UCB’s rozanolixizumab received priority review on its biologic license application by FDA and expects feedback during the second quarter of 2023.
UCB Pharma announced that the FDA has granted priority review for the biologic license application (BLA) for its agent rozanolixizumab), a potential treatment for adults with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.1 If the FDA approves the review, it could deliver significant improvements for the safety and effectiveness of the treatment, diagnosis, or preventative care for this patient population.
The application is based on data from the phase 3 MycarinG study (NCT03971422) which showed that rozanolixizumab significantly reduced Myasthenia Gravis-Activities of Daily Living (MG-ADL) score (P < .001) over a 43-day period. Notably, rozanolixizumab 7-mg/kg and 10-mg/kg had a least-square mean change on MG-ADL of –3.370 (difference vs placebo, –2.586; 95% CI, –4.091 to –1.249) and –3.403 (difference vs placebo, –2.619; 95% CI, –3.994 to –1.163) compared with –0.784 for those on placebo.
“People living with MG suffer from unpredictable, fluctuating, and debilitating symptoms that have a huge impact on their lives, and there is a clear need for additional targeted treatments. We are firmly committed to supporting the gMG community by providing solutions to help improve outcomes for patients and reduce the day-to-day burden of the disease,” said Charl van Zyl, executive vice president, Neurology Solutions, and head of EU/International Markets, UCB said in a statement.1 “The FDA’s decision to assess rozanolixizumab via their priority review process, as well as the recent filing of the MAA in Europe, brings us important steps further on our journey towards approvals for rozanolixizumab. We look forward to working with the FDA and EMA to help bring this new treatment option to patients.”
Two hundred patients were randomized 1:1:1 in the MycarinG study and given weekly rozanolixizumab 7 mg/kg (n = 66), 10 mg/kg (n = 67) or placebo (n = 67) for 6 weeks,followed with an observation period for 8 weeks. In the active treatment arm group, a higher proportion of treatment emergent adverse events (TEAEs) occurred in comparison with placebo (81.3% for 7 mg/kg, 82.6% for 10 mg/kg and 67.2% for placebo). Reports of the most common TEAEs were headache, diarrhea, pyrexia, and nausea; although, a higher incidence of headache was observed in the rozanolixizumab groups versus placebo.
“Patients living with MG may experience high disease and treatment burden resulting in a significant impact on their daily lives. If approved, rozanolixizumab has the potential to address unmet needs of gMG patients,” said Iris Loew-Friedrich, PhD, executive vice president and chief medical officer, UCB said in a statement.1 “Through rozanolixizumab and zilucoplan, we intend to bring two medicines with different mechanisms of action that have the potential to provide targeted treatment options to patients. With our gMG pipeline, we hope to address both drivers of disease pathology and which account for approximately 95% of people living with gMG. Priority Review Designation by the FDA for rozanolixizumab reflects the extent to which our science speaks for itself in potentially addressing the significant unmet needs still faced by the gMG community.”
Findings from MycarinG were further presented at the 2022 American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) annual meeting, September 21-24, in Nashville, Tennessee.2 Treatment response, considered improvement of at least 2 points on MG-ADL, and 3-point improvement on both Quantitative Myasthenia Gravis (QMG) and Myasthenia Gravis Composite (MGC) scores, was assessed. There, rozanolixizumab-treated patients continued to showed a better response on all outcomes relative to placebo (MG-ADL, 71.9% and 69.4% in 7 mg/kg and 10 mg/kg groups; placebo, 31.3%).
The QMG scores showed an achieved response in the rozanolixizumab 7-mg/kg, 10-mg/kg, and placebo groups (54.7%, 72.6%, and 39.1%). Similarly, the same respective groups achieved response on MGC (60.9%, 74.2%, and 40.6%). Only 3.0% in the placebo group had minimal symptom expression in comparison with the 7-mg/kg and 10-mg/kg active treatment groups, respectively (25.8% and 28.4%).