Roche’s fenebrutinib has received FDA filing acceptance under priority review for a New Drug Application (NDA) for relapsing multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS).

Fenebrutinib is an investigational non-covalent Bruton’s tyrosine kinase (BTK) inhibitor. The filing acceptance is based on data from the comprehensive clinical programme, including the phase 3 FENhance 1 and 2 RMS studies and the phase 3 FENtrepid PPMS study.

Fenebrutinib became the first investigational medicine in more than a decade to slow disability progression in a phase 3 PPMS trial (FENtrepid), with numerical benefit vs Ocrevus. Fenebrutinib cut relapses by 51% and 58% vs teriflunomide in phase 3 RMS trials (FENhance 1 & 2) while showing a consistent, positive trend in delaying disability.

Fenebrutinib is designed to address two drivers of MS – acute inflammation causing relapses and chronic brain inflammation driving disability progression. If approved, fenebrutinib would become the first BTK inhibitor and first high-efficacy oral treatment for both RMS and PPMS, offering a new option for the nearly one million Americans living with MS.

“Ocrevus transformed how we treat MS, helping more than 525,000 people live a life less defined by their disease. Yet, over a third of all people with MS still receive lower-efficacy treatment,” said Teresa Graham, Roche’s CEO, Pharma. “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.”

MS is a chronic disease that affects more than three million people worldwide. People with all forms of MS experience disease progression from the beginning of their disease. Therefore, an important goal of treating MS is to slow, stop and ideally prevent progression as early as possible.

Concurrently addressing the life-disrupting relapses and long-term disease progression – the slow, steady loss of physical and mental abilities over time – remains one of the greatest challenges in MS. Fenebrutinib is designed to act throughout the body and to cross the blood-brain barrier into the central nervous system to target both the acute inflammation that causes relapses and the chronic inflammation that is thought to drive disability progression.

Approximately 85% of people with MS are initially diagnosed with relapsing-remitting multiple sclerosis (RRMS). Relapsing forms of the disease (RMS) include RRMS and active secondary progressive MS, and people with RMS experience relapses and worsening disability over time. Primary progressive multiple sclerosis (PPMS) is a debilitating form of the disease marked by steadily worsening symptoms but typically without distinct relapses or periods of remission.

Approximately 15% of people with multiple sclerosis are diagnosed with the primary progressive form of the disease. Until the FDA approval of Ocrevus, there had been no FDA-approved treatments for PPMS, and Ocrevus is still the only approved treatment for PPMS.

“People living with multiple sclerosis need treatments that go beyond controlling relapses to help preserve function and independence as the disease progresses,” said Levi Garraway, Roche’s CMO and Head of Global Product Development. “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.”