The US Food and Drug Administration (FDA) has granted Rare Paediatric Disease designation to clinical-stage drug development company Catabasis Pharmaceuticals Inc's CAT-1004 for the treatment of Duchenne muscular dystrophy (DMD).

CAT-1004 is designed to inhibit activated NF-kB, a protein that plays an important role in muscle health. CAT-1004 has the potential to reduce muscle inflammation and degeneration and promote muscle regeneration in patients with DMD regardless of the underlying dystrophin mutation. The FDA has previously granted Fast Track and Orphan Drug designations for CAT-1004 for the treatment of DMD.

“We are pleased that the FDA's Office of Orphan Products Development has granted CAT-1004 a Rare Paediatric Disease designation. CAT-1004 has the potential to make a meaningful impact for the patients and families affected by DMD through a disease-modifying mechanism that could be applicable to all patients with this disorder,” said Jill C. Milne, Ph.D., co-founder and chief executive officer of Catabasis.

“We look forward to continuing to advance this product candidate in our phase 1/2 MoveDMD clinical trial, which is currently enrolling.”

The FDA defines a “rare pediatric disease” as a disease that primarily affects individuals aged from birth to 18 years and affects fewer than 200,000 individuals in the US. Under the FDA’s Rare Paediatric Disease Priority Review Voucher programme, upon the approval of a qualifying new drug application (NDA) or biologics license application (BLA) for the treatment of a rare paediatric disease, the sponsor of such an application would be eligible for a Rare Paediatric Disease Priority Review Voucher that can be used to obtain priority review for a subsequent NDA or BLA. The Priority Review Voucher may be sold or transferred an unlimited number of times.

CAT-1004 is an oral small molecule that inhibits activated NF-kB, a protein that coordinates cellular response to muscular damage, stress and inflammation and plays an important role in muscle health. In skeletal muscle, activated NF-kB drives muscle degeneration and suppresses muscle regeneration. In animal models of DMD, CAT-1004 inhibited activated NF-kB, reduced muscle inflammation and degeneration and increased muscle regeneration. In phase 1 clinical trials, CAT-1004 inhibited activated NF-kB and was well-tolerated with no observed safety concerns. The FDA has granted CAT-1004 orphan drug and fast track designations for the treatment of DMD. Catabasis is currently conducting the MoveDMD phase 1/2 trial of CAT-1004 in 4-7 year-old boys with DMD.