FDA Grants Rare Pediatric Disease Designation to Catabasis for DMD Drug

September 18, 2015

The FDA has granted Catabasis Pharmaceuticals a rare pediatric disease designation for an investigational drug used to treat Duchenne Muscular Dystrophy, paving the way for priority review, the company said.

CAT-1004, an oral inhibitor aimed at reducing muscle inflammation and degeneration and boosting muscle growth, already received fast track and orphan drug designations. It works by suppressing NF-kB, a key protein responsible for much of the muscular decay.

Rare pediatric diseases are considered those that target mostly young people up to the age of 18 and have less than 200,000 sufferers. The designation makes treatments eligible for a speedier approval process. Companies that win FDA approval are eligible for a voucher that can be redeemed for a priority review of an NDA for another drug or can be sold or transferred.

Such voucher sales can bring in millions of dollars, as was the case when Retrophin sold its voucher to Sanofi for $245 million in May.

Catabasis reported positive results in its Phase 1 trial when the drug was shown to inhibit the muscular protein as intended with no safety issues or adverse events.

It joins two other companies, Sarepta Therapeutics and BioMarin Pharmaceuticals, to receive pediatric disease designation for potential DMD therapies.

Catabasis, based in Massachusetts, is enrolling DMD patients in Phase 1/2 clinical trials for 4- to 7-year-old boys with DMD. —Victoria Pelham