FDA grants priority review to ‘high-efficacy’ drug for 2 types of MS
Oral therapy aims to cut inflammatory attacks that drive relapses, progression
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An application seeking approval of the oral therapy fenebrutinib for two forms of multiple sclerosis is now under review by the U.S. FDA. (Image from iStock)
- The U.S. FDA has granted priority review to the oral therapy fenebrutinib as a treatment for both relapsing and progressive forms of multiple sclerosis.
- The Roche drug aims to ease both acute inflammation causing relapses and chronic brain inflammation driving disability progression.
- With priority review, a decision by the regulatory agency on whether to approve fenebrutinib is expected by spring of next year.
The U.S. Food and Drug Administration (FDA) has agreed to review an application from Roche seeking approval of its BTK inhibitor fenebrutinib as a treatment for relapsing forms of both multiple sclerosis (MS) and primary progressive MS (PPMS).
Further, the FDA granted the application priority review, under which the regulatory agency aims to make its decision within six months, compared with the 10 months it usually takes. With priority review, a decision on whether or not to approve the treatment candidate — designed to address two drivers of MS — would be expected by late March or early April.
Roche said the filing makes fenebrutinib the first BTK inhibitor — a drug that blocks the abnormal growth of B cells — to have an application accepted for review for both relapsing forms of MS and PPMS.
“If approved, fenebrutinib could open a new chapter as the first high-efficacy oral therapy for both relapsing and primary progressive MS, helping to control disease activity while giving people more flexibility and choice,” Teresa Graham, CEO of Roche Pharmaceuticals, said in a company press release.
MS occurs when the immune system mistakenly launches an inflammatory attack against myelin, the protective coating around nerve fibers. In PPMS, symptoms worsen gradually from the onset of the disease, while in relapsing MS, periods of worsening or new symptoms, called relapses, are interspersed by time periods in which symptoms ease.
Fenebrutinib is designed to block a protein called Bruton’s tyrosine kinase (BTK), which is essential for the activity of certain immune cells involved in MS. Inhibiting BTK is expected to reduce the inflammatory attacks that drive MS relapses, as well as the smoldering inflammation inside the brain that contributes to gradual disease progression over time across MS types.
Roche noted that fenebrutinib aims to ease both “acute inflammation causing relapses and chronic brain inflammation driving disability progression.”
Roche exec says fenebrutinib aims to ‘go beyond’ relapse control
According to Levi Garraway, MD, PhD, Roche’s chief medical officer, the goal is a new drug that doesn’t just prevent relapses.
“People living with multiple sclerosis need treatments that go beyond controlling relapses to help preserve function and independence as the disease progresses,” Garraway said.
The application for fenebrutinib is supported by data from three ongoing Phase 3 clinical studies. In the FENhance 1 (NCT04586010) and FENhance 2 (NCT04586023) twin studies, fenebrutinib treatment for about two years significantly reduced the average number of relapses per year, by more than 50%, compared with Aubagio (teriflunomide), an approved MS treatment.
In these two studies, which together involve nearly 1,500 adults with relapsing MS, relapse rates equated to about one relapse every 17 years, which Roche said is the lowest ever seen in MS Phase 3 studies.
Fenebrutinib also significantly reduced the number of new and enlarging lesions as well as lesions with active inflammation, by more than 70%, and showed a positive trend toward slower disability progression.
Three [late-stage clinical trials] have demonstrated the potential for fenebrutinib to address both relapsing and progressive disease, thereby bringing us closer to an oral treatment that could make a meaningful difference across the MS spectrum.
In the other study, called FENtrepid (NCT04544449), fenebrutinib was shown to be at least as effective in adults with PPMS as the approved treatment Ocrevus (ocrelizumab). In fact, while the study was not designed to show significant differences between the drugs, the data demonstrated that fenebrutinib numerically reduced the risk of confirmed disability progression by 12% compared with Ocrevus, the researchers noted.
The scientists noted that a treatment effect was also observed in patients without signs of active inflammation on MRI scans, indicating that fenebrutinib may help slow disability progression even in individuals with less inflammatory activity. This is particularly relevant because patients without signs of active inflammation have been shown to benefit less from Ocrevus.
“Three Phase [3] studies have demonstrated the potential for fenebrutinib to address both relapsing and progressive disease, thereby bringing us closer to an oral treatment that could make a meaningful difference across the MS spectrum,” said Garraway, who doubles as head of global product development.
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