Riliprubart granted orphan drug designation in Japan for chronic inflammatory demyelinating polyneuropathy Two phase 3 studies are currently underway testing riliprubart in people with CIDP as a potential first-in-class treatmentJapanese Ministry of Health, Labour and Welfare decision adds to similar designations in the US and Europe, underscoring global regulatory recognition of the potential for riliprupart to address significant unmet medical needs for people living with this rare neurological condition ...

采写：科技日报记者 吴纯新 俞慧友 陈 曦 策划：赵英淑 滕继濮 近日，一名游客在海南省三亚市被蛇咬伤后不幸离世。这场意外引发公众对抗蛇毒蛋白血清产能不足问 题的关注。 其实相较于抗蛇毒蛋白血清，在全球范围内，有一种药物更加稀缺——罕见病药物。已知的罕见病已超 过7000种，患者总数超过3亿，但其中仅有不到10%的疾病拥有获批的治疗药物。在我国，罕见病患者 人数已突破2000万，且每年新增患者超20万。 罕见病不仅种类繁多，而且多数患者病情严重、诊断困难。然而，由于患者群体规模小、药物研发成本 高昂、市场回报有限，制药企业往往缺乏研发动力。这类药物也因稀缺而被称为"孤儿药"。 面对罕见病患者群体，如何打破市场惰性，破解"孤儿药"的研发与供应困境，已成为全球性挑战。 罕见病药物研发面临困难 佳佳1岁时被确诊杜氏肌营养不良症。这是一种因抗肌肉萎缩蛋白缺失而导致的遗传性疾病。我国目前 还没有治疗该疾病的特效药，只能依赖波尼松等激素药物延缓病情。 "2023年，国外出了一种迷你蛋白，药效不错，但一次性用药花费高达320万美元。"屈晓燕说，国外这 几年还研发出几款"跳跃药"，对孩子治疗都有帮助，可价格都很昂贵。 "天价 ...

Riliprubart earns orphan drug designation in the US for antibody-mediated rejection in solid organ transplantation Ongoing phase 2 study evaluating riliprubart for the potential prevention and treatment of active antibody-mediated rejection in kidney transplant recipientsRiliprubart was also designated orphan drug for the investigational use in chronic inflammatory demyelinating polyneuropathy in the US and EU Paris, June 25, 2025. The US Food and Drug Administration (FDA) has granted orphan drug designatio ...

TORONTO, June 24, 2025 (GLOBE NEWSWIRE) -- PharmaTher Holdings Ltd. (OTCQB: PHRRF) (CSE: PHRM) ("PharmaTher" or the "Company"), a company focused on unlocking the pharmaceutical potential of ketamine, is pleased to announce that the United States Patent and Trademark Office (USPTO) has granted U.S. Patent No. 12,128,012, expiring May 14, 2041, for the use of ketamine in the treatment of Amyotrophic Lateral Sclerosis (ALS). This newly issued patent significantly strengthens the Company's intellectual propert ...

｜2025年6月18日星期三｜ NO.4默沙东长效RSV单抗在中国拟纳入优先审评 CDE（国家药品监督管理局药品审评中心）官网显示，默沙东的 Clesrovimab（MK-1654)）注射液上市 申请拟纳入优先审评，适应证为用于即将进入或出生在第一个呼吸道合胞病毒（RSV）流行季的新生儿 和婴儿预防 RSV 所致的下呼吸道感染。 JZ-14胶囊为云南白药控股子公司征武科技自主研发的化学1类创新药，是First-in-Class（首创新药）的 小分子免疫调节剂。JZ-14可选择性结合特定的蛋白靶点，抑制其下游AKT/NF-κB等信号通路，从而在 溃疡性结肠炎与多种肿瘤模型中展现出显著的免疫调节及抗增殖活性。 点评：JZ-14胶囊若临床转化成功，不仅可填补免疫调节领域空白，还能拓展云南白药在化药创新领域 的布局。不过，需关注其临床安全性和疗效验证，以及未来与生物药的竞争态势。 NO.3 荣昌生物泰它西普获欧盟孤儿药资格认定 荣昌生物的产品泰它西普（商品名：泰爱®，研发代号：RC18）获得欧盟委员会（EC）授予的孤儿药 资格认定（Orphan Drug Designation，ODD），用于治疗重症肌无力。 点评： ...

Avadel Pharmaceuticals (AVDL) 2025 Conference June 05, 2025 10:30 AM ET Speaker0 Okay. Alright. We're gonna get started in our next session. I'm Andrew Tsai, senior biotech analyst at Jefferies. Thanks for tuning in. It's my pleasure to have the AVID all team. To my direct right is Greg Divis, CEO. To his right, Tom McHugh, CFO, and to his right, Susan, I believe I'm sorry. I missed your last name. Speaker1 Rodriguez. Speaker0 Rodriguez. Yes. Chief Commercial Officer? Speaker2 Chief Operating Officer. Speak ...

