Novartis: Rare muscle disease drug shows early promise
Novartis (NOVN.S), opens new tab said on Thursday an experimental drug, which it acquired as part of its $12 billion takeover of Avidity, showed promise in an early-to-mid-stage study in patients with a type of genetic muscle disorder characterized by slowly progressive muscle weakness.
- The Swiss drugmaker said the drug, known as del-brax, lowered two blood markers linked to the disease and showed reduced signs of muscle damage in patients with facioscapulohumeral muscular dystrophy.
- Novartis said the drug shows potential to become the first disease-modifying treatment for FSHD, which can cause weakness in the face, shoulders, arms and other muscles.
- The company estimates it affects about 45,000 to 87,000 people in the U.S. and EU.
- The drug’s safety profile was consistent with previous results, the company said.
- Novartis plans to discuss the data with health regulators around the world, while a late-stage study of the drug is currently enrolling patients.
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