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.

 The Thomson Reuters