Eton Pharmaceuticals, Inc.

五月，美国食品药品监督管理局（FDA）迎来一批关键药物审批决策期，涉及葛兰素史克（GSK）、赛诺菲（Sanofi）、默克（Merck）等多家知名药企的重要产品。这些审批结果不仅关乎企业发展，还将对相关疾病的治疗格局产生重大影响。哮喘药新适应症：GSK 挑战 COPD 治疗领域GSK 正寻求 FDA 批准其哮喘注射药物 Nucala 用于慢性阻塞性肺疾病（COPD）的治疗，FDA 将于 5 月 7 日做出决定。COPD 是一种肺部炎症性疾病，常表现为呼吸困难、咳嗽和气流阻塞。约 40% 的 COPD 患者会因 2 型炎症引发疾病恶化，而这与血液中高嗜酸性粒细胞计数有关。Nucala 作为一种单克隆抗体，能够靶向并阻断维持嗜酸性粒细胞活性的 IL - 5 细胞因子。GSK 依据 III 期 MATINEE 试验数据支持此次申请，该试验显示 Nucala 相较于安慰剂，能在统计学和临床意义上显著降低中重度疾病恶化的年发生率。目前，Nucala 已获批用于多种疾病，若成功获批用于 COPD，将进一步拓展其治疗领域。皮肤病药拓展：Arcutis 瞄准更多银屑病患者5 月 22 日，FDA 将对 Arcutis Biotherapeutics 公司的 Zoryve 扩展用于身体和头皮银屑病的申请做出裁决。Zoryve 是一种局部磷酸二酯酶 4 抑制剂，通过抑制皮肤炎症发挥作用，自 2011 年起已获批用于治疗 12 岁及以上患者的斑块状银屑病。此次 Arcutis 凭借 III 期 ARRECTOR 试验数据进行扩展申请，试验结果显示，接受 Zoryve 治疗的患者中，66.4% 的人皮肤达到清除或几乎清除状态，而安慰剂组仅为 27.8%；同时，Zoryve 组 65.3% 的患者瘙痒症状显著改善，安慰剂组仅为 30.3%。若获批，这将为症状控制不佳的患者带来新的治疗选择。疫苗适用人群扩展：赛诺菲关注婴幼儿健康赛诺菲的脑膜炎球菌病疫苗 MenQuadfi 目前已获批用于 2 岁及以上儿童的初次免疫，以及 13 岁及以上仍有感染风险人群的加强免疫。赛诺菲希望将其适用人群扩展至 6 周龄至 23 月龄的婴幼儿，FDA 预计 5 月 23 日给出审批结果。赛诺菲在 4 月向 CDC 免疫实践咨询委员会展示了多项试验数据，包括 III 期 MET42 研究，结果表明 MenQuadfi 在婴幼儿中使用安全，大多数副作用为轻度或中度，且在免疫反应和血清保护方面与已获批的商业疫苗相当。若获批，将为这一年龄段的儿童提供针对 A、C、W 和 Y 群脑膜炎奈瑟菌的保护。肺病新药获批在望：Liquidia 冲刺最后审批Liquidia 公司的吸入性粉末药物 Yutrepia 用于治疗间质性肺病相关肺动脉高压（PH - ILD），预计 5 月 23 日迎来 FDA 的最终批准。2024 年 8 月，FDA 已给予该药物临时批准，表明其在疗效、安全性和产品质量方面已达标，但需等待竞争产品的监管独占期结束。Yutrepia 是曲前列尼尔的吸入剂型，III 期 INSPIRE 研究显示，该药物在未使用过曲前列尼尔或从雾化曲前列尼尔转换过来的患者中，安全性和耐受性良好，且经过一年的给药，患者症状得到稳定或改善。抗癌药新征程：默克拓展 Welireg 适用范围默克希望将其口服抗癌药 Welireg 的适用范围扩展至晚期嗜铬细胞瘤和副神经节瘤（PPGL），FDA 将于 5 月 26 日做出决定。PPGL 是两种罕见的肾上腺肿瘤，每年约有 2000 名患者被诊断出，通常由特定基因突变引起，部分患者确诊时已处于转移阶段。默克依据 II 期 LITESPARK - 015 研究 A1 队列的数据进行申请，虽然未公布具体数据，但 FDA 已对该申请给予优先审评。Welireg 是一种 HIF - 2α 转录因子口服抑制剂，目前已获批用于肾细胞癌和伴有肾细胞癌的希佩尔 - 林道病患者。若获批，它将成为美国首个用于晚期 PPGL 的药物。儿科用药审批进展：Eton 等待最终定论Eton Pharmaceuticals 公司的口服氢化可的松 ET - 400 用于治疗婴儿肾上腺功能不全，FDA 将于 5 月 28 日发布审批结果。此前，FDA 因需要更多时间审查数据而推迟决定，Eton 在去年 12 月提交了补充信息。ET - 400 是一种在室温下稳定的氢化可的松口服制剂，若获批，将与 Eton 已获批的 Alkindi Sprinkle（氢化可的松口服颗粒）一起，进一步拓展公司在口服氢化可的松市场的份额，Eton 预计这两款产品每年可为公司带来超过 5000 万美元的峰值收益。新冠疫苗升级：Moderna 寻求新一代突破Moderna 的下一代新冠疫苗 mRNA - 1283 预计 5 月 31 日迎来 FDA 审批结果。2024 年 6 月，Moderna 公布了 mRNA - 1283 的 III 期数据，称其比目前已获批的 Spikevax 具有更好的疗效，能引发更高的免疫反应，尤其在 65 岁以上老年人中，对奥密克戎和原始毒株都能诱导强大的免疫反应。此前，CDC 疫苗顾问会议曾推迟讨论 mRNA - 1283，上个月终于召开。这一审批结果将影响 Moderna 在新冠疫苗市场的地位，也可能改变全球新冠疫苗的接种格局。五月的 FDA 审批潮备受瞩目，这些药物的审批结果将在未来一段时间内重塑相关疾病的治疗现状，无论是对患者、药企还是整个医疗行业都意义深远，各界正密切关注最终裁决。参考来源：https://www.biospace.com/fda/fda-action-alert-gsk-sanofi-merck-and-more识别微信二维码，可添加药时空小编请注明：姓名+研究方向！

Taylor Tieden for BioSpace On the FDA’s docket this month are two expansion bids, one for GSK’s asthma drug Nucala into COPD and another for Merck’s oral cancer drug for a pair of rare tumors. Amid the growing macro-level headwinds in the vaccine space, the FDA this month is expected to decide on Moderna’s mRNA COVID-19 shot, alongside a handful of other big verdicts. Read below for more. GSK Asks FDA to Approve Asthma Injection Nucala for COPD GSK is proposing its subcutaneous biologic Nucala to treat adult patients with chronic obstructive pulmonary disease (COPD) with an eosinophilic phenotype. The FDA’s decision is due on May 7. A pulmonary disease, COPD is characterized by inflammation in the lungs, manifesting as breathlessness, coughs and airflow obstruction. According to GSK, some 40% of patients suffer from disease exacerbations caused by type 2 inflammation, which in turn is linked to high eosinophil counts in the blood. Nucala , a monoclonal antibody, targets and blocks the IL-5 cytokine, which is crucial to maintain