Sanofi has secured two major regulatory approvals in Europe and Japan for its treatments targeting multiple sclerosis (MS) and immune thrombocytopenia (ITP).
The European Commission has approved Cenrifki (tolebrutinib) for adults with secondary progressive multiple sclerosis (SPMS) who have not experienced relapses in the last two years, following a positive recommendation from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP).
SPMS is an advanced stage of multiple sclerosis where patients experience continuous accumulation of disability, including fatigue, cognitive impairment, mobility difficulties, and loss of independence – often without available treatment options.
Cenrifki is an oral, once-daily brain-penetrant Bruton’s tyrosine kinase inhibitor specifically designed to target smouldering neuroinflammation, which is considered a key contributor to MS-related disability progression. According to Sanofi, it is the first disease-targeting therapy approved in the EU to address the underlying process of disability accumulation in adults with SPMS without recent relapses.
The approval was supported by findings from the phase 3 HERCULES study in non-relapsing SPMS (nrSPMS), along with data from the GEMINI 1 and GEMINI 2 studies in relapsing multiple sclerosis. Results showed that Cenrifki significantly delayed the onset of disability progression in nrSPMS.
Sanofi plans to launch Cenrifki in Germany later this year alongside patient support and risk management programmes.
Cenrifki is also approved in Australia to treat nrSPMS and slow disability progression in SPMS without relapse activity, and in the UAE for SPMS without relapses in the past two years.
Separately, Japan’s Ministry of Health, Labour and Welfare has approved Wayrilz (rilzabrutinib) for the treatment of persistent or chronic immune thrombocytopenia (ITP) in patients who have had an insufficient response to existing therapies or experienced tolerability issues.
Wayrilz is an oral reversible Bruton’s tyrosine kinase inhibitor (BTKi) designed to restore immune balance through multi-immune modulation. Sanofi said the treatment addresses underlying immune pathways involved in ITP, a rare disorder that causes low platelet counts and increases the risk of bleeding complications.
The approval was based on results from the phase 3 LUNA 3 study, which demonstrated improvements in sustained platelet counts and related symptoms.
Hisashi Kato, Department of Blood Transfusion and Cell Therapy, The University of Osaka Hospital, Suita, Japan, said, “Wayrilz is a novel BTKi that works through multi-immune modulation to help address the root cause of ITP pathophysiology. It is expected to become a new therapeutic option that may help patients with disease management, including not only improvement in platelet counts but also considering patients’ quality of life.”
Manuela Buxo, executive vice president and global head of specialty care at Sanofi, said Wayrilz offers new hope to patients who have not responded to prior therapy. He added that the approval reflects the company’s commitment to expanding treatment options for patients with rare diseases.
Wayrilz is already approved for ITP treatment in the US, EU, UAE and UK and continues to be investigated in other immune-mediated conditions, including IgG4-related disease, warm autoimmune haemolytic anaemia and sickle cell disease.
