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

Elevar Therapeutics Announces FDA Acceptance for Review of New Drug Application for Lirafugratinib as Second-line Cholangiocarcinoma Treatment

Elevar Therapeutics, Inc., a majority-owned subsidiary of HLB Co., Ltd. and a fully integrated biopharmaceutical company dedicated to elevating treatment experiences and outcomes for patients who have limited or inadequate therapeutic options, today announced that the U.S. Food and Drug Administration (FDA) has completed its filing review of the New Drug Application (NDA) for lirafugratinib, an investigational therapy, for the treatment of patients with cholangiocarcinoma (CCA) with FGFR2 fusions or rearrangements who have received prior therapy. The FDA determined that the NDA is sufficiently complete to permit a substantive review and granted Priority Review, with a Prescription Drug User Fee Act (PDUFA) target action date of September 27, 2026.

• With Priority Review designation, FDA set a Sept. 27 PDUFA date for approval decision
• Lirafugratinib achieved a 46.5% ORR, in CCA patients with FGFR2 fusion and rearrangement

Priority Review designations are given to applications for medicines that, if approved, would lead to “significant improvements in the safety or effectiveness of the treatment” of a serious condition, according to the FDA. The Priority Review designation of the lirafugratinib NDA was supported by positive clinical data from the Phase 1/2 ReFocus trial (NCT04526106), which demonstrated a confirmed objective response rate (ORR) of 46.5% in the patients with the proposed indication. Its safety profile in the clinical data has been shown to be predictable and manageable through dose adjustments.

“Lirafugratinib has established a compelling clinical profile that differentiates it from existing treatment options,” said Dong-Gun Kim, chief executive officer of Elevar. “We are very pleased with the FDA’s priority review designation and focused on advancing the review process efficiently to bring this therapy to patients as quickly as possible.”

CCA, also known as bile duct cancer, is rare, with about 8,000 people in the U.S. diagnosed each year, according to the American Cancer Society.

Elevar Therapeutics continues to evaluate lirafugratinib in ongoing clinical development programs, including studies in other FGFR2-altered solid tumors. Any future indications will be subject to regulatory review and approval.

For more information about lirafugratinib, visit ElevarTX.com.

About Lirafugratinib

Lirafugratinib (RLY-4008) is a potent, selective, and oral small molecule inhibitor of FGFR2, a receptor tyrosine kinase that is frequently altered in certain cancers. FGFR2 is one of four members of the FGFR family, a set of closely related proteins with highly similar protein sequences and properties. Preclinically, lirafugratinib demonstrated FGFR2-dependent killing in cancer cell lines and induced regression in in vivo models with minimal inhibition of other targets, including other members of the FGFR family. In addition, lirafugratinib demonstrated strong activity against known clinical on-target resistance mutations in vitro and in vivo preclinical models. Lirafugratinib is currently being evaluated in a clinical trial to enroll additional patients with previously treated, advanced or metastatic solid tumors other than CCA harboring FGFR2 fusion or rearrangement, who have not been treated with prior FGFR inhibitors.

Elevar Therapeutics Announces FDA Acceptance for Review of New Drug Application for Lirafugratinib as Second-line Cholangiocarcinoma Treatment

Deciphera Pharmaceuticals Announces U.S. Food and Drug Administration Acceptance for Filing of New Drug Application for Tirabrutinib in Patients with Relapsed or Refractory PCNSL

Deciphera Pharmaceuticals, a member of Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; “Ono”), today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) under the accelerated approval pathway for tirabrutinib, a highly selective irreversible, second generation Bruton tyrosine kinase inhibitor, for the treatment of relapsed or refractory primary central nervous system lymphoma (R/R PCNSL). The FDA has set an action date of December 18, 2026, under the Prescription Drug User Fee Act (PDUFA).

