Novartis’ Rhapsido® (remibrutinib) FDA-Approved as First Oral BTK Inhibitor for Chronic Spontaneous Urticaria

Novartis has announced that the U.S. Food and Drug Administration (FDA) has approved Rhapsido® (remibrutinib), marking it as the first oral Bruton's tyrosine kinase inhibitor (BTKi) approved for the treatment of chronic spontaneous urticaria (CSU). This approval provides patients with a targeted oral therapy option that does not require injections or regular laboratory monitoring.

Rhapsido® demonstrated efficacy in clinical trials, with approximately one-third of patients achieving complete symptom relief by week 12. It is taken twice daily and targets the BTK enzyme, which is involved in the inflammatory pathway of CSU.

In addition to the U.S., Novartis has filed for approval in the EU, Japan, and China, with Chinese regulators granting priority review. The global filings reflect Novartis’ strategy to expand access to this first-in-class therapy for patients with CSU worldwide.

The FDA approval of Rhapsido® represents a significant milestone in the management of CSU, a condition that can severely impact quality of life for patients who remain symptomatic despite standard therapies.

FDA-Approved BTK Inhibitors and Their Indications in the U.S.

Bruton's tyrosine kinase inhibitors (BTKis) are a class of medications that target the BTK enzyme, playing a crucial role in B-cell development and function. These inhibitors are primarily used in the treatment of various B-cell malignancies. Below is a list of FDA-approved BTKis and their approved indications in the United States:

1. Ibrutinib (Imbruvica)

• Manufacturer: AbbVie and Janssen Biotech
• Indications:
  • Chronic lymphocytic leukemia (CLL)
  • Small lymphocytic lymphoma (SLL)
  • Mantle cell lymphoma (MCL)
  • Waldenström macroglobulinemia (WM)
  • Marginal zone lymphoma (MZL)
  • Chronic graft-versus-host disease (cGVHD)
  • Primary central nervous system lymphoma (PCNSL)
  • Chronic spontaneous urticaria (CSU) (as of September 2025)

2. Acalabrutinib (Calquence)

• Manufacturer: AstraZeneca
• Indications:
  • Chronic lymphocytic leukemia (CLL)
  • Small lymphocytic lymphoma (SLL)
  • Mantle cell lymphoma (MCL)
  • Chronic graft-versus-host disease (cGVHD)
  • Primary central nervous system lymphoma (PCNSL)

3. Zanubrutinib (Brukinsa)

• Manufacturer: BeiGene
• Indications:
  • Chronic lymphocytic leukemia (CLL)
  • Small lymphocytic lymphoma (SLL)
  • Mantle cell lymphoma (MCL)
  • Chronic graft-versus-host disease (cGVHD)
  • Primary central nervous system lymphoma (PCNSL)

4. Pirtobrutinib (Jaypirca)

• Manufacturer: Loxo Oncology (a subsidiary of Eli Lilly)
• Indications:
  • Chronic lymphocytic leukemia (CLL)
  • Small lymphocytic lymphoma (SLL)
  • Mantle cell lymphoma (MCL)
  • Chronic graft-versus-host disease (cGVHD)
  • Primary central nervous system lymphoma (PCNSL)

5. Remibrutinib (Rhapsido)

• Manufacturer: Novartis
• Indications:
  • Chronic spontaneous urticaria (CSU) (as of September 2025)

These approvals underscore the expanding role of BTK inhibitors in treating a variety of hematologic and immune-mediated conditions. The recent approval of remibrutinib for CSU highlights the potential for BTK inhibitors to address conditions beyond hematologic malignancies, offering new therapeutic options for patients with chronic inflammatory diseases.

Reference:

1. Novartis Press Release, Novartis receives FDA approval for Rhapsido® (remibrutinib), the only oral, targeted BTKi treatment for chronic spontaneous urticaria (CSU), September 30, 2025