After a series of regulatory and clinical setbacks for Sanofi’s tolebrutinib, the French pharma has scored a victory for the multiple sclerosis (MS) candidate. Thursday, Europe’s Committee for Medicinal Products for Human Use (CHMP) recommended its approval for patients with secondary progressive MS (SPMS) who have not had relapses in the last two years.
The CHMP cited the benefits of tolebrutinib, including a 31% reduction in disability progression and a 38% reduction in new or enlarging lesions per year compared with patients on placebo. In Europe, the drug will carry the commercial moniker Cenrifki.
Last year, Sanofi announced two regulatory delays in the United States before the FDA rejected the treatment in the same indication in December, citing efficacy and safety issues. In its complete response letter (CRL), the agency said that “a favorable benefit-risk profile could not be established for any patient subpopulation” for tolebrutinib.
Also in December of last year, Sanofi disclosed the flop of a phase 3 trial assessing tolebrutinib as a treatment for primary progressive MS (PPMS), ending its hopes of gaining approval in the indication. That came on top of failures for tolebrutinib in two other late-stage MS trials in 2024.
Sanofi picked up the BTK inhibitor as part of its $3.7 billion buyout of Principia Biopharma in 2020. Holding tolebrutinib back in the U.S. has been the risk of drug-induced liver injury (DILI), which can’t be adequately managed by Sanofi’s proposed risk evaluation and mitigation strategy (REMS), according to the FDA in its most recent CRL.
Novartis’ Itvisma gains CHMP nod
Meanwhile, five months after the FDA signed off on Novartis’ gene therapy Itvisma, the CHMP has followed suit with a recommendation for the spinal muscular atrophy (SMA) treatment to be approved in Europe.
Itvisma is the Swiss company’s SMA follow-on to Zolgensma, which in 2019 became the first gene therapy approved to treat a neuromuscular disease and gained attention for its $2.1 million price tag.
In the U.S., Itvisma has been priced at $2.59 million, which is a significant reduction in the cost of standard treatments for SMA over a span of 10 years.
While their active drug substances are identical, Itvisma is given directly to the central nervous system by way of an intrathecal injection near the spinal cord, whereas Zolgensma is administered intravenously. Because of that formulation, Itvisma can be given to heavier patients in a more concentrated dose.
The CHMP also weighed in with a positive opinion for Arrowhead’s Redemplo, which is the first small interfering RNA (siRNA) medicine to treat patients with familial chylomicronemia syndrome (FCS). Five months ago, the FDA approved Redemplo as an adjunct to diet to reduce triglycerides in adults living with the rare genetic disorder.
Novartis withdraws Pluvicto expansion
Besides the approval recommendations, Novartis pulled a CHMP application for radioligand therapy Pluvicto to treat adults with PSMA-positive metastatic castration-resistant prostate cancer (mCRPC) who have no or mild symptoms after their cancer progressed despite treatment with a hormone-blocking androgen receptor pathway inhibitor (ARPI).
The withdrawal comes 13 months after Novartis secured an expansion in the U.S. for Pluvicto in the indication, which opened the therapy up to a significantly broader population. The FDA approval—and the EU effort—were to treat patients who have not had chemotherapy.
And earlier this month, Soleno Therapeutics withdrew its application for Viokat, a treatment for hyperphagia, an insatiable hunger condition associated with Prader-Willi Syndrome (PWS).
The California-based company pulled its application after the CHMP determined that data from its trial were not sufficient to determine that its benefits outweighed its risks. The FDA approved Viokat in March of last year as the first treatment for hyperphagia.
