FDA, Therapeutics

US FDA declines to approve Applied Therapeutics' genetic disease drug
Applied Therapeutics said on Wednesday that the U.S. FDA declined to approve its drug to treat galactosemia, a rare genetic metabolic disease, casting a cloud over the company's hopes for its first commercial product.
Applied Thera tanks, as FDA slaps CRL on govorestat NDA
The US Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for the New Drug Application (NDA) for govorestat, a novel, central nervous system (CNS)-penetrant aldose reductase inhibitor (ARI),
Applied Therapeutics: FDA Denial Of Govorestat NDA As Problematic As It Was Unexpected
Applied Therapeutics faces challenges after FDA rejection, but future opportunities exist. Learn why APLT stock could recover with other potential approvals.
Wait for galactosemia therapy goes on after FDA shuns Applied Therapeutics’ govorestat
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 disease.
Applied Therapeutics Receives Complete Response Letter from U.S. FDA Regarding New Drug Application for Govorestat for Classic Galactosemia
Govorestat is a central nervous system (CNS) penetrant Aldose Reductase Inhibitor (ARI) being developed for the treatment of multiple rare diseases including Classic Galactosemia, Sorbitol Dehydrogenase (SORD) Deficiency, and PMM2- congenital disorder of glycosylation (CDG).
Applied Therapeutics Down 76% After Hours on FDA Complete Response Letter
An FDA complete response letter indicates that an application can't be approved in its current form. Govorestat, the company's lead drug candidate, is also being developed for treatment of sorbitol dehydrogenase deficiency. Write to Josh Beckerman at [email protected]
pharmaphorum4h
Applied Tx craters as FDA rejects lead product candidate
The sell-off started last night and continued in after-hours trading after the New York-based biopharma company revealed that ...
Applied Therapeutics down 84% after receiving CRL from FDA on NDA for govorestat
In after-hours trading, shares are down 84% to $1.34.Don't Miss our Black Friday Offers:Unlock your investing potential with TipRanks Premium - ...
BioSpace3d
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 ...
Here are 4 biotech stocks that could get a boost from a more open FDA regulatory climate
Own stocks of companies that get drugs approved. That hasn’t been so easy. But the U.S. Food & Drug Administration (FDA) has ...
ENDPOINTS NEWS17h
Ap­plied Ther­a­peu­tic­s' rare dis­ease drug gets a CRL
The FDA rejected Applied Therapeutics’ experimental treatment, govorestat, for a rare disease called classic galactosemia, ...
FDA approves Kebilidi for aromatic L-amino acid decarboxylase deficiency
The U.S. Food and Drug Administration has approved PTC Therapeutics' Kebilidi (eladocagene exuparvovec-tneq) for the ...
Applied Therapeutics Receives FDA Complete Response Letter for Govorestat New Drug Application
Applied Therapeutics said it has received a complete response letter from the Food and Drug Administration related to its new drug application for govorestat due to deficiencies in the clinical ...
Soleno Therapeutics slides after FDA review extension of its Prader-Willi syndrome treatment
Soleno Therapeutics ( NASDAQ: SLNO) fell about 17% in premarket trading after the U.S. FDA extended the review period for its ...