the activity of eosinophils. GSK is supporting Nucala’s COPD application with data from the Phase III MATINEE trial. The pharma released a topline readout from the study in September 2024, noting that Nucala elicited a “statistically significant and clinically meaningful reduction” in yearly rates of moderate or severe disease exacerbations as opposed to placebo. Nucala is currently approved for eosinophilic granulomatosis with polyangiitis and hypereosinophilic syndrome, and as an add-on maintenance treatment for asthma and chronic rhinosinusitis with nasal polyps. Arcutis Seeks Zoryve Expansion into Body and Scalp Psoriasis By May 22, the FDA will release its verdict on Arcutis Biotherapeutics’ proposed expansion for Zoryve into body and scalp psoriasis. Arcutis is backing its expansion bid with data from the Phase III ARRECTOR trial, which found that 66.4% of Zoryve-treated patients achieved clear or almost-clear skin, as opposed to 27.8% in placebo patients. Results also showed that 65.3% of patients in the Zoryve arm saw significant improvements in itch, versus 30.3% in the placebo group. Arcutis also filed findings from a Phase IIb study as well as long-term efficacy and safety data for Zoryve in plaque psoriasis. Zoryve is a topical phosphodiesterase 4 inhibitor that works by helping to suppress inflammation in the skin. The drug won FDA approval in 2011 for the treatment of plaque psoriasis in patients aged 12 years and up. If approved for body and scalp psoriasis, Zoryve would be a “truly meaningful innovation” for patients who suffer from inadequately controlled symptoms, Arcutis CEO Frank Watanabe said in a September 2024 release. Sanofi Eyes Extension of Meningococcal Disease Shot to Infants, Toddlers Sanofi’s meningococcal disease vaccine MenQuadfi is currently approved for the primary immunization of children aged two years and up. As a booster, it can be used in those 13 years and older who continue to be at risk of meningococcal disease. Sanofi is seeking to expand MenQuadfi’s label to also include infants and toddlers from six weeks to 23 months of age. The FDA is currently reviewing this proposal and is due to release its decision on May 23. If approved, MenQuadfi could protect this younger population from the A, C, W and Y serogroups of Neisseria meningitidis . In an April presentation before the CDC’s Advisory Committee for Immunization Practices, Sanofi presented data from several trials, including the Phase III MET42 study. Results showed that MenQuadfi was safe to use in infants and toddlers, with most side effects being mild or moderate in severity. Efficacy-wise, MenQuadfi was able to match a commercially approved vaccine in immune response and seroprotection. Liquidia Awaits Final Yutrepia Approval in Interstitial Lung Disease On May 23, Liquidia is expecting to win final approval of its inhalation powder Yutrepia for the treatment of patients with pulmonary arterial hypertension associated with interstitial lung disease (PH-ILD). The FDA gave the drug tentative approval in August 2024, indicating that it had cleared all efficacy and safety hurdles and satisfied regulatory standards of product quality. However, Liquidia still needed to wait out regulatory exclusivity for a competing product. Yutrepia is Liquidia’s inhalable version of treprostinil, a vasodilator that has known activity against pulmonary hypertension. Yutrepia is backed by data from the Phase III INSPIRE study, which found that