Applied Therapeutics is pumping the brakes on requesting FDA approval for its first drug after the agency requested clinical outcomes data on the treatment's impact in a rare metabolic disease.
Applied will pause a request for AT-007's green light in galactosemia after the FDA said clinical outcomes data would be needed before the approval was considered, the biotech said Monday.
Galactosemia is a rare disease that leads to impairments to speech, behavior, cognition and motor skills because of the body’s inability to break down galactose, a simple sugar found in many foods. The disease impacts about 3,000 people in the U.S., including 80 new babies each year.
The clinical outcomes data—or, data that can show how patients fare after treatment—could be months or quarters away. AT-007 is currently in a phase 3 pediatric study, and the first assessment of clinical outcomes will take place this quarter and every six months thereafter until statistical significance is reached. Applied is examining whether the treatment can have significant impacts on cognition, speech, behavior and motor skills.
RELATED: Bad to worse for Applied Therapeutics as FDA slaps partial clinical hold on rare drug
Applied shares closed at $8.91 apiece Friday and have since slumped to $5.94 as of 11:29 a.m. ET Tuesday.
This is the latest setback for the drug. The FDA hit the biotech with a partial clinical hold in August 2020 asking for more technical information to ensure patients had "access to the prospect of direct benefit of the drug." The biotech was hit by a short report from bloggers weeks prior to that insinuating the company was cutting corners and had integrity issues related to AT-007.
Applied had previously discussed using the accelerated approval pathway for AT-007 with the FDA. The new delays for the drug have "disappointed" Applied, said Shoshana Shendelman, Ph.D., CEO and founder, in a statement.
“We will continue to work with the FDA to determine the most expeditious path forward to regulatory approval and will provide an update on timing and plans accordingly," the CEO said.
The oral Aldose reductase inhibitor is also being tested in a phase 3 registrational trial in SORD deficiency and is phase 2 ready in PMM2-CDG. SORD deficiency is a progressive hereditary condition in which toxic levels of sorbitol are formed, and PMM2-CDG is a congenital disorder that leads to central nervous system complications.