Belzutifan

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.

声明：因水平有限，错误不可避免，或有些信息非最及时，欢迎留言指出。本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及）;本文不构成任何投资建议。在5月，将有4款重磅药物有望获得批准，其中包括：潜在首个获批用于COPD患者每月给药的生物制剂mepolizumab；罗氟司特泡沫（0.3%）新适应症，用于头皮和全身银屑病；潜在用于治疗符合资格的晚期PPGL患者的首款疗法Welireg以及Moderna下一代COVID-19疫苗mRNA-1283。Mepolizumab适应症：嗜酸性慢性阻塞性肺疾病PDUFA日期：2025年5月7日12月9日，GSK宣布，FDA已接受MATINEE研究数据的审查，以支持美泊利珠单抗（Nucala，mepolizumab）作为嗜酸性表型慢性阻塞性肺病（COPD）患者的附加维持治疗药物获得新适应症的监管审查程序，PDUFA日期为2025年5月7日。该药物有望成为首个获批用于COPD患者每月给药的生物制剂。Mepolizumab（美泊利珠单抗）于2015年11月获FDA批准上市，是一种抗IL-5的人源化单克隆抗体，可阻断IL-5与嗜酸粒细胞表面上IL-5受体α链的相互作用，进而阻断IL-5对嗜酸性粒细胞等效应细胞的作用。BLA得到了3期MATINEE试验(NCT04133909)数据的支持，该研究是一项为期52-104周的多中心、随机、双盲、安慰剂对照临床试验（n=806），评估了美泊利珠单抗（100mg，每4周1次，皮下注射）对比安慰剂作为疾病频繁恶化且血嗜酸性粒细胞水平升高的中度至重度COPD患者在维持治疗之外的附加治疗方案的有效性和安全性。研究的主要终点为中度至重度COPD的年恶化率。结果显示，治疗104周后，美泊利珠单抗组患者的年恶化率低于安慰剂组，数据具有统计学意义和临床意义。罗氟司特泡沫（0.3%）适应症：头皮和身体牛皮癣PDUFA日期：2025年5月22日7月23日，Arcutis Biotherapeutics宣布罗氟司特泡沫（0.3%，每日1次）治疗头皮银屑病和全身银屑病的新药补充申请（sNDA）获FDA受理。罗氟司特泡沫（0.3%）是Arcutis开发的一种改良型新药，于2023年12月在美国获批上市，用于治疗脂溢性皮炎。Arcutis还开发了一款罗氟司特乳膏（0.3%），该产品已于2022年7月获FDA批准上市，适应症为斑块状银屑病。2023年8月，华东医药与Arcutis达成合作，引进了罗氟司特泡沫和罗氟司特乳膏在大中华区和东南亚地区的开发、注册、生产和商业化权益。此次sNDA主要是基于III期ARRECTOR研究的积极结果。该研究是一项随机、双盲、安慰剂对照临床试验（N=432），评估了罗氟司特泡沫（0.3%）治疗12岁及以上青少年和成人头皮银屑病和全身银屑病患者的疗效和安全性。研究结果显示，与安慰剂相比，罗氟司特泡沫（0.3%）在头皮和身体银屑病症状方面有统计学显著改善。据报道，使用罗氟司特泡沫（0.3%）后，头皮和身体瘙痒明显减轻。Welireg适应症：晚期嗜铬细胞瘤和副神经节瘤PDUFA日期：2025年5月26日2025年1月27日，默沙东宣布美国FDA已受理其缺氧诱导因子-2α（HIF-2α）抑制剂Welireg（belzutifan）的补充新药申请（sNDA），并授予优先审评资格，用以治疗12岁及以上的晚期、不可切除或转移性嗜铬细胞瘤和副神经节瘤（PPGL）患者。此次申请基于LITESPARK-015临床2期研究的客观缓解率（ORR）和缓解持续时间（DOR）数据。该申请的PDUFA日期为2025年5月26日。根据新闻稿，如果获批，Welireg可能成为美国FDA批准用于治疗符合资格的晚期PPGL患者的首款疗法。Welireg是FDA加速批准的首个HIF-2α抑制剂，用于需要治疗的成人von Hippel-Lindau（VHL）疾病患者，特别是那些伴有相关透明细胞肾细胞癌（RCC）、中枢神经系统血管母细胞瘤或胰腺神经内分泌肿瘤且不需要立即手术的情况。2023年12月，Welireg获美国FDA批准成为首个获批用于晚期RCC患者的HIF-2α抑制剂。Welireg的获批标志着自2015年以来，晚期RCC首次有新机制疗法获得批准。mRNA-1283适应症：COVID-19PDUFA日期：2025年5月31日FDA已接受Moderna下一代COVID-19疫苗mRNA-1283的生物制品许可申请 (BLA)，并指定了PDUFA 目标日期为2025年5月31日。mRNA-1283 的设计旨在增强冷藏条件下的稳定性，并将采用预充式注射器包装，这为流感和新冠联合疫苗的研发铺平了道路，从而增强了公司的整体呼吸系统产品组合。根据第3阶段NextCOVE研究(NCT05815498)的结果，mRNA-1283与 Spikevax 相比表现出非劣效的相对疫苗效力 (rVE）。Spikevax是Moderna已批准上市的一款mRNA新冠疫苗，能编码全长SARS-CoV-2 S蛋白。该疫苗在2020年的关键性试验中达到了94.5%的有效率。然而，实际使用表明Spikevax还有提升空间，尤其是在对抗新变异株方面，另外其还需简化物流。mRNA-1283疫苗则是Moderna尝试实现这些改进的下一代新冠疫苗。参考资料：各公司公告