EU regulators back the first drug shown to slow disability in progressive MS

The European Medicines Agency's human medicines committee recommended five new medicines for EU approval at its April 20-23 meeting, but one stood apart from the usual mix of biosimilars, generics, and indication extensions: tolebrutinib, sold as Cenrifki, recommended for non-relapsing secondary progressive multiple sclerosis (nrSPMS) [s1]. If the European Commission follows the recommendation, as it typically does, tolebrutinib would become the first drug-specific treatment in the EU targeting disability progression in this particular MS population, rather than the relapses that most existing MS therapies are built to prevent [s1].

A form of MS most drugs don't touch

Secondary progressive MS describes a phase some patients with relapsing MS eventually enter, in which disability accumulates steadily rather than through discrete relapse-and-recovery episodes. "Non-relapsing" narrows that further, to patients whose disease has stopped producing clinical relapses but continues to worsen. Most approved MS therapies work by reducing relapse frequency — a mechanism that offers little to patients whose disability is now accumulating without relapses to prevent. That gap is what the CHMP recommendation addresses.

The trial behind the recommendation

The EMA's meeting summary does not detail the clinical evidence supporting the tolebrutinib recommendation [s1]. The pivotal data comes from the Phase 3 HERCULES trial, published in the New England Journal of Medicine in April 2025: a double-blind, randomized study that assigned patients with nrSPMS 2:1 to daily oral tolebrutinib or placebo for approximately 48 months [s2]. Tolebrutinib delayed the time to six-month confirmed disability progression by 31% relative to placebo (hazard ratio 0.69, 95% CI 0.55-0.88, p=0.003) [s2]. Patients enrolled had Expanded Disability Status Scale scores of 3.0 to 6.5, no relapses in the prior 24 months, and documented disability accumulation in the year before enrollment — a population selected specifically to represent steady, non-relapsing progression [s2].

A 31% delay in the time to confirmed disability progression is a real, statistically significant effect — not a cure, and not a reversal of existing disability, but a slowing of the rate at which new disability accumulates. Framed against a population with essentially no approved disability-targeting option before this, that partial effect is what makes the drug notable, rather than any claim of transforming the disease's course outright. Sanofi's release of the underlying data also reported a benefit on a secondary measure — time to three-month confirmed disability progression — though it did not include the specific numerical result for that endpoint [s2].

Tolebrutinib is a Bruton's tyrosine kinase inhibitor, a class of drug more established in blood cancers and, more recently, in other autoimmune conditions, repurposed here for its ability to cross into the brain and act on immune cells implicated in the smoldering inflammation thought to drive non-relapsing progression. The EMA's recommendation is separate from, and does not depend on, a parallel review of tolebrutinib underway at the FDA.

What else the committee recommended

Tolebrutinib was one of five positive opinions from the April meeting. The others were onasemnogene abeparvovec (Itvisma), an orphan gene therapy for 5q spinal muscular atrophy; plozasiran (Redemplo), an orphan medicine for familial chylomicronaemia syndrome, a rare genetic lipid disorder; a ranibizumab biosimilar (Rexatilux) for neovascular age-related macular degeneration and related retinopathies; and a generic version of palbociclib for breast cancer [s1]. The committee also recommended extending the approved indications of nine already-authorized medicines, including Comirnaty, Opdivo, and Skyrizi [s1].

What a CHMP recommendation is, and isn't

A positive CHMP opinion is not itself a marketing authorization. It is a recommendation that goes to the European Commission, which formally grants or denies EU-wide approval — typically, though not automatically, following the committee's recommendation within roughly two months. Until that decision, tolebrutinib is not yet approved for use anywhere in the EU; the April 20-23 meeting represents the scientific review stage, not market access.

What to watch

The European Commission's formal decision, expected within the next two months if it follows the usual timeline, will determine whether and when tolebrutinib becomes available to patients in EU member states. Separately, tolebrutinib's regulatory status in the United States remains a distinct process under FDA review, unaffected by the EU committee's recommendation.

This article is informational and is not medical advice.

Sources

• [s1] European Medicines Agency, "Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP), 20-23 April 2026," published 24 April 2026. https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-20-23-april-2026
• [s2] Sanofi, "Tolebrutinib phase 3 data published in NEJM demonstrate benefit on disability progression in multiple sclerosis," 8 April 2025. https://www.sanofi.com/en/media-room/press-releases/2025/2025-04-08-17-11-11-3057931