Clinical Galactosemia treatment Gavorestat receives Fast Track and Orphan designation...

Galactosemia treatment Gavorestat receives Fast Track and Orphan designation from European Medicines Agency


Applied Therapeutic’s Gavorestat (AT-007) was able to successfully receive both Fast Track designation and orphan medicinal product designation from the European Medicines Agency (EMA). Gavorestat is a treatment for Galactosemia. 

Applied Therapeutics regarded this orphan designation for the medicine by EMA as an important step into the European market.

CEO of Applied Therapeutics expressed that she was happy to see EMA recognize the need for a Galactosemia treatment as the disease wreaks havoc on its patients. Further adding that “Orphan designation for AT-007  marks an important step towards advancing our regulatory initiatives in Europe.  We plan to meet  with the EMA  in the third  quarter to discuss  a potential  MAA submission in Europe  for conditional  approval based  on available  biomarker data or for full approval based on expected clinical outcomes data.”

Through this Orphan medicinal product status by the European Medicines Agency, the drug will be able to gain 10 years of market exclusivity in the EU, protocol assistance, and fee reductions.

Galactosemia is a disease that affects patients’ ability to metabolize galactose- a simple sugar found in food and in the body. The disease is both genetic and rare. Due to galactose not being metabolized effectively, a toxic substance is produced in the body known as a metabolite. This leads to significant complications such as deficiencies in cognition, speech, motor skills, and behavior along with neurological and reproductive damage (in women). Every year in the European Union (EU) over 100 babies are born with the disease while 4,000 patients exist in the region. 

Gavorestat (AT-007) works on the central nervous system and has been developed for the treatment of not only Galactosemia, but also Phosphomannomutase 2 Deficiency, a congenital disorder of Glycosylation and SORD Deficiency. 

The mode of action that Galactosemia follows is to block the Aldose Reductase enzyme, slowing down the development of the disease. The medicine is to be consumed orally and daily.

Galactosemia was shown to have a significant impact on reducing galactitol in both adults and children affected by Galactosemia when evaluated in the Phase I and II of a Registrational Study and compared to a placebo.

The study found that the medicine was safe to take and produced no significant adverse effects while also being effective in reducing galactitol levels. The drug is undergoing a Phase III clinical trial in children over 17 and a long term open-label study involving adults. 

In addition to the EMA, the US Food and Drug Administration (FDA) has also granted AT-007 both Fast Track designation and Orphan Drug designation. 

Applied Therapeutics is a biopharmaceutical company that develops novel drugs for the treatment of diseases that have high and unmet medical demand through clinical evaluations. Apart from its star candidate AT-007, for the treatment of Galactosemia, the company is also developing AT-001 which is a treatment for Diabetic Cardiomyopathy. In addition, AT-003 is in the preclinical stages within the company which is a treatment for Diabetic retinopathy.

Latest news

Life Sciences Voice Top Five Newsletter

Welcome to the latest edition of the Life Sciences Voice Top Five Newsletter, your source for the top developments...

Pfizer’s Phase 3 Trial for Gene Therapy in Boys with Duchenne Muscular Dystrophy Fails to Show Functional Improvement

Pfizer's gene therapy for Duchenne muscular dystrophy failed to improve motor function in young boys in a key late-stage...

Phase 3 Trial Results for Loqtorzi Show Positive Results in Advanced Hepatocellular Carcinoma Patients

Phase 3 trial results for Loqtorzi (toripalimab) and Avastin (bevacizumab) show that in first-line patients suffering from advanced hepatocellular...

Must read

Surrounded by controversy, FDA approves Biogen’s Alzheimer’s drug Aduhelm

In the middle of the debate about the Alzheimer’s drug approval, the United States FDA has authorized Aduhelm

You might also likeRELATED
Recommended to you