Novartis has announced that Rhapsido (remibrutinib) received US Food and Drug Administration (FDA) approval as an oral treatment for adult patients with chronic spontaneous urticaria (CSU) who remain symptomatic despite undergoing H1 antihistamine treatment.
Rhapsido is a pill taken twice daily and does not require injections or lab monitoring.
It is the first FDA-approved Bruton's tyrosine kinase inhibitor (BTKi) for CSU.
Rhapsido helps to inhibit the release of histamine and other proinflammatory mediators by targeting BTK, offering a novel approach to CSU treatment.
Mark Lebwohl, Dean for Clinical Therapeutics at the Icahn School of Medicine at Mount Sinai and member of the steering committee for the remibrutinib REMIX Phase III clinical trial programme, said: "CSU is a serious disease that can cause debilitating symptoms and unpredictable flares."
"It's difficult to diagnose and manage. Remibrutinib represents a new way of treating CSU."
"By blocking the activity of BTK, remibrutinib stops a key pathway of the immune response in CSU. This is an exciting new option that has the potential to help a broad range of patients get fast relief."
CSU is a mast cell-driven condition thought to be caused by immune dysregulation.
In people with CSU, the immune system can become activated through allergic or autoimmune pathways.
This causes certain immune cells (mast cells and basophils) to activate the BTK protein.
Whilst not fully understood yet, it is believed that once activated, BTK leads to the release of histamine and other proinflammatory mediators that may cause the red, swollen and itchy hives commonly seen in CSU.
CSU symptoms are unpredictable, recurring for six weeks or more without an identified cause. Diagnosis can take up to 24 months.
Antihistamines are the first-line treatment, but more than half of patients still have symptoms, even at higher doses. Injectable treatments exist for those who don't respond to antihistamines, yet fewer than 20% of eligible patients receive them.
"The approval of remibrutinib is an important development in CSU care."
"It quickly reduces symptoms, offering patients control of the hives and itching that they experience daily," said Giselle Mosnaim, an Allergist and Immunologist from Endeavor Health, Clinical Associate Professor at the University of Chicago Pritzker School of Medicine and REMIX trial investigator.
"This is significant because it expands beyond existing injectable treatments and gives patients an oral option that can easily be incorporated into their daily lives."
Lynda Mitchell, CEO of Allergy & Asthma Network, added: "Many CSU patients feel misunderstood and settle for treatments that don't fully meet their needs."
"We support new treatment options that empower patients to choose what works best for them."
"This convenient new oral therapy offers a promising new way to manage CSU and potentially improve daily life for those living with this challenging condition."
The FDA approval of Rhapsido in CSU is based on results from the Phase III REMIX-1 and REMIX-2 clinical trials in patients who remained symptomatic on second-generation H1 antihistamines.
Rhapsido demonstrated superiority in change from baseline versus placebo in itch, hives and weekly urticaria activity at Week 12.
Significantly more patients treated with Rhapsido versus placebo achieved well-controlled disease — some as early as Week 2 — and about one-third of patients achieved complete absence of itch and hives at Week 12.
Rhapsido has a demonstrated safety profile that requires no lab monitoring.
The most common adverse events were nasal congestion, sore throat and runny nose (nasopharyngitis), bleeding, headache, nausea and abdominal pain.
Novartis has completed regulatory submissions for Rhapsido for the treatment of CSU across many countries, including in the European Union, Japan and China, with priority review granted in China.
"This approval of Rhapsido as the first and only BTK inhibitor in CSU is an important milestone in our journey to reshape care for overlooked immune-related conditions and offer more patients the potential to find fast relief," said Victor Bultó, President, US, Novartis.
"Building on our legacy in advancing the treatment of allergic, dermatologic and rheumatologic conditions, we are deeply committed to further investing in innovative, patient-focused therapies across immunology."
Discovered and developed by Novartis to target the BTK pathway as a driver of inflammation, remibrutinib is being investigated in ongoing clinical trials across a variety of immune-related conditions.