FDA grants orphan drug designation to myotonic dystrophy treatment

Key takeaways:

• The treatment, JUV-161, is Juvena’s lead candidate of engineered biologics for myopathies and metabolic diseases.
• Human clinical trials of JUV-161 are expected to begin in 2024.

The FDA has granted orphan drug designation to an investigational therapeutic to treat myotonic dystrophy type 1, the most common form of muscular dystrophy in adults, according to its manufacturer.

According to a release from Juvena Therapeutics, JUV-161 is the company’s lead candidate of engineered biologics for multiple myopathies and metabolic diseases. Human trials of JUV-161, which possesses an IGF-2 protein developed for subcutaneous administration, are expected to begin this year.

“People living with DM1 deserve safe, effective, and rejuvenating treatments that can repair and restore tissue health to improve muscle function and metabolism,” Juvena Therapeutics CEO and co-founder Hanadie Yousef, PhD, said in the release. “As we advance Juvena’s first biologic initially focused on the treatment of [myotonic dystrophy type 1], we look forward to exploring potential treatments for an entire class of disorders that remain major unmet medical needs.”

The FDA grants orphan status to support development of therapeutics for rare disorders affecting fewer than 200,000 people in the United States, along with certain benefits, including market exclusivity upon regulatory approval, exemption of FDA application fees and tax credits for qualified clinical trials.

“We are excited about the unique approach Juvena Therapeutics is advancing because of its potential to safely promote muscle regeneration, improve metabolism, rejuvenate lives and ultimately to more holistically address the disease,” Myotonic Dystrophy Foundation CEO Tanya Stevenson, Ed.D, MPH, said in the release.