date not specified.
Immunology and Rare Diseases
- Rilzabrutinib (Sanofi): Oral Bruton's tyrosine kinase (BTK) inhibitor for idiopathic thrombocytopenic purpura; orphan and fast track status, PDUFA date: August 29, 2025.
- Depemokimab (GSK): Injection therapy for asthma and chronic rhinosinusitis with nasal polyps, PDUFA target: December 16, 2025.
- Dapirolizumab (UCB): Injectable anti-CD40L monoclonal antibody for systemic lupus erythematosus, PDUFA date: not specified.
Neurology
- Tolebrutinib (Sanofi): Oral BTK inhibitor for multiple sclerosis; breakthrough and priority review designations, PEDUFA date: September 28, 2025.
- Latozinemab (GSK/Alector): Injection for frontotemporal dementia; orphan, breakthrough therapy, and fast track designations, PDUFA date: not specified.
Rare Diseases
- Elamipretide (Stealth Biotherapeutics): Injectable mitochondrial therapy for Barth syndrome and cardiomyopathy; first-in-class mitochondria-targeted therapeutic, PDUFA date: April 29, 2025.
- Anavex 2-73 (Anavex Life Sciences): Oral agent for Rett syndrome, Alzheimer's, and Parkinson disease dementia; orphan, fast track, and rare pediatric disease designations, PDUFA date: not specified.
- Sebetralstat (KalVista Pharma): Oral plasma kallikrein inhibitor for hereditary angioedema (HAE), first oral on-demand therapy in this space, PDUFA target: June 17, 2025.
- Batoclimab (Roivant/Immunovant): Injection for Graves' disease and Myasthenia Gravis, PDUFA date: not specified.
Oncology Pipeline Overview
Cancer incidence is projected to rise to approximately 2 million new cases in 2025, with breast, prostate, lung, colon, and rectal cancers making up half of all diagnoses. Notable oncology pipeline updates include:
- Bemarituzumab (Amgen): First-in-class FGFR2b monoclonal antibody for stomach cancer; breakthrough therapy designation, PDUFA date: not specified.
- Camizestrant (AstraZeneca): Oral selective estrogen receptor degrader for breast cancer; fast track status, PDUFA date: not specified.
- Zimberelimab (Gilead): Injectable anti-program cell death protein 1 (anti-PD-1) monoclonal antibody targeting non-small cell lung cancer (NSCLC) and upper gastrointestinal cancers, PDUFA date: not specified.
- Dorrdaviprone (Chimerix Inc): Oral agent for recurrent HC K27M–mutant diffuse glioma (rare brain cancer); rare pediatric disease designation; PDUFA date: August 18, 2025.
- Domvanalimab (Arcus Biosciences): Injectable T-cell immunoreceptor with immunoglobulin (Ig) and immunoreceptor tyrosine-based inhibitory motif (ITIM) domains (TIGIT) (anti-TIGIT) monoclonal antibody for NSCLC and upper GI cancers, with Phase 3 data expected in 2026.
- RMC-6236 (Revolution Medicines): Oral therapy for pancreatic ductal adenocarcinoma demonstrating promising clinical activity.
- Zanzalintinib (Exelixis): Oral multi-tyrosine kinase inhibitor for colorectal and renal cell carcinomas, PDUFA target: April 3, 2025.
- Mezigdomide (Bristol Myers Squibb): Oral cereblon modulator for multiple myeloma; orphan drug designation, PDUFA date: not specified.
- Tipifarnib (Kura Oncology): Oral farnesyltransferase inhibitor for head and neck cancers; breakthrough therapy and fast track status, PDUFA date: not specified.
The Bottom Line
The specialty pharmacy pipeline entering 2025 is vibrant, with innovation spanning a broad range of therapeutic areas. The year ahead promises the launch of several first-in-class and best-in-class therapies, particularly in rare disease and oncology, offering payers and clinicians both opportunities and challenges as they plan for integration into clinical pathways and formularies.
Reference
McElya B. Specialty pharmacy pipeline. Presented at: Asembia 2025; April 28, 2025; Las Vegas, NV.
