Regeneron scores US nod for Veopoz to treat ultra-rare CHAPLE disease
2023-08-18
上市批准临床结果优先审批临床3期
The FDA on Friday approved Regeneron Pharmaceuticals' C5 antibody Veopoz (pozelimab-bbfg) for adult and paediatric patients ages one year and older with CHAPLE disease, making it the first therapy indicated specifically for the ultra-rare hereditary syndrome. The company noted that the decision also addresses some pre-approval inspection issues that recently scuttled its high-dose Eylea (aflibercept) application, and paves the way for FDA action on that filing "in the next few weeks."
The FDA submission for Veopoz, which was granted a priority review in February, was based on data from an open-label Phase II/III trial involving 10 patients ages three to 19 years old. CHAPLE disease, also referred to as CD55-deficient protein-losing enteropathyCD55-deficient protein-losing enteropathy, can cause potentially life-threatening gastrointestinal and cardiovascular symptoms.
Participants were given a single loading dose of Veopoz intravenously on the first day, followed by subcutaneous weekly weight-based doses. Regeneron said all the patients achieved normalisation of serum albumin and serum IgG concentrations by week 12, and maintained these concentrations through at least 72 weeks of treatment.
The company noted that half the study participants had received a total of 60 albumin transfusions in the 48 weeks leading up to treatment, whereas only one patient received a single albumin transfusion in the 48 weeks after starting on Veopoz. Hospitalisations also decreased – with nine of the 10 patients having been hospitalised for a total of 268 days in the 48 weeks prior to treatment, compared with two patients hospitalised for a total of seven days in the 48 weeks post-treatment. The most common adverse reactions associated with Veopoz included upper respiratory tract infection, fracture, urticaria and alopecia.
"Beyond CHAPLE, we believe Veopoz has promise in a variety of complement-mediated diseases and are driving forward several clinical programmes to explore its broader potential," remarked George Yancopoulos, chief scientific officer at Regeneron. Veopoz is also being evaluated in combination with Alnylam Pharmaceuticals' C5-targeting RNAi therapeutic cemdisiran for paroxysmal nocturnal haemoglobinuria and myasthenia gravis.
Meanwhile, in June, the FDA issued a complete response letter (CRL) regarding Regeneron's application seeking approval of a new 8mg version of Eylea for patients with wet age-related macular degeneration (AMD)AMD), diabetic macular oedema (DME) and diabetic retinopathy. At the time, Regeneron noted that the CRL was "solely due" to an ongoing review of inspection findings at third-party manufacturer Catalent, and that there were "no issues with clinical efficacy or safety, trial design, labeling or drug substance manufacturing…identified."
Analysts have said an approval of the higher-dose version of Eylea could provide Regeneron a defence against rivals such as Roche's Vabysmo (faricimab-svoa), which won US approval early in 2022 to treat both wet AMDAMD and DME. Roche's bispecific antibody, designed to block pathways involving Ang-2 and VEGF-A, allows less frequent dosing than standard therapy with Eylea 2mg.
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