Group 1 - Sanofi's investigational BTK inhibitor, rilzabrutinib, received orphan drug designation from the European Medicines Agency (EMA) for treating IgG4-related disease (IgG4-RD) [1][7] - IgG4-RD is a chronic, immune-mediated rare condition that can affect multiple organs, leading to inflammation, swelling, and fibrosis [1] - Rilzabrutinib is currently undergoing a phase II study for IgG4-RD, showing a reduction in disease flare and glucocorticoid sparing over 52 weeks [2][7] Group 2 - Rilzabrutinib is also being developed for immune thrombocytopenia (ITP) and has orphan drug designation for this indication in the United States, EU, and Japan [3] - A regulatory application for rilzabrutinib's approval for ITP is under review in the U.S., with a decision expected by August 29, 2025 [4] - The FDA has granted orphan drug designation to rilzabrutinib for warm autoimmune hemolytic anemia (wAIHA) and sickle cell disease (SCD) [5] Group 3 - Upon potential approval, rilzabrutinib may face competition from existing therapies, such as Rigel Pharmaceuticals' Tavalisse, which recorded $68.5 million in sales in the first half of 2025, a 44% increase year-over-year [6] - Amgen's Uplizna became the first FDA-approved drug for IgG4-RD in April 2025, following its acquisition of Horizon Therapeutics [8]

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]