Regulatory submission is based on positive results from the Phase 2 PROSPECT Study

FDA sets PDUFA Date of December 18, 2026

“R/R PCNSL is a rare and aggressive form of non-Hodgkin lymphoma with particularly poor clinical outcomes. Patients often experience difficulty and delay in diagnosis, and once they are diagnosed, there is a high unmet need for a treatment with a favorable safety profile,” said Matthew L. Sherman, M.D., Chief Medical Officer of Deciphera. “The FDA’s acceptance of tirabrutinib’s NDA for filing is an exciting milestone as it brings us one step closer to our goal of providing patients with R/R PCNSL an important new treatment option.”

“We are very pleased that the NDA for tirabrutinib has been accepted for filing,” said Toichi Takino, President and COO of Ono Pharmaceutical Co., Ltd. “This is an important milestone on the way to expanding our commercial pipeline and achieving our goal of becoming a global specialty pharma. Tirabrutinib’s potential to address unmet patient needs embodies our corporate philosophy and we will continue to focus on developing and delivering innovative medicines to benefit patients worldwide.”

The NDA is supported by the positive results from the Phase 2 PROSPECT study, presented at the 2025 American Society for Clinical Oncology (ASCO) Annual Meeting, in which tirabrutinib demonstrated an overall response rate of 67%, a complete response rate of 44%, and a manageable safety profile. If approved, tirabrutinib will be the first BTK inhibitor therapy commercially available in the U.S. for the treatment of patients with R/R PCNSL, and the third commercial therapy for Ono group available in the U.S. The study is currently recruiting patients with R/R PCNSL in a global Phase 3 randomized trial, which will serve as a confirmatory study for this indication (ClinicalTrials.gov NCT07104032.)

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

Epizyme Announces FDA Filing Acceptance of New Drug Application and Priority Review for Tazemetostat for the Treatment of Epithelioid Sarcoma

Epizyme, Inc. (Nasdaq: EPZM), a late-stage biopharmaceutical company developing novel epigenetic therapies, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the company’s New Drug Application (NDA) for accelerated approval of tazemetostat, its lead investigational agent. Epizyme has proposed an indication of metastatic or locally advanced epithelioid sarcoma not eligible for curative surgery. The FDA granted Priority Review for the NDA and has set a Prescription Drug User Fee Act (PDUFA) target action date of January 23, 2020. Priority Review is granted to investigational therapies that, if approved, may offer significant improvements in the treatment, prevention or diagnosis of a serious condition.

“We are thrilled with FDA’s acceptance of this first tazemetostat NDA submission for priority review, and to be an important step closer to achieving our mission of rewriting treatment for patients with cancer and other serious diseases,” said Robert Bazemore, president and chief executive officer of Epizyme. “This is a significant achievement in the development of this potentially first-in-class EZH2 inhibitor, and we look forward to working with FDA during the review. If approved, we believe tazemetostat could become an important new option in the treating physicians’ arsenal. We would like to extend our sincerest gratitude to those patients, families and medical teams who have participated in our clinical studies and helped bring tazemetostat to this stage.”

Epizyme’s NDA submission is based primarily on data from the 62 patient epithelioid sarcoma cohort of its ongoing Phase 2 study of tazemetostat. These data, recently reported at the 2019 American Society of Clinical Oncology (ASCO) Annual Meeting, showed that tazemetostat treatment resulted in clinically meaningful and durable responses, and was generally well-tolerated.

To support full approval of tazemetostat for epithelioid sarcoma, Epizyme will initiate a global confirmatory trial. The company plans to conduct a 1:1 randomized, controlled clinical trial in the front-line treatment setting comparing tazemetostat in combination with doxorubicin versus placebo plus doxorubicin in approximately 150 patients. The primary efficacy endpoint will be progression-free survival, and secondary efficacy endpoints will include overall survival, disease control rate, overall response rate and duration of response. The confirmatory study will include a safety run-in that is expected to begin in the second half of 2019.

