Fitusiran

CSU is a chronic skin disease driven partly by type 2 inflammation, leading to severe itching and hives. Credit: Pormezz / Shutterstock.com. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending the approval of Sanofi and Regeneron’s Dupixent (dupilumab) in the European Union (EU) for paediatric patients with chronic spontaneous urticaria (CSU). The recommendation applies to children aged two to 11 years experiencing moderate-to-severe CSU who do not respond adequately to histamine-1 antihistamines and are naïve to anti-immunoglobulin E (IgE) therapy. It is based on results from the LIBERTY-CUPID clinical programme, including two Phase III trials (Study A and Study C) involving children aged six to 11 years, and the single-arm CUPIDKids Phase III study for children aged two to 11 years with CSU. A final decision is anticipated in the coming months. Dupixent is already approved for use in certain adults and adolescents with CSU in regions including the EU, Japan, and the US. In the US, a supplemental biologics licence application is under review to extend approval of Dupixent for children aged two to 11 years, with a decision from the US Food and Drug Administration (FDA) anticipated by April 2026. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence CSU is a chronic skin disease driven partly by type 2 inflammation, leading to severe itching and hives. Dupixent is a human monoclonal antibody targeting interleukin-4 and interleukin-13 pathways without serving as an immunosuppressant. It is being jointly developed by Sanofi and Regeneron and studied across over 60 clinical trials involving more than 12,000 patients with type 2 inflammation-driven diseases. Ongoing Phase III studies are evaluating further potential indications. In December 2025, China’s National Medical Products Administration (NMPA) granted approval for Sanofi’s rare haematologic disease treatments, Qfitlia (fitusiran) and Cablivi (caplacizumab), for patients with haemophilia and acquired thrombotic thrombocytopenic purpura (aTTP), respectively.

The negative reaction to the impending departure of Sanofi CEO Paul Hudson, and Merck KGaA leader Belén Garijo as his replacement, is perhaps surprising given the French company’s muted performance under Hudson’s leadership. Sanofi’s shares fell about 3% in Paris and 4% in New York, a cold shoulder that may simply reflect the scale of the challenge that Garijo faces when she takes the reins in late April. She’ll have less than five years before the company’s immunological juggernaut Dupixent goes generic. Investors are concerned Garijo doesn’t have a track record of delivering R&D productivity at Merck KGaA, Jefferies managing director Michael Leuchten told Endpoints News in an interview. He considers that criticism “a little unfair” because the German conglomerate focused its capital allocation on areas other than pharma. “We thought the negative reaction on share price was harsh and kind of misses some of the nuances of what may be going on here,” Leuchten said. The company’s shares have lost about a quarter of their value in the past year. Garijo and her team will have to find pragmatic ways to extend Dupixent’s exclusivity, which could be possible through patent settlements and building on its joint venture with Regeneron, because there isn’t time to “rebuild an R&D organization from scratch” before Dupixent’s patent expires, Leuchten said. “One scenario that I think is possible is for Sanofi to go and acquire some assets that they fully fund and put those assets into the joint venture,” Leuchten said. Sanofi’s strategic priorities will remain unchanged under Garijo, a company spokesperson told Endpoints in an email. At Merck KGaA, Garijo’s history of dealmaking wasn’t scintillating. The biggest deal the company closed under her leadership was the $3.9 billion SpringWorks acquisition , and the rare cancer drugs Merck KGaA obtained haven’t been particularly lucrative so far. Another aspect driving some of the skepticism is that Garijo has had trouble delivering on promises in the past. In a letter in early 2022, she set out a goal for Merck KGaA known as “25 by 25” — achieving €25 billion ($27 billion) in total sales by 2025. The group won’t report its 2025 earnings until next month, but last October it guided to 2025 net sales between €20.8 billion and €21.4 billion. This missed goal is undeniably partly due to pipeline misfires. The failure of Merck KGaA’s evobrutinib in multiple sclerosis in late 2023 was followed by the cancellation of the pivotal trial of the head and neck cancer drug xevinapant in June 2024, after an interim analysis concluded the study was headed for failure. But Sanofi’s efforts to improve its drug development under Hudson didn’t always go as planned either. In 2019, at the beginning of his tenure as CEO, Hudson highlighted six programs as priorities. Two made it to market, Qfitlia (for hemophilia) and Beyfortus (for respiratory syncytial virus), but the others have either disappointed or failed entirely. Hudson also stopped developing a GLP-1 program for diabetes called efpeglenatide, just as other drugmakers were stepping up development efforts of this mechanism in obesity. There is no guarantee that asset would have gone on to do well, but the decision looks poor in retrospect. Sanofi’s current strategy involves going big on vaccines and investing in deals with a budget of up to €15 billion to spend across four key therapeutic areas: immunology, vaccines, neurology and rare diseases. This seems unlikely to compensate for Dupixent’s loss of exclusivity, since the drug accounted for 36% of Sanofi’s 2025 revenues. It was perhaps Sanofi’s eagerness on immunology that led things to go wrong at the French drugmaker under Hudson in the first place, Leuchten said. Sanofi assumed that immunology can be approached rationally and with acceptable risk, but time has shown that the space is “a lot more complicated than we thought,” Leuchten said. “The placebo response rates are all over the shop, you get intratrial variability, so the rational approach doesn’t work as well as anybody thought.” Sanofi’s change at the top seems to have happened fast; CEO transitions can be planned around a year in advance in the normal run of things. But Sanofi has ousted leaders with unceremonious haste before. In 2014 it sacked then-CEO Chris Viehbacher after a boardroom clash over Viehbacher’s management style.