Viltolarsen

George Tidmarsh, director of the FDA’s drug center, said in a now-deleted social media post that the agency would reconsider the use of surrogate endpoints that have been used to approve drugs more quickly than the standard approval process. While noting that “there is no doubt” that the use of surrogate endpoints “has benefited patients by bringing valuable treatments to patients sooner” through the FDA’s accelerated approval pathway, “there have been notable failures in confirmatory trials,” Tidmarsh wrote Monday. “We will be taking a close look at the use of surrogate endpoints to see where we can further accelerate promising drugs faster while requiring companies to perform the trials necessary to confirm actual clinical benefit.” He pointed specifically to confirmatory trials “for exon skipping therapies in [Duchenne muscular dystrophy].” NS Pharma said in May 2024 that its DMD drug Viltepso proved to be no better than placebo in its confirmatory study, but has yet to be removed from the market, while Sarepta Therapeutics’ first DMD drug Exondys 51 has yet to complete its confirmatory study despite winning an accelerated approval in 2016. His comment was made in a post on LinkedIn — an unusual venue for what could be a highly consequential regulatory action. He also took the unusual step of directly criticizing a specific drug: Aurinia Pharmaceuticals’ Lupkynis, which won an accelerated approval in January 2021 to treat a form of lupus and later won full approval, with a label update in 2024. In his post, Tidmarsh said that Aurinia’s drug “has not been shown to provide a direct clinical benefit for patients,” a statement that was followed by a sharp 17% drop in the company’s stock $AUPH . Regulators rarely call out specific companies or drugs by name, instead relying on the FDA’s official channels for action. Holly Fernandez Lynch, an assistant professor of medical ethics at University of Pennsylvania’s medical school and an FDA expert, said she’d noticed far more cases of FDA officials making policy by social media. “It makes it feel a lot less formal and official,” Lynch said. “It doesn’t seem like it has kind of the imprimatur of the government behind it.” Soon after Endpoints’ story ran, Tidmarsh deleted his original post and put up a new comment. “The views in my last post were my own personal views,” he said in the new post . “They do not reflect the views of FDA or HHS. All official guidance or comments from the Agency or Department are made through official channels. For the avoidance of confusion I have deleted the post.” That post now also appears to have been deleted. Jefferies analysts said the selloff in Aurinia stock “was unwarranted” as Lupkynis “ continues to demonstrate a consistent profile (w/ ph.III) in the real-world commercial setting, based on FAERS data we have tracked ; at worst case, maybe FDA urges the co to do something voluntarily.” Aurinia said in a statement that it “stands behind the favorable benefit/risk profile of” Lupkynis. Tidmarsh also has at least a tangential connection to Aurinia. The company’s largest shareholder is the biotech investor Kevin Tang. Tang was the chair of the board at La Jolla Pharmaceuticals when Tidmarsh exited as CEO in 2019 after the failure of a pivotal trial. At the time, La Jolla said Tidmarsh left “to pursue other interests.” Tang didn’t respond to a request for comment. Tidmarsh also didn’t respond to a request for comment about the FDA’s apparent new policy, or his time at La Jolla. Another Aurinia board member, Craig Johnson, also served on the La Jolla board at the time. Endpoints News wasn’t able to find a way to reach Johnson. Since at least 2023, the FDA has made clear that it’s looking to speed up confirmatory trials for accelerated approvals, since when those trials lag, patients may continue to rely on therapies with limited or no clinical benefit. In a draft guidance from last January , the agency officially spelled out that if a confirmatory trial isn’t underway before an accelerated approval, the FDA won’t clear the drug for use. A 2022 review in the Journal of Cancer Policy found that of the 15 surrogate analyses conducted by the FDA, “only one demonstrated a strong correlation between a surrogate outcome and overall survival.” Researchers at Yale and elsewhere in 2024 called on the FDA to share more about how the agency considers the use of surrogate markers as primary endpoints outside of oncology trials, too. Editor’s note: The article was updated to note that Tidmarsh deleted his original post on LinkedIn and added a new comment. It was updated again on Tuesday after he deleted the new comment.

July 8, 2025 I Taiho Pharmaceutical Co., Ltd. announced that the results of a phase III, randomized, placebo-controlled, double-blind and open-label, extension study of TAS-205 in patients with Duchenne muscular dystrophy (DMD) (REACH-DMD) showed no significant difference in the mean change from baseline to 52 weeks in the time to rise from the floor, which was the primary endpoint in the ambulatory cohort of the study. The ambulatory cohort of this study was conducted in Japan as a placebo-controlled, multi-center, double-blind comparative study in male DMD patients aged 5 years and older. The purpose of this study is to evaluate the efficacy of TAS-205 by orally administering TAS-205 or a placebo twice daily for 52 weeks. A total of 82 patients were enrolled in 26 sites from November 2020 to December 2023. Detailed results from this study will be presented at an appropriate upcoming academic conference. For more information on this study (NCT04587908/jRCT2041200055), please visit: www.clinicaltrials.gov/ / https://jrct.mhlw.go.jp/en-top In December 2019, development of TAS-205 was selected as one of the 4th Cyclic Innovation for Clinical Empowerment (CiCLE*) program for fiscal 2019, under the application title: Development of an innovative therapeutic drug for DMD with a novel mechanism of action originating from Japan by utilizing patient registry. This program is operated by the Japan Agency for Medical Research and Development (AMED). *CiCLE：Cyclic Innovation for Clinical Empowerment About TAS-205 TAS-205 (INN: pizuglanstat) is a selective hematopoietic prostaglandin D synthase (HPGDS*) inhibitor discovered by Taiho Pharmaceutical. It is under development as a DMD treatment which can be used regardless of the dystrophin gene mutation type, controlling the decline in motor function in DMD patients by inhibiting HPGDS, which exacerbates the inflammatory response in DMD patients’ muscles. *HPGDS：Hematopoietic prostaglandin D synthase About CiCLE This program aims at creating a foundation (including human resources) for a radical transformation in the way research and development is conducted in response to needs arising in clinical settings and for accelerating the practical application to pharmaceutical products. It also seeks to drive the creation of an environment that strongly promotes the fostering of open innovation in the field of medical research and development through the combined efforts of government, academia, and the private sector. About DMD (Duchenne muscular dystrophy) DMD is a genetic disorder with a higher prevalence in young males. It is caused by a genetic mutation in a gene, which codes dystrophin protein supporting the framework of muscle cells. It is a refractory, serious illness leading to a loss of muscle strength caused by the inability to produce normal dystrophin protein. The prevalence of DMD is 1.9 to 3.4 per 100,000 individuals 1 , and there are an estimated 3,000 to 4,000 patients in Japan. Currently, oral steroids and viltolarsen are approved and delandistrogene moxeparvovec has received conditional and time-limited approval for DMD in Japan, however new treatment options are being awaited. SOURCE: Taiho Pharmaceutical Co