EU experts back tolebrutinib approval for SPMS patients with no relapses
Oral med, now called Cenrifki, shown in trial to delay disability
Written by |
A tablet with a European Union flag on its screen is seen on a doctor's table. (Photo from iStock)
- A regulatory committee in the European Union has recommended that tolebrutinib be approved to treat nonrelapsing secondary progressive multiple sclerosis.
- The oral therapy, now called Cenrifki, was shown to significantly delay disability progression in SPMS patients without recent relapses.
- The European Commission will make the final approval decision; the FDA rejected developer Sanofi's application in the U.S. late last year.
Regulators in the European Union are recommendeding that tolebrutinib be approved as a treatment for people with secondary progressive multiple sclerosis (SPMS) who have not experienced a relapse in the last two years.
That new positive opinion was issued by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP), a regulatory body tasked with reviewing data for experimental treatments in the EU.
It was mainly based on data from the now-completed HERCULES Phase 3 trial (NCT04411641), in which tolebrutinib significantly delayed disability progression in people with nonrelapsing SPMS.
The CHMP’s recommendation will now be reviewed by the European Commission, which has final say over medication approvals in the EU. A decision is expected in the coming months, according to a press release from therapy developer Sanofi.
If approved, the oral therapy will be marketed under the brand name Cenrifki.
Multiple sclerosis (MS) is a chronic disorder marked by inflammation that damages the brain and spinal cord. Most people with MS initially develop relapsing-remitting MS (RRMS), characterized by relapses in which symptoms suddenly worsen followed by periods of remission when symptoms ease.
Over time, some people with RRMS will progress to SPMS, which is marked by symptoms that gradually worsen over time, even in the absence of relapses.
Some people with SPMS still occasionally experience relapses, and therapies that are used for RRMS may still benefit those patients. But for people with SPMS who don’t experience relapses, there are hardly any therapies that have been proven effective for slowing disease progression.
FDA rejected tolebrutinib for US use late last year
Tolebrutinib takes a different approach than most disease-modifying therapies. It was designed to block Bruton tyrosine kinase (BTK), an enzyme that plays key roles in the activation of certain inflammatory immune cells, such as microglia and B-cells. By inhibiting BTK, the therapy aims to dampen the inflammation that drives MS progression.
Sanofi is seeking European approval of tolebrutinib based primarily on data from HERCULES, which tested the therapy against a placebo in people with nonrelapsing SPMS. The results showed that tolebrutinib significantly reduced the risk of six-month confirmed disability progression, by 31% relative to a placebo.
The developer’s application is also supported by data from the GEMINI 1 and 2 trials (NCT04410978 and NCT04410991), which tested tolebrutinib in people with relapsing forms of MS.
Those trials failed to meet their main goal, which was to show that tolebrutinib was better at reducing relapse rates than an older therapy called Aubagio (teriflunomide). Nonetheless, data indicated that tolebrutinib significantly decreased the risk of six-month confirmed disability worsening, by 29% relative to Aubagio.
A fourth Phase 3 clinical trial called PERSEUS (NCT04458051) tested tolebrutinib in primary progressive MS, which is marked by symptoms that worsen gradually from disease onset. But that study failed to show tolebrutinib was better than a placebo at delaying disability progression, and Sanofi has said it will not pursue approval of tolebrutinib for this indication.
Tolebrutinib is already approved in the United Arab Emirates to treat nonrelapsing MS, and to slow disability accumulation independent of relapse activity in adults with MS. Sanofi had also sought a similar approval in the U.S., but the U.S. Food and Drug Administration rejected the application a few months ago due to severe liver injury risks and insufficient evidence of effectiveness.
Leave a comment
Fill in the required fields to post. Your email address will not be published.