The rejection appears to be related to pre-approval inspection issues that need to be resolved before it can be approved.
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It won’t be a happy weekend for UCB as the U.S. Food and Drug Administration issued a Complete Response Letter (CRL) to the Belgium-based company regarding its Biologics License Application (BLA) for bimekizumab for adults with moderate to severe plaque psoriasis. The rejection appears to be related to pre-approval inspection issues that need to be resolved before the drug can be approved.
Bimekizumab was approved in August 2021 in the U.K. and the European Union and in January 2022 in Japan for plaque psoriasis, generalized pustular psoriasis and psoriatic erythroderma in patients who don’t respond to existing treatments. It was also approved in Canada in February and in Australia in March.
Psoriasis is a chronic inflammatory disease of the skin. Bimekizumab is a humanized monoclonal IgG1 antibody that selectively inhibits interleukin 17A (IL-17A) and interleukin 17F (IL-17F), both of which are cytokines that drive inflammation.
Company stock dropped 12% at the news. UCB indicated it was reviewing its financial guidance for the year in the letter’s context. It had previously projected 2022 revenue ranging from $5.35 billion to $5.6 billion.
Jean-Jacques Le Fur, an analyst with Bryan Garnier, wrote to investors that the CRL might be related to an inspection of a Belgium manufacturing facility in the first quarter of the year. If that’s the case, the only risk for the company would be an additional delay because there is no apparent reference to clinical issues.
Earlier this week, UCB presented results from two Phase III trials of zilucoplan and rozanolixizumab in adults with generalized myasthenia gravis (gMG). Zilucopan is a self-administered, subcutaneous peptide inhibitor of complement component 5 (C5 inhibitor). Rozanolixizumab is a subcutaneous-infused monoclonal antibody targeting the neonatal Fc receptor (FcRn).
The Phase III RAISE trial demonstrated that zilucoplan resulted in clinically meaningful and statistically significant improvements in key gMG-specific outcomes compared with placebo in patients with AChR+ gMG.
“The results from the RAISE study are an exciting development in the gMG treatment paradigm and reinforce the critical role that complement inhibition could play for physicians treating patients with this debilitating illness,” said Dr. James F. Howard, M.D., distinguished professor of neuromuscular disease, professor of neurology, medicine and Allied Health at The University of North Carolina at Chapel Hill School of Medicine and lead investigator in the RAISE trial.
The Phase III MycarinG trial demonstrated that rozanolixizumab significantly reduced MG-ADL from baseline to Day 43 compared to placebo in patients with AChR or muscle-specific tyrosine kinase (MuSK) autoantibody-positive gMG. It demonstrated a placebo-corrected mean improvement of 2.586 points at the ~7 mg/kg dose and 2.619 points in the MG-ADL at the ~10m mg/kg dose compared to placebo.
“The one constant in gMG is unpredictability,” said Vera Bril, professor of medicine (neurology), University of Toronto, director of the neuromuscular section, division of neurology, University of Toronto and University Health Network, Toronto, and lead investigator of the MycarinG study.
“People living with this disease experience symptoms that are nebulous, fluctuating, and which vary from one day to the next. For this reason, there is an urgent need for more effective, targeted, and convenient treatments that reduce the burden of disease on patients’ daily lives. The results from the MycarinG study are extremely encouraging, and demonstrate the potential of rozanolixizumab as a new, effective and flexible treatment option to help ease the day-to-day burden of this challenging disease and improve treatment outcomes for patients.”
