FDA grants rare pediatric disease designation to Descartes-08 for juvenile dermatomyositis

Key takeaways:

• Descartes-08 is an autologous mRNA cell therapy indicated for many autoimmune diseases.
• The FDA granted the designation to the drug for juvenile dermatomyositis due to the life-threatening nature of the disease.

The FDA has granted rare pediatric disease designation to Descartes-08, an autologous mRNA cell therapy, for the treatment of juvenile dermatomyositis, Cartesian Therapeutics announced in a press release.

Juvenile dermatomyositis is a rare pediatric autoimmune disorder that manifests as a pathognomonic skin rash accompanied by muscle inflammation that affects the joints, heart, lungs and other organs. Affecting only about 4,000 people in the U.S., juvenile dermatomyositis can be life-threatening.

“We are pleased that the FDA recognizes the potential of Descartes-08 to serve as a meaningful therapeutic option for this underserved pediatric patient population,” Carsten Brunn, PhD, president and CEO of Cartesian, said in the release. “Leveraging our novel mRNA platform, we are committed to our mission of expanding the reach of cell therapy to patients with autoimmune diseases.”

Granted only for serious and life-threatening diseases, the FDA’s rare pediatric disease designation allows drugs to receive priority review once the drug is approved for marketing in an effort to quickly treat highly at-risk populations.

According to the press release, Cartesian Therapeutics is currently investigating Descartes-08 in phase 2b trials for multiple autoimmune indications and is also pursuing a new drug application for the drug.

“We remain on track to file an investigational new drug application for a phase 2 pediatric basket study focused on neurology and rheumatology autoimmune indications, including [juvenile dermatomyositis], by year-end,” Brunn said.