Mirum Pharmaceuticals

Mirum Pharmaceuticals' LIVMARLI (maralixibat) has received FDA approval for a new tablet formulation to treat cholestatic pruritus in patients with Alagille syndrome and Progressive Familial Intrahepatic Cholestasis.

• The FDA has approved novel therapies for Duchenne Muscular Dystrophy, including ITF Therapeutics' Duvyzat (givinostat), which targets histone deacetylases to potentially slow muscle deterioration. • Breakthrough gene therapy Lenmeldy received approval for Metachromatic Leukodystrophy, offering the first targeted treatment option with significant survival benefits for pediatric patients. • Multiple rare diseases saw first-ever treatments, including Xolremdi for WHIM syndrome, dual approvals for Niemann-Pick Disease Type C, and Ctexli for Cerebrotendinous Xanthomatosis.

The FDA has granted historic approval to chenodiol tablets (Ctexli) as the first-ever treatment for cerebrotendinous xanthomatosis, a rare genetic lipid storage disease affecting multiple organ systems.

A Phase 3 clinical trial (EXPAND) is underway to assess maralixibat's efficacy in treating pruritus associated with rare cholestatic conditions, excluding Alagille syndrome and PFIC.

• The FDA is set to decide on Ionis Pharmaceuticals' olezarsen for familial chylomicronemia syndrome (FCS), a rare genetic disease, by December 19, following positive Phase III results. • Lexicon Pharmaceuticals awaits the FDA's decision on sotagliflozin as an adjunct therapy for type 1 diabetes by December 20, despite concerns raised by an advisory committee. • AstraZeneca and Daiichi Sankyo's Dato-DXd for non-squamous NSCLC is under FDA review, with a decision expected by December 20, based on Phase III data showing progression-free survival benefits. • Neurocrine Biosciences anticipates FDA decisions on crinecerfont for congenital adrenal hyperplasia (CAH) by December 29 and 30, potentially marking the first new treatment in 70 years.

Mirum Pharmaceuticals plans to initiate a Phase 2 clinical trial of MRM-3379, an oral treatment for fragile X syndrome, in 2025.

GSK's linerixibat met the primary endpoint in the Phase III GLISTEN study, demonstrating a statistically significant improvement in itch score for PBC patients.

The FDA granted Breakthrough Therapy Designation to Mirum Pharmaceuticals' volixibat for treating cholestatic pruritus in patients with primary biliary cholangitis (PBC).

The FDA granted Breakthrough Therapy Designation to volixibat for treating cholestatic pruritus in patients with primary biliary cholangitis (PBC).

The FDA has approved Mirum Pharmaceuticals' Livmarli oral solution for treating cholestatic pruritus in PFIC patients aged 12 months and older.