Healthcare Business

Why Stealth BioTherapeutics FDA Update Is a Big Deal

Stealth BioTherapeutics Corp. (NASDAQ: MITO) stock more than doubled on Tuesday after the company announced a critical update from the U.S. Food and Drug Administration (FDA). This is one of the few biotech stocks moving Tuesday that does not have direct ties to COVID-19.

Accordingly, the FDA granted a Rare Pediatric Disease (RPD) designation for elamipretide for the treatment of Barth syndrome, an ultra-rare genetic condition.

Under the FDA’s RPD designation program, the FDA may grant a priority review voucher to a sponsor who receives approval for a “rare pediatric disease,” a serious and life-threatening disease that primarily affects individuals aged from birth to 18 years and fewer than 200,000 people in the United States.

Upon FDA approval of elamipretide for Barth syndrome, Stealth Bio is eligible for a voucher that can be used to obtain priority review for a subsequent human drug application upon meeting relevant statutory requirements associated with the RPD program.

Barth syndrome is an ultra-rare genetic condition characterized by cardiac abnormalities often leading to heart failure and reduced life expectancy, recurrent infections, muscle weakness and delayed growth.

Reenie McCarthy, CEO of Stealth Bio, commented:

This designation for elamipretide underscores our alignment with the FDA around the significant and urgent unmet medical need for Barth patients. We hope to improve the healthspan of Barth patients, for whom cardiomyopathy, exercise intolerance and debilitating fatigue limit life expectancy and impair quality of life.  We are also expanding our development efforts to consider trials in patients suffering from overlapping metabolic cardiomyopathies in diseases such as Duchenne and Becker muscular dystrophies, and Friedreich’s ataxia.

Shares of Stealth Bio were last seen up about 145% at $4.65, in a 52-week range of $1.76 to $20.99. The consensus price target is $7.88.