Allergies, Author Interviews, Dermatology / 06.12.2025
ACAAI: Remibrutinib As Oral option for Patients with Chronic Spontaneous Urticaria who have failed high dose antihistamines
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Dr. Gogate[/caption]
MedicalResearch.com Interview with:
Dr. Shaila Gogate, MD
Colorado Allergy & Asthma Centers
Allergist
REMIX-1/-2 Investigator
MedicalResearch.com: What is the background for this study? What are the main findings?
A: The Phase III REMIX-1/-2 studies were randomized, double-blind, placebo-controlled studies assessing the efficacy and safety of oral remibrutinib 25 mg twice daily or placebo over a 24-week double-blind period, followed by a 28-week open-label remibrutinib treatment period.
The purpose of this pooled analysis was to examine the early and long-term efficacy data of remibrutinib vs. placebo over time, looking at the mean percentage change from baseline (CFB) in weekly scores of disease activity (UAS7), itch severity (ISS7) and hives severity (HSS7) over 52 weeks. This analysis demonstrated that remibrutinib showed reductions in urticaria symptoms vs. placebo as early as week 1:
Dr. Gogate[/caption]
MedicalResearch.com Interview with:
Dr. Shaila Gogate, MD
Colorado Allergy & Asthma Centers
Allergist
REMIX-1/-2 Investigator
MedicalResearch.com: What is the background for this study? What are the main findings?
A: The Phase III REMIX-1/-2 studies were randomized, double-blind, placebo-controlled studies assessing the efficacy and safety of oral remibrutinib 25 mg twice daily or placebo over a 24-week double-blind period, followed by a 28-week open-label remibrutinib treatment period.
The purpose of this pooled analysis was to examine the early and long-term efficacy data of remibrutinib vs. placebo over time, looking at the mean percentage change from baseline (CFB) in weekly scores of disease activity (UAS7), itch severity (ISS7) and hives severity (HSS7) over 52 weeks. This analysis demonstrated that remibrutinib showed reductions in urticaria symptoms vs. placebo as early as week 1:
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- 38.4% vs. 10.3% in UAS7
- 37.0% vs. 9.8% in ISS7
- 39.8% vs. 10.1% HSS7 Greater reductions vs. placebo were seen over 24 weeks and results were observed in the open-label period through week 52 in all remibrutinib-treated patients (including those transitioned from placebo).
Dr. Tauscher-Wisniewski,[/caption]
Sitra Tauscher-Wisniewski, MD
Vice President Clinical Development & Analytics
Novartis Gene Therapies
MedicalResearch.com: What is the background for this study? Would you briefly describe the condition of Spinal muscular atrophy (SMA)?
Response: At the 2023 Muscular Dystrophy Association Conference, we presented new data from two of our Long-Term Follow-Up (LTFU) studies, LT001 and LT002, which show the continued efficacy and durability of Zolgensma across a range of patient populations, with an overall benefit-risk profile that remains favorable. LT001 is a 15-year ongoing observational LTFU study following the Phase 1 START patients, who were the very first patients to receive our gene replacement therapy. LT-002 is a voluntary Phase 4 15-year ongoing follow-up safety and efficacy study of Zolgensma IV and investigational intrathecal (IT) OAV101 in patients previously treated in the Phase 3 IV studies (STR1VE-US, STR1VE-EU, STR1VE-AP, SPR1NT) and the Phase 1 IT study (STRONG).
Spinal muscular atrophy (SMA) is a rare, devastating genetic disease that leads to progressive muscle weakness, paralysis, and when left untreated in one of its most severe forms (SMA Type 1), permanent ventilation or death in 90% of cases by age 2. It is caused by a lack of a functional survival motor neuron 1 (SMN1) gene, and in the most severe forms results in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.