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Novartis says rare muscle disease drug shows promise in early trial

By Thomson Reuters Jun 11, 2026 | 4:41 PM

June 11 (Reuters) – Novartis 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.

(Reporting by Kunal Das ​in Bengaluru)