Update on the US regulatory review of tolebrutinib in non-relapsing, secondary progressive multiple sclerosis Paris, September 22, 2025. The US Food and Drug Administration (FDA) has extended by three months the target action date of its review of the new drug application (NDA) of tolebrutinib, an oral and brain-penetrant investigational Bruton's tyrosine kinase (BTK) inhibitor to treat non-relapsing, secondary progressive multiple sclerosis (nrSPMS) and to slow disability accumulation independent of relaps ...

Core Opinion - The European Medicines Agency's Committee for Medicinal Products for Human Use has issued a positive opinion for Jaypirca (pirtobrutinib) for treating adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) previously treated with a BTK inhibitor [1][2][5] Clinical Trial Results - The BRUIN CLL-321 trial is the first randomized Phase 3 study in CLL conducted exclusively in patients previously treated with a BTK inhibitor [1][3] - The primary endpoint of progression-free survival (PFS) was met, showing pirtobrutinib was superior to the standard care options, reducing the risk of disease progression or death by 46% (median PFS: 14.0 months vs. 8.7 months) [3][7] - The median time to next treatment or death (TTNT) was 24 months for pirtobrutinib compared to 11 months for the control arm, indicating a 63% improvement [3][7] Safety Profile - The overall safety profile of pirtobrutinib was consistent with previous studies, with common adverse reactions including neutropenia, fatigue, diarrhea, anemia, rash, and contusion [3][18] - Serious adverse reactions occurred in 56% of patients with CLL, with pneumonia (18%) and COVID-19 (9%) being the most common [24][26] Regulatory Status - Following the positive opinion, the application for Jaypirca is now referred to the European Commission for final action, with a decision expected in one to two months [2][5] - Jaypirca has also received conditional marketing authorization in the EU for treating adult patients with relapsed or refractory mantle cell lymphoma (MCL) previously treated with a BTK inhibitor [2][5] Market Context - There are currently no treatment options specifically studied in a randomized Phase 3 trial for patients with relapsed or refractory CLL in the post-BTK inhibitor setting, highlighting the unmet need in this area [2][5] - Jaypirca was approved in the U.S. in 2023 under the FDA's Accelerated Approval pathway for similar indications, indicating a growing market for targeted therapies in hematologic malignancies [5][6]