Product information and disease description Product description

Phase of product development

10 evidence topics · 7 sources

Regulatory status

“QTORIN™ rapamycin is for investigational use only and has not been approved or cleared by the FDA” (opens the source at this quote in a new tab)

Development status

“The safety or efficacy has not been established for any use.” (opens the source at this quote in a new tab)

Regulatory submission: NDA submitted August 2026

“August 31, 2026” (opens the source at this quote in a new tab)
“the Company has completed the rolling submission of its New Drug Application (NDA) to the FDA” (opens the source at this quote in a new tab)

FDA submission: regulatory pathway

“505(b)(2)” (opens the source at this quote in a new tab)

Anticipated approval and commercial launch

“our first potential FDA approval in the first half of 2027” (opens the source at this quote in a new tab)

“commercial launch of QTORIN™ rapamycin for microcystic lymphatic malformations in the first half of 2027, if approved” (opens the source at this quote in a new tab)

Launch: Anticipated in the first half of 2027

“a patient services organization established in preparation for a potential launch in the first half of 2027, if approved” (opens the source at this quote in a new tab)

Special FDA designations: Breakthrough Therapy designation

“Breakthrough Therapy Designation to QTORIN rapamycin™ for the treatment of Microcystic Lymphatic Malformations (Microcystic LMs).” (opens the source at this quote in a new tab)

Special FDA designations: Fast Track designation

“the FDA previously granted Fast Track Designation” (opens the source at this quote in a new tab)

Special FDA designations: FDA orphan-drug database

Information	Source quotation
Date designated	“08/03/2021”
Orphan designation	“Treatment of Lymphatic Malformations”
FDA orphan approval status	“Not FDA Approved for Orphan Indication”

Special designations: European Medicines Agency

“Orphan Drug Designation has also been granted by EMA.” (opens the source at this quote in a new tab)