A Look at Key Specialty Pharmaceuticals in the Pipeline

A session at the AMCP 2025 Annual Meeting outlined the rapidly expanding specialty pharmacy industry, offering important information on therapeutics in development and how they may affect healthcare for clinicians, payers, and policymakers.

This article outlines some key areas of innovation in specialty pharmaceuticals in development.

• Gastroenterology
  • Short bowel syndrome
    • Glepaglutide is a glucagon-like peptide-2 agonist under development for this condition. The FDA declined approval in late December 2024. The phase 3 EASE SBS 1 trial assessed twice-weekly glepaglutide and found that 65.7% of patients achieved a clinical response compared with 38.9% receiving placebo (P=0.0243). The total weekly volume of parenteral support at 24 weeks declined with glepaglutide (P=0.0039).
• Endocrinology and metabolism
  • Fabry disease
    • Isaralgagene civaparvovec is a gene therapy in development for this condition. The phase 1/2 STAAR trial showed a positive mean annualized estimated glomerular filtration rateslope with treatment.
  • Hypercholesterolemia
    • Lerodalcibep is a subcutaneous PCSK9 inhibitor being assessed for its ability to reduce low-density lipoprotein cholesterol (LDL-C) in patients with or at high risk for atherosclerotic cardiovascular disease and primary hyperlipidemia. The phase 3 LIBerate-CVD trial showed a 62% reduction in LDL-C over 52 weeks and a 50.5% reduction in non–high-density lipoprotein cholesterol with lerodalcibep.
• Hematology
  • Hemophilia
    • Denecimig, fitusiran, and giroctocogene fitelparvovec are all under development for hemophilia treatment and are expected to receive regulatory updates in early 2025.
  • Immune thrombocytopenic purpura (ITP)
    • Rilzabrutinib is an oral Bruton tyrosine kinase (BTK) inhibitor in development for ITP, with an expected Prescription Drug User Fee Act (PDUFA) date of late August 2025. The phase 3 LUNA 3 trial showed an increased proportion of patients with durable platelet response after rilzabrutinib treatment (23%) compared with placebo (0%; P<0.0001).
• Neurology
  • Amyotrophic lateral sclerosis (ALS)
    • NP-001, an IV macrophage activation regulator, is under assessment for ALS. The phase 2b North America trial showed that NP-001 did not slow ALS progression. A retrospective pooled analysis of phase 2 trials showed that the treatment increased survival by 4.8 months in the intention-to-treat population.
  • Duchenne muscular dystrophy (DMD)
    • Deramiocel is an IV stem cell therapy for the treatment of DMD cardiomyopathy that has an expected PDUFA date of late August 2025. The phase 3 HOPE-2 open-label extension study observed improvements in cardiac function in deramiocel-treated patients.
  • Multiple sclerosis (MS)
    • Tolebrutinib, an oral BTK inhibitor, is being studied for nonrelapsing secondary progressive MS, with an expected PDUFA of late September 2025. The phase 3 HERCULES trial showed delayed time to onset of 6-month confirmed disability progression by 31% with tolebrutinib compared with placebo (P=0.0026).

Reference Lowe MM. Specialty pharmaceuticals in development 2025. Presented at: AMCP 2025; March 31-April 3, 2025; Houston, TX. Session K3.