When the FDA lifted a clinical suspension on Applied Therapeutics’ main program in galactosemia last February, New York Biotech signaled that it was on track to fast track approval by then, with the intention of filing an NDA in the third quarter of 2021.
Regulators, however, have apparently changed their minds.
Applied has decided to suspend the submission of an NDA for AT-007 as a treatment for galactosemia, the company revealed, following discussions with the FDA in which the agency said that “data on clinical results will likely be required for approval. “
The company says this came as a surprise, because it had thought – based on previous conversations – that it could seek expedited approval by showing that its drug reduces a key biomarker called galactitol.
The $ APLT stock fell 30.50% to $ 6.22.
Patients with galactosemia have trouble breaking down the galactose sugar in their bloodstream and may suffer from toxic waste products that build up in tissues and organs, causing long-term complications. Galactitol is one of these toxic metabolites.
Applied’s medicine is designed to inhibit the enzyme aldose reductase and cross the blood-brain barrier into the central nervous system.
Future plans and timing will depend on discussions with the FDA, according to Biotech.
“While disappointed with this shift in focus by the FDA, we remain committed to bringing this important treatment to patients with galactosemia,” Founder and CEO Shoshana Shendelman said in a statement.
The original NDA involved the use of AT-007 in adults. But Applied is also conducting a Phase III study with children, in which clinical outcomes over time are measured, including cognition, behavior and motor skills. After clinical suspension and subsequent protocol changes, the company said a firewall committee will complete its first efficacy review in the first quarter of this year (and, if it does not reach statistical significance, will repeat the process every six months until it does).