After a series of regulatory and clinical setbacks for Sanofi’s trebrutinib, the French drugmaker has scored a victory with its multiple sclerosis (MS) candidate. On Thursday, Europe’s Committee for Medicinal Products for Human Use (CHMP) recommended approval for patients with secondary progressive MS (SPMS) who have not had a relapse in the past two years.
The CHMP cited benefits of trebrutinib including a 31% reduction in disability progression and a 38% reduction in new or expanding lesions per year compared to patients who received a placebo. In Europe, the drug will be sold under the brand name Cenrifki.
Sanofi announced two regulatory delays in the U.S. last year until the FDA rejected the treatment for the same indication in December, citing efficacy and safety issues. In a complete response letter (CRL), the agency said trebrutinib “could not establish a favorable benefit-risk profile in any patient population.”
Similarly, in December, Sanofi disclosed the failure of a Phase 3 trial evaluating trebrutinib as a treatment for primary progressive MS (PPMS), ending hopes of gaining approval for that indication. This was in addition to the failure of trebrutinib in two other late-stage MS trials in 2024.
Sanofi picked up BTK inhibitors in its $3.7 billion acquisition of Principia Biopharma in 2020. According to the FDA’s latest CRL, keeping trebrutinib in the United States poses a risk of drug-induced liver injury (DILI) that cannot be adequately managed with Sanofi’s proposed risk evaluation and mitigation strategy (REMS).
Novartis’ Itvisma receives CHMP approval
Meanwhile, five months after the FDA approved Novartis’ gene therapy Itvisma, the CHMP followed suit and recommended European approval of the treatment for spinal muscular atrophy (SMA).
Itvisma is the successor to the Swiss company’s SMA, Zolgensma, which became the first gene therapy approved to treat neuromuscular diseases in 2019 and attracted attention with its $2.1 million price tag.
In the United States, Itvisma is priced at $2.59 million, which represents a significant reduction in the cost of standard treatment for SMA over a 10-year period.
Although their active drug substances are the same, Zolgensma is administered intravenously, whereas Itobisma is administered directly into the central nervous system by intrathecal injection near the spinal cord. Thanks to its formulation, Itvisma can be administered in more concentrated doses to heavier patients.
CHMP also expressed a positive opinion of Arrowhead’s Redenpro, the first small interfering RNA (siRNA) drug to treat patients with familial chylomicronemia syndrome (FCS). Five months ago, the FDA approved Redenpro as a dietary supplement to reduce triglycerides in adults living with this rare genetic disease.
Novartis withdraws Pluvicto expansion
In addition to the approval recommendation, Novartis has withdrawn its CHMP application for its radioligand therapy Pluvicto to treat adults with PSMA-positive metastatic castration-resistant prostate cancer (mCRPC) who remain asymptomatic or mildly symptomatic after their cancer has progressed despite treatment with hormone-blocking androgen receptor pathway inhibitors (ARPIs).
The withdrawal comes 13 months after Novartis secured expansion in the U.S. indication for Purvict, opening the treatment to a wider range of people. The FDA approval and EU initiative were aimed at treating patients who have not received chemotherapy.
And earlier this month, Soleno Therapeutics withdrew its application for Biocut, a drug to treat bulimia, the insatiable hunger associated with Prader-Willi syndrome (PWS).
The California-based company withdrew its application after the CHMP determined there was not enough data from the trial to determine that the risks outweighed the risks. The FDA approved Viokat last March as the first treatment for bulimia.