About the Tazemetostat Clinical Trial Program 

Tazemetostat, an oral, potent, first-in-class EZH2 inhibitor, is currently being studied as a monotherapy in ongoing clinical programs in patients with certain molecularly defined solid tumors, including epithelioid sarcoma and other INI1-negative tumors, and in patients with follicular lymphoma, both with and without EZH2 activating mutations. Multiple clinical studies are underway through collaborations assessing tazemetostat as a combination treatment for patients with diffuse large B-cell lymphoma. Epizyme also plans to conduct multiple additional clinical trials designed to evaluate the potential benefit of tazemetostat in earlier lines of therapy for follicular lymphoma, as well as new combinations and cancer indications.

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

Allergan Announces FDA Acceptance of New Drug Application for Ubrogepant for the Acute Treatment of Migraine

   Allergan plc (NYSE: AGN)  announced that the U.S. Food and Drug Administration (FDA) has accepted the company's New Drug Application (NDA) for ubrogepant for the acute treatment of migraine in adults. The NDA filing is based on the successful completion of four clinical trials – two pivotal studies, ACHIEVE I and ACHIEVE II, which demonstrated the efficacy, safety and tolerability of ubrogepant, as well as two additional safety studies. A 10-month review period has been assigned with the Prescription Drug User Fee Act (PDUFA) in the fourth quarter of 2019.

"Following the acceptance and expected review of this NDA, we anticipate ubrogepant to be the first approved oral CGRP receptor antagonist for the acute treatment of migraine, and may be used in conjunction with other available migraine treatments," said David Nicholson, Chief Research and Development Officer, Allergan. "As a leader in Chronic Migraine research for more than 20 years, Allergan looks to provide options for patients who need new acute and preventative treatments for migraine. In addition to ubrogepant, we are continuing to advance the Phase 3 clinical program for atogepant, the company's second orally-administered investigational CGRP receptor antagonist specifically for migraine prevention."

In the four clinical studies (ACHIEVE I, ACHIEVE II, UBR-MD-04 and 3110-105-002) supporting the NDA, ubrogepant demonstrated efficacy, safety and tolerability in the acute treatment of migraine among a broad patient population, including those who had an insufficient response to a triptan or those patients in whom triptans were contraindicated, as well as in patients who had moderate to severe CV risk profile. Following are topline results from the key clinical studies:

Phase 3 Clinical Trials (ACHIEVE I & ACHIEVE II)

The two pivotal Phase 3 clinical trials demonstrated the efficacy, safety and tolerability of orally administered ubrogepant (ACHIEVE I at 50 mg and 100 mg; and ACHIEVE II at 25 mg and 50 mg) compared to placebo to treat a single migraine attack in adults.

• The 50 mg and 100 mg doses met the studies' co-primary endpoints, demonstrating a statistically significant greater percentage of ubrogepant patients achieving pain freedom and absence of most bothersome symptom at two hours after the initial dose as compared to placebo patients; there was continued separation from placebo up to 48 hours.
• The 50 mg and 100 mg doses also met key secondary endpoints, demonstrating a statistically significant greater percentage of ubrogepant patients achieving pain relief at two hours after the initial dose compared to placebo patients.

Additional Safety Studies (UBR-MD-04 & 3110-105-002)

Study UBR-MD-04: Phase 3, multicenter, randomized, 52-week open-label extension trial in which adults with migraine were randomized 1:1:1 to usual care, ubrogepant 50 mg, or ubrogepant 100 mg to assess long-term safety and tolerability.

• Intermittent use of ubrogepant 50 mg or 100 mg for the acute treatment of migraine attacks over one year was well-tolerated. The three most frequently reported adverse events were nasopharyngitis, upper respiratory tract infection and sinusitis.

Study 3110-105-002: Phase 1, multicenter, double-blind, parallel-group trial evaluated the safety and tolerability of ubrogepant 100 mg, focusing on hepatic safety in healthy participants administered high frequency intermittent  dosing (2 days of ubrogepant 100 mg followed by 2 days of placebo for 8 consecutive weeks).

