- In March 2025, Catalyst Pharmaceuticals submitted a New Drug Application to the FDA for an extended-release amifampridine formulation.Catalyst Pharmaceuticals advanced its drug pipeline by filing a New Drug Application (NDA) with the U.S. FDA for an extended-release version of amifampridine. This formulation is designed to maintain stable therapeutic levels over a longer period, potentially reducing dosing frequency and improving patient adherence. If approved, it would offer a more convenient alternative for LEMS patients currently reliant on multiple daily doses
- In December 2024, Grifols announced the expansion of its IVIG production facility to meet rising global demand for rare disease treatments.Grifols responded to growing demand for intravenous immunoglobulin (IVIG) therapies, commonly used in the treatment of neuromuscular autoimmune disorders like LEMS, by expanding its manufacturing capacity. The facility upgrade aims to enhance global supply resilience and reduce shortages, particularly as demand for rare disease therapies increases across developed and emerging markets
- In August 2024, Hansa Biopharma began clinical trials for its anti-IgG enzyme therapy in antibody-positive LEMS patients.Hansa Biopharma initiated clinical trials of its investigational anti-IgG enzyme therapy, which is designed to break down pathogenic antibodies in patients with antibody-positive LEMS. This targeted approach seeks to offer a novel immunomodulatory treatment pathway, potentially benefiting patients who do not respond adequately to current potassium channel blockers or immunoglobulin therapies
- In May 2024, Takeda initiated a multicenter study on its investigational immunotherapy for refractory neuromuscular autoimmune syndromes.Takeda Pharmaceuticals launched a multicenter clinical study evaluating the efficacy of a novel immunotherapy for patients with refractory neuromuscular autoimmune syndromes, including LEMS. The study aims to explore alternative pathways for disease control in patients who have failed to respond to existing treatments, reinforcing the company’s focus on advancing care in rare and underserved indications
