Disc Medicine, Inc. · Complete Response Letter (CRL, NDA 220707)

FDA issued a complete response letter for NDA 220707 dated 2026-02-13.

Kernfakten

Eingereicht: 13. Feb. 2026, 00:00
Ereignis: Complete Response Letter
Richtung: Negativ
Unternehmen: IRONDisc Medicine
Asset: bitopertin
Quelle: openFDA
Belegstatus: Belegt

Auszug aus der Quelle

PN U.S. FOOD & DRUG ADMINISTRATION NDA 220707 COMPLETE RESPONSE Disc Medicine, Inc. Attention: Steve Caffé, MD Chief Regulatory Officer 321 Arsenal Street, Suite 101 Watertown, MA 02472 Dear Dr. Caffé: Please refer to your new drug application (NDA) or bitopertin oral tablets. We have completed our review of this application, as amended, and have determined that we cannot approve this application in its present form. We have described our reasons for this action below and, where possible, our recommendations to address these issues. CLINICAL/BIOSTATISTICS ou submitted NDA 220707 for bitopertin for , Proposing the primary biomarker endpoint of percen’ ole blood metal-free protoporphyrin IX (PPIX) as a surrogate endpoint that is reasonably likely to predict clinical benefit. In support of approval, you submitted the results of Study DISC-1459-201 (Trial 201) and Study DISC-1459-202 (Trial 202). Trial 201 was a randomized, double-blind, placebo-controlled trial in subjects 18 years of age and older with EPP. The trial included a 28-day screening period and a randomized 120-day treatment period. The trial enrolled | subjects, including |@ randomized to bitopertin 20 mg, @ randomized to bitopertin 60 mg, and | randomized to placebo. The trial was conducted at 9 sites in the United States. Trial was a randomized, open-label trial of bitopertin 60 mg vs. 20 mg in subjects 12 years of age and older with EPP or XLP. The trial included a 28- day screening period and a rand

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