• Ubrogepant 100 mg was well-tolerated following frequent dosing and was not associated with persistent increases in ALT/AST compared to placebo, supporting liver safety.

"Despite its prevalence and burden, migraine remains an undertreated disease, with many patients continuing to seek additional treatment options from their physicians," said Dr. Jessica Ailani, a neurologist and Director of the Medstar Georgetown Headache Center. "If approved, ubrogepant, the first innovation in the acute treatment of migraine in over 25 years, will be used across the entire spectrum of the disease (from episodic to chronic) helping patients achieve relief in the moments when they most demand it."

About Ubrogepant

Ubrogepant is a novel, highly potent, orally-administered CGRP receptor antagonist in development for the acute treatment of migraine. CGRP and its receptors are expressed in regions of the nervous system associated with migraine pathophysiology. CGRP receptor antagonism is a novel mechanism of action for the acute treatment of migraine that clearly differs from the mechanisms of currently available triptans (serotonin 1B/1D agonists), opioids and ergots.

https://pubchem.ncbi.nlm.nih.gov/compound/Ubrogepant

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

Allergan Announces FDA Acceptance of New Drug Application for Ubrogepant for the Acute Treatment of Migraine

Harmony Biosciences Announces File Acceptance Of Its New Drug Application For Pitolisant

   Harmony Biosciences, LLC (Harmony), announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) for its investigational product, pitolisant, and has granted Priority Review for this NDA. Pitolisant is a first-in-class molecule with a novel mechanism of action; it is a potent and highly selective histamine 3 (H₃) receptor antagonist/inverse agonist for the potential treatment of excessive daytime sleepiness (EDS) and/or cataplexy in adult patients with narcolepsy. A Priority Review designation by the FDA indicates that, if approved, pitolisant would provide a significant improvement in the safety or effectiveness of the treatment of EDS and/or cataplexy in adult patients with narcolepsy when compared to existing treatments. Harmony’s goal is to obtain FDA approval to market pitolisant in the U.S. in 2019.

“The impact of narcolepsy can be significant and severely disruptive to everyday life for up to 200,000 Americans living with this disorder,” said John C. Jacobs, President and CEO at Harmony. “This is an important step for patients and for our company, whose mission is to develop novel treatment options for people living with rare and orphan diseases.”

“Pitolisant offers a novel approach to the treatment of both EDS and cataplexy in patients with narcolepsy, for which there have been no new treatment options in over 15 years,” said Jeffrey M. Dayno, M.D., Chief Medical Officer at Harmony. “We look forward to working with the FDA during its review of the pitolisant NDA, with our hope of being able to offer this new treatment option to help address an important unmet medical need for people living with narcolepsy.”

The NDA submission is based on results from the clinical development program in narcolepsy, which included over 300 patients, some of whom were treated for up to five years. It also included safety data in over 1500 patients across multiple patient populations.

About Pitolisant

Pitolisant is an investigational medication in the U.S. that is not approved by the FDA. It was granted orphan designation for the treatment of narcolepsy, Fast Track designation for the treatment of excessive daytime sleepiness (EDS) and cataplexy in patients with narcolepsy, and Breakthrough Therapy designation for the treatment of cataplexy in patients with narcolepsy. Pitolisant, a first-in-class medication, is a potent and highly selective histamine 3 (H₃) receptor antagonist/inverse agonist; it enhances the activity of histaminergic neurons in the brain that function to improve a patient’s wakefulness and inhibit attacks of cataplexy. It was designed and developed by Bioprojet, who has marketed the product in Europe since its approval by the European Medicines Agency in 2016. Harmony’s goal is to obtain FDA approval to market this new medication in the U.S. in 2019. If approved, pitolisant would represent the first new therapy in the U.S. in over 15 years for the treatment of both EDS and cataplexy in adult patients with narcolepsy.