Current paediatric standard-of-care for galactosemia is a galactose-free diet. Image credit: Shutterstock / Photoongraphy. Nearly three months after an experimental treatment for classic galactosemia ...

Current paediatric standard-of-care for galactosemia is a lactose-free diet. Image credit: Shutterstock / Photoongraphy. Nearly three months after an experimental treatment for classic galactosemia ...

The phase 3 ACTION-Galactosemia Kids trial evaluated govorestat vs placebo in children (n=47) aged 2 to 17 years with classic galactosemia. The primary endpoint was the global statistical test ...

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 ...

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 around 3,300 patients in the US with galactosemia, an inherited metabolic disorder that affects the body’s ability to properly convert galactose to glucose. A deficiency in the enzyme ...

Applied Therapeutics received a Complete Response Letter from the FDA for govorestat's NDA for Classic Galactosemia. Quiver AI Summary. Applied Therapeutics, Inc. announced that the FDA has issued ...

Nicole Casale, president of the Galactosemia Foundation, stated:. The Galactosemia community is devastated by the FDA's decision to not approve the first and currently only potential treatment for ...

Galactosemia affects approximately 3,300 patients in the U.S. and 4,400 in the E.U., with mandatory newborn screening in these regions. Currently, there are no treatment options for this ...