Egetis Announces FDA Acceptance and Priority Review of NDA for Emcitate (tiratricol) for MCT8 Deficiency

Egetis Therapeutics AB (publ) (“Egetis” or the “Company”) (NASDAQ Stockholm: EGTX), today announced that the U.S. Food and Drug Administration (FDA) has accepted the filing of its New Drug Application (NDA) for Emcitate® (tiratricol) for the treatment of MCT8 deficiency. Egetis completed the rolling NDA in January 2026. The application has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date, or FDA decision date, of September 28, 2026.

NDA submission for Emcitate® successfully validated by the FDA

Priority Review granted and PDUFA target action date set to September 28, 2026

Emcitate® (tiratricol) is eligible to receive a Priority Review Voucher (PRV) upon approval

If approved, Emcitate® (tiratricol) would become the first approved treatment in the United States for MCT8 deficiency

Nicklas Westerholm, CEO of Egetis, commented: “MCT8 deficiency is a rare, devastating, life-shortening disorder with no approved treatment in the U.S. The acceptance of our NDA with Priority Review represents an important step in the path to making Emcitate® (tiratricol) available to MCT8 deficiency patients in the U.S. The Priority Review also highlights the robust dataset we have in this rare genetic disease, with the NDA based on results from Triac Trial I, Triac Trial II, ReTRIACt, EMC Cohort Study, EMC Survival Study and the U.S. Expanded Access Program. We look forward to collaborating with the FDA during the NDA Priority Review process and will in parallel continue to build our medical affairs and market access capabilities towards a potential U.S. launch in the fourth quarter of this year.”

A Priority Review will direct overall attention and resources to the evaluation of applications for drugs that, if approved, would represent significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. A Priority Review means FDA’s goal is to take action on an application within 6 months, compared to 10 months under standard review.

Emcitate® (tiratricol) has previously been granted Rare Pediatric Disease Designation by the FDA and is eligible to receive a Priority Review Voucher (PRV) upon approval, and a PRV was consequently requested in connection with the submission. The transferability of PRVs has created an active secondary market for these vouchers, selling for $150-$205 million per voucher in 2025/26.

https://en.wikipedia.org/wiki/Emetine

Egetis Announces FDA Acceptance and Priority Review of NDA for Emcitate (tiratricol) for MCT8 Deficiency

Submission of HTX-019 (aprepitant) NDA for the Prevention of Postoperative Nausea and Vomiting to FDA

 

 

 

 

Heron Therapeutics, Inc.  announced the submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for HTX-019 (aprepitant) injectable emulsion for the prevention of postoperative nausea and vomiting (PONV) in adults. HTX-019 is a proprietary intravenous (IV) formulation of aprepitant, a substance P/neurokinin-1 (NK₁) receptor antagonist (RA) that is approved for PONV prevention.

The NDA filing includes data demonstrating the bioequivalence of HTX-019 32 mg as a 30-second IV injection to oral aprepitant 40 mg, supporting its efficacy for the prevention of PONV. Results also showed HTX-019 was well-tolerated with a similar safety profile compared to oral aprepitant. Because HTX-019 is administered as an IV injection, it provides convenient, rapid, consistent, and reliable exposure in all patients and overcomes the need to take the oral formulation 1 to 3 hours before anesthesia. The HTX-019 injectable emulsion formulation intended for PONV prevention is identical to the approved CINVANTI^® (aprepitant) injectable emulsion formulation for prevention of chemotherapy-induced nausea and vomiting (CINV).

Despite advances in postoperative care, nausea and vomiting has remained a challenge for many patients undergoing surgery. There are approximately 65 million diagnostic and surgical procedures at risk of resulting in PONV in the U.S. each year. More than half of these patients are at moderate to high risk of developing PONV. Recent data has also shown that PONV can lead to increases in medical costs and delays in discharge and recovery following procedures. Aprepitant is the first and only NK₁ RA to be approved for prevention of PONV based on showing superiority to ondansetron, the current standard of care.

"In a recent Cochrane Meta-Analysis, aprepitant was found to be the most effective agent for PONV prevention with activity similar to two-drug combinations. In fact, the use of oral aprepitant has grown by almost 80% in the past three years without any promotional efforts. Our IV formulation is designed to directly deliver the active form of the drug, aprepitant, to patients over 30 seconds so it can take effect much more quickly than when taken orally," said Barry Quart, Pharm.D., Chairman and Chief Executive Officer of Heron. "The HTX-019 NDA was filed with the same FDA division that previously approved CINVANTI without delays."

"There remains an important unmet need for effective and convenient products for PONV prevention," said Ashraf Habib, MBBCh, MSc, MHSc, FRCA, Chief, Division of Women's Anesthesia at Duke University Hospital. "If approved, this innovative IV formulation of aprepitant, with rapid achievement of therapeutic drug levels, will enable physicians to provide patients with a well-established agent that effectively prevents nausea and vomiting after surgery, using a route of administration that fits well in the perioperative workflow."

About HTX-019 for PONV

HTX-019 is an IV injectable emulsion formulation designed to directly deliver aprepitant, the active ingredient in EMEND^® (aprepitant) capsules, which is the only substance P/neurokinin-1 receptor antagonist to be approved in the U.S. for the prevention of postoperative nausea and vomiting (PONV) in adults. The FDA-approved dose of oral EMEND is 40 mg for PONV prevention, which is given within 3 hours prior to induction of anesthesia for surgery. In a Phase 1 clinical trial, 32 mg of HTX-019 as a 30-second IV injection was demonstrated to be bioequivalent to oral aprepitant 40 mg. The NDA for HTX-019 for PONV was submitted in November 2021.

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 https://en.wikipedia.org/wiki/Aprepitant