Nearly three months after an experimental treatment for classic galactosemia was rejected by the US Food and Drug Administration (FDA), there has been little progress in developing therapies to ...

The US Food and Drug Administration (FDA) has declined to approve Applied Therapeutics’ govorestat for classic galactosemia, prolonging the wait for the first marketed treatment for the genetic ...

There are approximately 3,300 patients with Galactosemia in the U.S. and 80-100 new births per year, and approximately 4,400 patients with Galactosemia in the E.U. and approximately 120 new births ...

Galactosemia is a rare genetic condition that prevents babies from processing galactose, an important sugar in breast milk. Though the disease can cause many issues, it’s easily diagnosed and ...

Galactosemia is an inherited genetic disorder of metabolism where the body is unable to metabolize a sugar called galactose. Galactose forms half of the sugar lactose, which is found in milk. The ...

Summary. Applied Therapeutics is a clinical-stage biotech focused on treating symptoms of galactosemia in the central nervous system. Govorestat (AT-007) is in late-stage regulatory approval for ...

The NDA included data from the phase 1/2 ACTION-Galactosemia study (ClinicalTrial.gov Identifier: NCT04117711) and the phase 3 ACTION-Galactosemia Kids trial (ClinicalTrial.gov Identifier ...

Shares of Applied Therapeutics APLT surged 68.8% on Wednesday after it announced a regulatory update regarding the ongoing new drug application (NDA) review of APLT’s lead candidate, govorestat ...

The company estimates that ~75–80 new classic galactosemia patients in the U.S. and ~115–120 new classic galactosemia patients in the EU are born every year.

Nov 27 (Reuters) - Applied Therapeutics (APLT.O), opens new tab said on Wednesday that the U.S. FDA declined to approve its drug to treat galactosemia, a rare genetic metabolic disease, casting a ...

Galactosemia is a rare genetic disorder that causes an inability to digest galactose, which is a breakdown product of lactose. Lactose is one of the main carbohydrates in milk.