Applied Tx craters as FDA rejects lead product candidate
Shares in Applied Therapeutics have lost more than 80% of their value after the FDA turned down its marketing application for lead drug govorestat as a treatment for galactosaemia.
The sell-off started last night and continued in after-hours trading after the New York-based biopharma company revealed that the FDA had rejected its filing for govorestat (formerly AT007) due to "deficiencies in the clinical application."
In a statement, Applied Tx said it is reviewing the complete response letter (CRL) from the FDA and "plans to immediately request a meeting to discuss requirements for a potential resubmission […] or appeal of the decision, along with appropriate next steps."
The FDA had already delayed its decision on the drug in order to consider additional data from the company to support the application. Meanwhile, it remains under review in Europe, with a decision due in the first quarter of next year.
The outcome of the review has come as a major disappointment for patients and their families, with the Galactosemia Foundation calling the outcome "devastating" and urging the FDA to "reconsider on behalf of patients, families, and future generations."
Galactosaemia is an inherited metabolic disorder caused by a defect in the ability to process galactose, a sugar found in milk, causing harmful metabolites to build up in the body. It can lead to vomiting and diarrhoea, jaundice, liver problems, and neurological problems.
At the moment, the rare disease can only be managed by restricting galactose from the diet, which can be challenging as many food products contain milk-based ingredients. Moreover, some galactose is also produced by the body itself.
There are no approved drugs to treat the disorder, which affects approximately 3,000 patients with galactosaemia in the US and 80 new births each year, according to Applied Therapeutics. Govorestat is an aldose reductase inhibitor (ARI) designed to replace the enzyme that is deficient in people with the disorder, restoring their ability to metabolise galactose.
"We are disappointed by the FDA’s decision," commented Shoshana Shendelman, the founder and chief executive of Applied Therapeutics.
"Our strong commitment to the galactosaemia community is rooted in our belief that govorestat has the potential to change the lives of patients […] which we believe is evidenced by the breadth of efficacy and safety data demonstrating its ability to stop the decline on progressive clinical outcomes, including cognition and behaviour," she added.
Govorestat is also being developed for the treatment of sorbitol dehydrogenase (SORD) deficiency, a rare and progressive neuromuscular disease, and the company has said it hopes to file for approval in that indication in the first quarter of 2025.
