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 unique approach to CSU treatment.1
"CSU is a serious disease that can cause debilitating symptoms and unpredictable flares. It's difficult to diagnose and manage," said Mark Lebwohl, MD, 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 program. "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 (IgE) or autoimmune (IgG) pathways.4 This causes certain immune cells—mast cells and basophils—to activate the BTK protein. While not fully understood, 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.5,6
CSU symptoms are unpredictable, recurring for six weeks or more without an identified cause.7 Diagnosis can take up to 24 months.8 Many CSU patients say their symptoms negatively impact their sleep, work, and mental health.9,10,11 Antihistamines are the first-line treatment, but over half of patients still have symptoms, even at higher doses.2 Injectable treatments exist for those who don't respond to antihistamines, yet fewer than 20% of eligible patients receive them.12
"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 on a daily basis," said Giselle Mosnaim, MD, MS, 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."
"Many CSU patients feel misunderstood and settle for treatments that don't fully meet their needs," said Lynda Mitchell, CEO of Allergy & Asthma Network. "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."
Clinical data supporting approval
The FDA approval of Rhapsido in CSU is based on results from the Phase III REMIX-1 (NCT05030311) and REMIX-2 (NCT05032157) clinical trials in patients who remained symptomatic on second-generation H1 antihistamines. Rhapsido demonstrated superiority in change from baseline versus placebo in itch (ISS7), hives (HSS7), and weekly urticaria activity (UAS7) at Week 12.13 Significantly more patients treated with Rhapsido versus placebo achieved well-controlled disease (UAS7≤6) as early as Week 2 and at Week 12, and about one-third of patients achieved complete absence of itch and hives at Week 12.13 Rhapsido has a demonstrated safety profile that requires no lab monitoring.13 The most common adverse events (incidence ≥3%) were nasal congestion, sore throat, and runny nose (nasopharyngitis), bleeding, headache, nausea, and abdominal pain.13
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.
Transforming care in Immunology
"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, including chronic inducible urticaria (CIndU), hidradenitis suppurativa (HS), and food allergy.
RHAPSIDO® (remibrutinib) Indication
RHAPSIDO is a prescription medicine used to treat adults with chronic spontaneous urticaria (CSU) who continue to have symptoms that are not controlled with antihistamine treatment. RHAPSIDO should not be used to treat any other forms of hives (urticaria). It is not known if RHAPSIDO is safe and effective in children.
RHAPSIDO Important Safety Information
Before taking RHAPSIDO, patients should tell their health care provider about all of their medical conditions, including if they:
- Have had a recent surgery or plan to have surgery. Health care providers may tell patients to stop taking RHAPSIDO for at least 3 to 7 days before and after any planned medical or surgical procedures
- Have bleeding problems or are taking a blood thinner medicine
- Have liver problems
- Are pregnant or plan to become pregnant. It is not known if RHAPSIDO will harm an unborn baby. If patients become pregnant during treatment with RHAPSIDO, they should talk to their health care provider about registering in the pregnancy exposure registry for RHAPSIDO
- Are breastfeeding or plan to breastfeed. It is not known if RHAPSIDO passes into breast milk
- Have recently received or are scheduled to receive an immunization (vaccine). Patients should talk to their health care provider about receiving any immunizations before taking RHAPSIDO. They should not receive live or live-attenuated vaccines during treatment with RHAPSIDO
Patients should tell their health care provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking RHAPSIDO with certain other medications may affect how RHAPSIDO or other medications work, and can cause side effects.
RHAPSIDO may cause serious side effects, including the risk of bleeding. Bleeding may happen while being treated with RHAPSIDO. Patients' health care providers will monitor them for signs and symptoms of bleeding and may stop their treatment if bleeding happens. Patients' risk of bleeding may increase if they are also taking a blood thinner medicine. Patients should tell their health care provider right away if they get any signs or symptoms of bleeding, such as: bruising or red or purple skin marks; pink or brown urine; headache, dizziness, confusion, or feeling weak; red or black stools that look like tar.
The most common side effects with RHAPSIDO may include nasal congestion, sore throat, and runny nose (nasopharyngitis), bleeding, headache, nausea, and abdominal pain.
About Novartis
Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people's lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach nearly 300 million people worldwide.
For more information please visit, https://www.novartis.us
