Applied Therapeutics receives USFDA approval for treatment of PMM2-CDG

▴ Applied Therapeutics receives USFDA approval for treatment of PMM2-CDG
US FDA grants AT-007 pediatric rare disease designation and orphan designation for treatment of PMM2-CDG

Applied Therapeutics, Inc., a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, announced today that the U.S. Food and Drug Administration (FDA) has granted AT-007 both Pediatric Rare Disease Designation and Orphan Drug Designation for treatment of PMM2-CDG. PMM2-CDG is a debilitating rare disease caused by a deficiency in the critical enzyme phosphomannomutase-2, required for systemic glycosylation of proteins. PMM2-CDG causes multiple organ failure and severe disability, resulting in approximately 20% mortality in the first four years of life. There are currently no drugs approved to treat PMM2-CDG.

AT-007, a novel CNS penetrant Aldose Reductase inhibitor (recently given the INN scientific name gavorestat) has demonstrated biological activity in a validated model of PMM2-CDG. Applied Therapeutics plans to initiate a clinical study in 2021 and is currently working with CDG experts and the FDA to design a robust clinical program.

“PMM2-CDG is a debilitating disease with no drugs available, which is central to Applied Therapeutics’ mission of creating life-changing treatments for patients who desperately need them,” said Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics. “Over the past year, we have been working closely with clinicians, researchers and families of PMM2-CDG patients, and are grateful for their collaboration and support. We are pleased that the FDA recognizes the transformative potential of AT-007 in treating patients with PMM2-CDG.”

Applied Therapeutics is a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need. The Company’s lead drug candidate, AT-007, is a novel central nervous system penetrant aldose reductase inhibitor (ARI) for the treatment of CNS rare metabolic diseases, including Galactosemia and PMM2-CDG. The Company initiated a Galactosemia clinical program in June 2019, read out positive data in adult Galactosemia patients in April 2020, and commenced a pediatric Galactosemia study in June 2020. Applied plans to initiate a clinical study of AT-007 in PMM2-CDG in 2021.

The Company is also developing AT-001, a novel potent ARI that is being developed for the treatment of Diabetic Cardiomyopathy, or DbCM, fatal fibrosis of the heart. The Company initiated a Phase 3 registrational study in DbCM in September 2019. The preclinical pipeline also includes AT-003, an ARI designed to cross through the back of the eye when dosed orally, for the treatment of diabetic retinopathy, as well as novel dual PI3k inhibitors in preclinical development for orphan oncology indications.

Tags : #AppliedTherapeutics #LatestUSFDAApproval #MultipleOrganFailure #RiccardoPerfetti

About the Author


Team Medicircle

Related Stories

Loading Please wait...

-Advertisements-




Trending Now

NRAI cautions restaurants about the dangers of aggregator payment gateways and deep discounting on diningDecember 09, 2024
Hot Cocoa: A Surprising Ally in Fighting Stress and Boosting Heart HealthDecember 09, 2024
What Your Headache Says About Your Health And Why It Can Be More Than Just a Temporary PainDecember 09, 2024
Hot Cocoa: A Surprising Ally in Fighting Stress and Boosting Heart HealthDecember 09, 2024
Is Your Favorite Snack a Secret Weapon Against Diabetes?December 07, 2024
Is the Fountain of Youth Hidden in Your Cells? Discover the Future of Ageing With Indian AIDecember 07, 2024
Does Your Heart Have a Mind of Its Own? Science Says Yes!December 07, 2024
Does Your Heart Have a Mind of Its Own? Science Says Yes!December 07, 2024
Heart Risks Are Shrinking Men’s Brains: What Science Reveals About Gender and AgingDecember 06, 2024
P&G India Celebrates 20 Years of P&G Shiksha with its largest ever Employee Volunteer Day Initiative across all offices and sitesDecember 06, 2024
Air India SATS Scales up Green Initiative with AeroWash Now Serving Two of India's Busiest AirportsDecember 06, 2024
The Silent Culprit Behind Osteoarthritis: Are Ultra-Processed Foods Eating Away our BonesDecember 06, 2024
From Daily Pills to 2 Shots Per Year: The Future of HIV ProtectionDecember 06, 2024
BREAKTHROUGH CLINICAL STUDY, FIRST OF ITS KIND, PROVES TRAYA CUSTOMIZED REGIMEN DELIVERS 3X* BETTER RESULTS THAN THE MOST PRESCRIBED HAIR GROWTH SERUM December 06, 2024
The Airborne Mutation: Is Bird Flu Becoming a Human Threat? December 05, 2024
Aster CMI Hospital collaborate with Government of Arunachal Pradesh Provide Affordable Liver Transplantation Services.December 05, 2024
Women missing out on treatment for their number one killerDecember 05, 2024
Packaged Water Under Fire: What FSSAI’s ‘High-Risk’ Tag Means for YouDecember 05, 2024
Life or Death: A New Breakthrough in Detecting Sepsis Before It’s Too LateDecember 05, 2024
Marble Centre International Unveils 'A Shared Memory' at Designuru 4.5December 05, 2024