Tag: genetic disorder

European Commission approval expands the indication of lomitapide in the European Union (EU) to include children 5 years of age and older with HoFH, an ultra-rare genetic disorder affecting LDL-cholesterol levels

Zurich, July 29, 2025 – MetrioPharm AG, a pharmaceutical company developing drugs for inflammatory and infectious diseases, announced today that the European Medicines Agency has granted Orphan Drug Designation (ODD) to its lead compound, MP1032 for the treatment of children affected by Duchenne muscular dystrophy (DMD). This milestone follows the U.S. FDA's ODD, underscoring MP1032's potential as a first-in-class therapy that could minimize reliance on high-dose corticosteroids, which cause severe side effects like osteoporosis, growth stunting, and metabolic issues.

– EZMEKLY is the first and only therapy to receive marketing authorization in the EU for both adults and children (≥2 years) with NF1-PN, a rare genetic disorder with debilitating symptoms –

– EZMEKLY is the first and only therapy to receive marketing authorization in the EU for both adults and children (≥2 years) with NF1-PN, a rare genetic disorder with debilitating symptoms –

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