复星医药（600196）公告，控股子公司Henlius Europe GmbH的HLX22(重组人源化抗HER2单克隆抗体 注射液)用于胃癌治疗获得欧盟委员会孤儿药资格认定。HLX22为公司自AbClon,Inc.许可引进并自主研 发，拟用于胃癌和乳腺癌等实体瘤治疗。2025年3月，HLX22已获美国食品药品监督管理局授予孤儿药 资格认定。截至2025年4月，公司针对HLX22的累计研发投入约为3.1亿元。根据IQVIA MIDAS 最新数 据，2024年全球范围内靶向HER2的单抗产品销售额约为90.29亿美元。本次认定有助于HLX22在欧盟的 后续研发、注册及商业化等方面享受政策支持。 ...

Financial Data and Key Metrics Changes - At the end of Q1 2025, the company had over $172 million in cash and short-term investments [23] - A $200 million debt facility was executed with Hercules, providing a cash runway into the second half of 2027 [23] Business Line Data and Key Metrics Changes - The company is focused on a single development program for a rare disease called autoimmune pulmonary alveolar proteinosis (APAP) [2] - The Phase III pivotal clinical trial IMPALA two showed statistically significant improvement in DLCO compared to placebo at week 24, with sustained improvement at week 48 [7][8] - 97% of patients completed the double-blind treatment period, with no withdrawals due to drug-related adverse events [8] Market Data and Key Metrics Changes - The diagnosed prevalence of APAP in the U.S. is estimated at approximately 3,600 patients, with an additional 3,700 likely undiagnosed patients [13][14] - The potential market opportunity in the U.S. is significant, with a total of over 7,000 patients identified [15] Company Strategy and Development Direction - The company aims to establish relationships with pulmonologists and treatment centers to gain visibility into the patient population before the launch of Molbrevi [17] - A U.S. commercial team is being built prior to approval, consisting of 25 to 30 individuals responsible for patient profiling and disease awareness campaigns [22] - The pricing power for Molbrevi is projected between $300,000 and $500,000 per patient per year, aligning with other orphan drug analogues [24] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of Molbrevi, highlighting the lack of approved medicines for APAP in the U.S. and Europe [4][11] - The company anticipates a potential PDUFA date around November if priority review is granted by the FDA [11] - There is a strong interest from U.S. pulmonologists and payers regarding Molbrevi, with 83% of pulmonologists likely to prescribe it [18][19] Other Important Information - The company launched a free blood antibody test called APAP ClearPath to facilitate quicker diagnosis of APAP [20] - The test has been piloted at an interstitial lung disease clinic, aiming to identify undiagnosed APAP patients [21] Q&A Session Summary Question: What is the current status of the regulatory submission for Molbrevi? - The company completed the submission of the BLA to the FDA and is awaiting feedback within a 60-day window [11] Question: How many patients does the company aim to reach by launch? - The company aims to have line of sight to 1,000 known APAP patients by launch, with a goal to confirm the total of 3,600 patients [15][17] Question: What are the expectations regarding payer coverage for Molbrevi? - 87% of payers indicated they intend to provide coverage with simple pre-authorization criteria, recognizing the disease burden of APAP [19]

Jaguar Health (JAGX) Q1 2025 Earnings Call May 15, 2025 04:15 PM ET Company Participants Lisa Conte - Founder, CEO, President & DirectorCarol Lizak - Chief Financial Officer Operator Greetings, and welcome to Jaguar Health's May fifteen, twenty twenty five Investor Webcast. Before I turn the call over to management, I'd like to remind you that management may look forward looking statements relating to such matters as continued growth prospects for the company, uncertainties regarding market acceptance of pr ...

Core Insights - The FDA has granted Orphan Drug Designation (ODD) to NeuroNOS's lead investigational therapy, BA-102, for treating Phelan-McDermid Syndrome (PMS), a condition associated with Autism Spectrum Disorder (ASD) [1][3] - NeuroNOS plans to initiate first-in-human clinical trials for ASD in the U.S. in 2026 [1] Company Overview - NeuroNOS is a biopharmaceutical company focused on developing treatments for autism and Alzheimer disorders, and is a subsidiary of Beyond Air [1][5] - The company specializes in therapies based on small molecules that can cross the blood-brain barrier to regulate Nitric Oxide (NO) levels in the brain [5] Disease Background - Phelan-McDermid Syndrome (PMS) is a rare genetic disorder primarily caused by deletions or mutations affecting the SHANK3 gene, leading to symptoms such as global developmental delay, intellectual disability, and severe speech impairments [2][4] - There are currently no FDA-approved treatments specifically indicated for PMS [2] Development Incentives - The ODD provides several key development incentives, including seven years of market exclusivity upon approval, tax credits for qualified clinical trials, waiver of FDA application fees, and access to FDA protocol assistance [3] Commitment to Development - NeuroNOS is committed to collaborating with the FDA, patient advocacy groups, scientific foundations, and clinical investigators to accelerate the development of therapies for PMS [4]