FDA Action Alert: Applied Therapeutics Awaits Verdict on Rare Disease Therapy

 

With two decisions originally scheduled for this week already announced, including BridgeBio’s approval in ATTR-CM, the regulator has just one PDUFA on its plate this holiday week.

Leading up to the U.S. Thanksgiving holiday, the FDA got two approvals originally scheduled for this week out of the way early. On Nov. 21, the regulator greenlit Jazz and Zymeworks’ Ziihera for HER2-positive biliary tract cancer, followed Friday evening by BridgeBio’s Attruby for transthyretin amyloid cardiomyopathy.

That leaves us with just one decision to watch this week, for Applied Therapeutics’ govorestat for the rare metaboilic disorder galactosemia.

Read below for more.

Applied Therapeutics Seeks First-Ever Approval in Galactosemia

Applied Therapeutics is developing its investigational aldose reductase inhibitor govorestat for the treatment of galactosemia. The deadline for the FDA’s verdict is Nov. 28.

With around 3,000 diagnosed cases in the U.S., galactosemia is a rare, genetic and metabolic disorder characterized by the impaired or complete lack of ability to break down galactose, which in turn leads to production of galactitol, a toxic metabolite that damages the nervous system. As a result, patients with galactosemia suffer from cognitive, behavioral, motor and speech problems. There are no approved therapies for galactosemia.

Govorestat is a brain-penetrant drug that addresses galactosemia by disabling the aldose reductase enzyme, which is responsible for the disease-causing conversion of galactose into galactitol. Applied Therapeutics is backing govorestat’s regulatory bid with data from the Phase I/II ACTION-Galactosemia and Phase III ACTION-Galactosemia Kids studies, as well as findings from preclinical trials.

ACTION-Galactosemia showed a rapid and clear reduction in galactitol levels in adult patients, with its effects starting from day one and persisting through one month. ACTION-Galactosemia Kids, meanwhile, pointed to govorestat’s clinical benefits in pediatric patients, resulting in improvements in activities of daily living, cognition, tremors and behaviors.

Govorestat’s initial target action date was Aug 28, with an advisory committee meeting planned. In March 2024, however, the FDA announced it needed more time to finish its review following supplemental analyses and data from the biotech. In September, the regulator said that it would no longer convene an advisory committee for govorestat.

https://www.biospace.com/fda/fda-action-alert-applied-therapeutics-awaits-verdict-on-rare-disease-therapy