Core Insights - The U.S. FDA granted orphan drug designation to Sanofi SA's rilzabrutinib for two rare diseases: warm autoimmune hemolytic anemia (wAIHA) and IgG4-related disease (IgG4-RD) [1] - Rilzabrutinib is under regulatory review in the US, EU, and China for immune thrombocytopenia (ITP) [2] - The FDA's target action date for the regulatory decision on ITP is August 29, and rilzabrutinib has also received orphan drug designation for ITP in the US, EU, and Japan [3] Clinical Study Results - Phase 2b study results for wAIHA presented at ASH 2024 indicated that rilzabrutinib treatment led to clinically meaningful outcomes in response rate and disease markers [3] - In a phase 2a study for IgG4-RD, rilzabrutinib treatment over 52 weeks resulted in reduced disease flare and other disease markers, along with glucocorticoid sparing [4] - The safety profile of rilzabrutinib in both studies was consistent with previous studies [4] Recent Developments - The FDA recently approved Sanofi's Qfitlia (fitusiran), the first antithrombin-lowering therapy for hemophilia A or B, based on data from the ATLAS phase 3 studies [5] - Sanofi's stock increased by 3.54% to $55.86 during the premarket session following these developments [6]

Core Insights - The FDA has granted orphan drug designation to rilzabrutinib for two rare diseases, warm autoimmune hemolytic anemia (wAIHA) and IgG4-related disease (IgG4-RD), which currently have no approved treatments [1][2] - Rilzabrutinib is also under regulatory review for immune thrombocytopenia (ITP) in the US, EU, and China, with a target action date for FDA decision set for August 29, 2025 [2][8] Rilzabrutinib Overview - Rilzabrutinib is an investigational, oral, reversible Bruton's tyrosine kinase (BTK) inhibitor, showing potential as a first- and best-in-class treatment for several immune-mediated diseases [5] - The drug utilizes Sanofi's TAILORED COVALENCY® technology to selectively inhibit BTK, potentially minimizing off-target side effects [5] Clinical Data - A phase 2b study on wAIHA indicated that rilzabrutinib treatment resulted in clinically meaningful outcomes regarding response rates and disease markers [3] - In a phase 2a study for IgG4-RD, rilzabrutinib treatment over 52 weeks led to a reduction in disease flare and other disease markers, along with glucocorticoid sparing [4] Disease Background - wAIHA affects 1 to 3 individuals per 100,000 in the US annually and is characterized by the premature destruction of red blood cells, leading to severe fatigue and other symptoms [6] - IgG4-RD affects approximately 8 out of 100,000 adult patients in the US each year and is a chronic condition that can cause organ damage and dysfunction [7]

Core Insights - Pharvaris has received orphan designation from the European Commission for its investigational drug, deucrictibant, aimed at treating bradykinin-mediated angioedema [1][3] - The U.S. FDA had previously granted orphan drug designation to deucrictibant in March 2022 [2] - The company is currently executing a Phase 3 development program to evaluate the efficacy and safety of deucrictibant in hereditary angioedema (HAE) [3][5] Company Overview - Pharvaris is a late-stage biopharmaceutical company focused on developing oral bradykinin B2 receptor antagonists to address bradykinin-mediated diseases [1][5] - The company aims to provide injectable-like efficacy with the convenience of oral therapy for preventing and treating angioedema attacks [5] - Deucrictibant is being developed in two formulations: an extended-release tablet for sustained absorption and an immediate-release capsule for rapid onset of action [4] Clinical Development - Pharvaris is conducting pivotal Phase 3 studies for both the prevention of HAE attacks (CHAPTER-3) and the on-demand treatment of HAE attacks (RAPIDe-3) [5] - The company is also in discussions with regulators regarding a pivotal trial for acquired angioedema due to C1 inhibitor deficiency (AAE-C1INH) [3]

Core Viewpoint - Palatin Technologies, Inc. has received FDA orphan drug designation for PL7737, an oral treatment for leptin receptor deficiency-related obesity, which could offer a more convenient option compared to the current injectable treatment [1][2]. Company Overview - Palatin Technologies is a biopharmaceutical company focused on developing first-in-class medicines that modulate the melanocortin receptor system, targeting diseases with significant unmet medical needs [6]. - The company is exploring PL7737 for hypothalamic obesity and plans to initiate a Phase 1 study in late 2025 [2]. Clinical Development - The FDA orphan drug designation is a significant milestone for Palatin's MC4R receptor agonists aimed at rare obesity conditions [2]. - Palatin has completed statistical analysis for its Phase 2 clinical studies involving MC4R bremelanotide and GLP-1/GIP tirzepatide for obesity, as well as PL8177 for ulcerative colitis, with topline data expected to be released soon [2]. Mechanism of Action - PL7737 acts as an MC4R agonist, designed to restore impaired signaling due to genetic mutations in the LEPR gene, which is crucial for regulating hunger and body weight [2][4]. - The melanocortin receptor system plays a vital role in various physiological processes, including metabolism and food intake, making it a promising target for obesity treatments [5]. Regulatory Insights - The FDA's orphan drug designation provides several incentives, including tax credits for clinical trials, exemption from user fees, and potential market exclusivity for seven years post-approval [7].