With two drugs cleared by the U.S. FDA for Niemann-Pick disease type C (NPC) in less than seven days, Wall Street was pondering the differences between the compounds, given what’s known so far about each. Most recently, the FDA approved Intrabio Inc.’s Aqneursa (levacetylleucine) on its PDUFA date for the treatment of neurological manifestations of NPC in adults and pediatric patients weighing at least 15 kg, making Aqneursa the only approved stand-alone therapy indicated for NPC. On Sept. 20, Zevra Therapeutics Inc. won FDA clearance for Miplyffa (arimoclomol) as the first treatment for NPC.

At the 2024 European College of Neuropsychopharmacology (ECNP) Congress, researchers have presented work that could lead to ways to boost brain development and prevent neurodegeneration in individuals with Down syndrome.

Chimeric antigen receptor (CAR) T-cell therapy moved the needle at the 2024 ECTRIMS (European Committee for Treatment and Research in Multiple Sclerosis) congress and will continue to do so – not just in multiple sclerosis (MS), but in other autoimmune diseases as well.

The Annual Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) meeting in Copenhagen this week is celebrating its 40th edition. In recognition of this landmark, the plenary session and opening lecture were attended by Queen Margrethe of Denmark. Afterward, the hot topic session on neuroprotective therapies set the stage for the subsequent discussions on the latest trends in the management and treatment of multiple sclerosis (MS).

As Novo Nordisk A/S and Eli Lilly and Co. go head-to-head in the U.S. and Chinese glucagon-like peptide-1 receptor agonists (GLP-1RA) market for diabetes and obesity, Novo Nordisk is in innovator gear once more with leading studies of GLP-1s in Alzheimer’s disease.

Chinese pharmaceutical and biotech companies are leading development of glucagon-like peptide-1 receptor agonists as Novo Nordisk A/S and Eli Lilly and Co. edge closer to launching blockbuster therapies in China. At the heart of the GLP-1 boom is a nationwide obesity problem driven by a confluence of factors, including the rise of a modern, sedentary lifestyle, according to Clarivate. Despite the rising prevalence of obesity and type 2 diabetes, the gap in obesity therapeutics is “substantial and leaves a solid market opportunity for weight loss drugs,” Karan Verma, principal analyst of healthcare research & data analytics at Clarivate, said.

A novel gene therapy that leads to cellular rejuvenation could restore vision after non-arteritic anterior ischemic optic neuropathy (NAION) and glaucoma. The technique is based on a reprogramming process that reverses the epigenetic DNA alterations caused by aging. Preclinical studies in glaucoma mice and nonhuman primates (NHP) models for this stroke-like disorder that affects the eye, showed an improvement of vision and restoration of the damaged axons of the optic nerve.

Two leading glucagon-like peptide-1 (GLP-1) receptor agonists for obesity and type 2 diabetes – Novo Nordisk A/S’s semaglutide (Wegovy/Ozempic) and Eli Lilly and Co.’s tirzepatide (Mounjaro/Zepbound) – are advancing in China after taking the U.S. market by storm. China represents the world’s largest population of diabetes and obesity patients. Its GLP-1 market, valued at about $1.7 billion in 2023 according to Clarivate, is expected to grow as the number of obesity patients is projected to exceed 500 million by 2033.