the drug was both safe and well-tolerated in patients who were naïve to treprostinil or who were switching to Yutrepia from nebulized treprostinil. The INSPIRE trial found that patients treated with Yutrepia saw symptoms stabilize or improve over one year of dosing. Merck Hopes to Tack Two Rare Cancers Onto Welireg’s Label Merck is hoping to expand its oral cancer drug Welireg into advanced pheochromocytoma and paraganglioma (PPGL). The FDA’s decision deadline is May 26. PPGL refers to two types of rare tumors of the adrenal gland. Diagnosed in some 2,000 patients per year, they are typically caused by specific genetic mutations. Symptoms include high blood pressure, palpitations and headaches. According to Merck, up to 25% of PPGL cases are already at the metastatic stage at diagnosis. Merck is supporting Welireg’s PPGL bid with data from Cohort A1 of the Phase II LITESPARK-015 study. The pharma did not provide specific data in a January news release , revealing only that the application is based on objective response rate and duration of response in PPGL patients after Welireg treatment. The FDA has granted the drug priority review in this indication. Welireg is an orally available inhibitor of the HIF-2α transcription factor, which is involved in oxygen sensing and in the body’s response to hypoxia. The drug is indicated for patients with renal cell carcinoma (RCC) and those with von Hippel-Lindau disease—a rare genetic disease involving the formation of tumors across various organs—who also require treatment for associated RCC. If approved, Welireg will become the only drug for advanced PPGL in the U.S., Merck said. After Delay, Eton Nears Approval for Oral Hydrocortisone By May 28, the FDA will release its verdict on Eton Pharmaceuticals’ ET-400, an oral formulation of hydrocortisone, for the treatment of adrenal insufficiency in infants. This new target action date comes after the FDA in February announced that it needed more time to review Eton’s data package for ET-400. Eton submitted supplemental information in December last year, in response to a request from the regulator. ET-400 is a proprietary oral formulation of hydrocortisone that, according to the biotech, is stable at room temperature. If approved, ET-400 will follow in the footsteps of Eton’s Alkindi Sprinkle, which also has hydrocortisone as an active ingredient, but is formulated as oral granules. Alkindi Sprinkle, approved in 2020 , is designed to rapidly deliver glucocorticoids to children with adrenocortical insufficiency. In a July 2024 announcement , Eton CEO Sean Brynjelsen said that the approval of ET-400 would allow the biotech to “capture a greater percentage of the oral hydrocortisone market” and, alongside Alkindi Sprinkle, would open up a peak opportunity of “more than $50 million annually.” Moderna Seeks Approval for Next-Generation COVID-19 Shot The FDA’s last big milestone for the month belongs to Moderna and its investigational next-generation COVID-19 vaccine mRNA-1283. The regulator is expected to release its decision on May 31. In June 2024, the company released Phase III data for the experimental shot, touting better efficacy than Spikevax, its currently approved COVID-19 vaccine. Moderna did not provide specific data, only claiming that mRNA-1283 elicited a “higher immune response” against SARS-CoV-2 than Spikevax. Of note, Moderna emphasized at the time that mRNA-1283 could induce robust responses against Omicron and original strains of the virus—a benefit the company claimed was most evident in seniors over 65 years of age. The CDC’s vaccine advisors were supposed to convene in late February to discuss various vaccines, including mRNA-1283, before the meeting was postponed . The panel finally convened last month.

