Brand-Name Rituximab Will Get FDA Priority Review for a New Pediatric Indication

Drug maker Genentech, developer of the brand-name rituximab, Rituxan, has announced that the FDA has accepted a supplemental Biologics License Application for the drug’s first proposed pediatric indication: 2 rare forms of vasculitis.
The Center for Biosimilars Staff
June 13, 2019
Drug maker Genentech, developer of the brand-name rituximab, Rituxan, has announced that the FDA has accepted a supplemental Biologics License Application (sBLA) for the drug’s first proposed pediatric indication: 2 rare forms of vasculitis.

The BLA, which was granted priority review, seeks approval for rituximab in combination with glucocorticoids for granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) in children aged 2 years or more. GPA and MPA are rare diseases that cause inflammation of small blood vessels, impacting blood flow and leading to organ damage.

Cases of pediatric-onset GPA and MPA are rare, and are associated with severe, sometimes life-threatening symptoms, and to date, there are no approved therapies to treat children with these disorders.

“We are committed to delivering new treatment options for rare diseases, such as pediatric GPA and MPA…,” said Sandra Horning, MD, chief medical officer and head of global product development at Genentech, in a statement. “We will continue to work closely with the FDA to bring Rituxan to children with these [2] serious and potentially life-threatening diseases.”

The sBLA relies on data from the PePRS study, a phase 2a open-label, single-arm study of rituximab in pediatric patients with newly diagnosed or relapsing GPA or MPA.1 The study in 25 patients at 11 centers included 4 rituximab infusions and a 6-month remission induction phase. In total, 56% of patients achieved remission by month 6, and 100% achieved remission by month 18. All patients in the study had 1 or more adverse event, with infusion reactions being the most commonly reported.

While both forms of vasculitis are rare, and cases in children are rarer still, the new indication could be another means by which Genentech can defend some sales of its brand-name drug against upcoming biosimilar competition.

The FDA has approved 1 biosimilar rituximab, Truxima, but due to issues related to intellectual property, its developers, Celltrion and Teva, sought approval only in malignant indications, and none in other approved Rituxan indications such as rheumatoid arthritis, in which the biosimilar was also studied.

Reference
1. Brogan P, Cleary G, Hersch AO, et al. Pediatric open-label clinical study of rituximab for the treatment of granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA). Presented at: the American College of Rheumatology 2018 Annual Meeting; October 19-24, 2018; Chicago, Illinois. Abstract L04.

 

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