Sanofi has received a significant boost for its multiple sclerosis treatment tolebrutinib after European regulators recommended the drug for approval, marking a turnaround following previous clinical and regulatory challenges.
The European Medicines Agency’s Committee for Medicinal Products for Human Use has endorsed tolebrutinib for patients with secondary progressive multiple sclerosis who have not experienced relapses in the past two years. The recommendation follows a difficult period for the therapy, which had faced delays and rejection in the United States.
According to the committee, tolebrutinib demonstrated a 31 per cent reduction in disability progression and a 38 per cent decrease in new or enlarging brain lesions compared with placebo. If approved, the drug will be marketed in Europe under the name Cenrifki.
The positive opinion contrasts with the stance taken by US regulators. The Food and Drug Administration rejected the treatment in December, citing concerns over both efficacy and safety, including the risk of drug-induced liver injury. Earlier trial failures in primary progressive multiple sclerosis and other late-stage studies had also cast doubt on the drug’s prospects.
Tolebrutinib was acquired through Sanofi’s purchase of Principia Biopharma in 2020 and represents a key asset in the company’s neurology pipeline.
In parallel developments, the CHMP also recommended approval of Novartis’ gene therapy Itvisma for spinal muscular atrophy. The treatment, which follows the earlier gene therapy Zolgensma, is administered via intrathecal injection, allowing delivery directly into the central nervous system.
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Meanwhile, Novartis withdrew its European application to expand the use of its radioligand therapy Pluvicto for prostate cancer patients who have not yet received chemotherapy. The decision comes despite a broader approval for the drug in the United States last year.
The CHMP also issued a positive opinion for Arrowhead Pharmaceuticals’ Redemplo for a rare lipid disorder, while another application for a treatment targeting hyperphagia was withdrawn due to insufficient supporting data.
This news item has been summarised using AI and checked by humans before publication.
Sources
fiercepharma.com
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