- NDA submitted for the treatment of central diabetes insipidus -- Expected 10-month review; commercial preparations underway for a potential Q1 2026 launch – DEER PARK, Ill., April 28, 2025 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or the “Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for ET-600, Eton’s proprietary, patented oral solution of desmopressin under development for the treatment of central diabetes insipidus. The Company expects the application to be assigned a 10-month FDA review, allowing for potential approval and launch in the first quarter of 2026. “The NDA submission for ET-600 is another important milestone for Eton, and for the thousands of children impacted by diabetes insipidus. If approved, ET-600 will be the only FDA-approved oral liquid formulation of desmopressin, providing the small, precise, and titratable doses required to treat pediatric patients,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals. “Leading pediatric endocrinologists have long expressed a need for this product, and we’re pleased to be one step closer to bringing it to patients. Pre-launch commercial activities are already underway as we plan for a potential approval in the first quarter of 2026.” Eton has been issued a patent by the U.S. Patent and Trademark Office on ET-600, which expires in 2044 and has additional patent applications under review. In a bioequivalence study conducted in 75 human subjects, ET-600 demonstrated pharmacokinetic equivalence to the FDA-approved reference product of the same active ingredient. The company estimates that central diabetes insipidus impacts approximately 3,000 pediatric patients in the United States. About Eton Pharmaceuticals Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has seven commercial rare disease products: INCRELEX®, ALKINDI SPRINKLE®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has six additional product candidates in late-stage development: ET-400, ET-600, Amglidia®, ET-700, ET-800 and ZENEO® hydrocortisone autoinjector. For more information, please visit our website at www.etonpharma.com. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the expected ability of Eton to undertake certain activities and accomplish certain goals and objectives. These statements include but are not limited to statements regarding Eton’s business strategy, Eton’s plans to develop and commercialize its product candidates, the safety and efficacy of Eton’s product candidates, Eton’s plans and expected timing with respect to regulatory filings and approvals, and the size and growth potential of the markets for Eton’s product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “intends,” “will,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Eton’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. These and other risks concerning Eton’s development programs and financial position are described in additional detail in Eton’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Eton undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Investor Relations:Lisa M. Wilson, In-Site Communications, Inc.T: 212-452-2793E: lwilson@insitecony.com Source: Eton Pharmaceuticals, Inc.