FDA Accepts Shionogi’s Ensitrelvir NDA for Review as the First Oral Therapy for the Prevention of COVID-19 Following Exposure

Shionogi & Co., Ltd. (Head Office: Osaka, Japan; Chief Executive Officer: Isao Teshirogi, Ph.D.; hereafter “Shionogi”) announced the U.S. Food and Drug Administration (FDA) has accepted for review a New Drug Application (NDA) from Shionogi Inc., a New Jersey-based subsidiary of Shionogi, for ensitrelvir (Generic name: ensitrelvir fumaric acid, Code No.: S-217622), an investigational oral antiviral for the prevention of COVID-19 following exposure to an infected individual. The FDA has set an action date of June 16, 2026 under the Prescription Drug User Fee Act (PDUFA).

The NDA is supported by results from the global, double-blind, randomized, placebo-controlled Phase 3 study, SCORPIO-PEP, which studied ensitrelvir as post-exposure prophylaxis (PEP). If approved, ensitrelvir would be the first and only oral therapy for the prevention of COVID-19 following exposure to an infected individual.1,2

SARS-CoV-2 remains highly transmissible and up to half of people living with an infected individual may develop COVID-19.3,4 The virus is constantly evolving, with novel symptoms reported.5,6 COVID-19 continues to impact daily life and may lead to absences from school and work, may cause long COVID, and in some cases, may progress to severe disease.7-12 Even patients with mild COVID-19 may experience worsening of preexisting chronic conditions.13-17

Following exposure to COVID-19, the best way to avoid its potentially serious and long-term complications is to stop viral replication, which prevents the development of the disease.1,2 There are currently no approved antiviral therapies proven to prevent COVID-19 following exposure.1,2,18 COVID-19 vaccines are received before exposure, and they do not stop viral replication.19,20 Other COVID-19 antivirals are taken following exposure and diagnosis, after viral replication has occurred and disease is already established.21

“Shionogi has a long history of innovation in infectious disease treatment and prevention. Our dedication to this field has led to significant breakthroughs in the development of novel antimicrobials and antivirals for HIV/AIDS, influenza and COVID-19. If approved, ensitrelvir will be the first and only oral therapy to help protect people in the U.S. from COVID-19 following exposure," said Nathan McCutcheon, MBA, President and CEO, Shionogi Inc.

About ensitrelvir

Ensitrelvir is a SARS-CoV-2 main protease inhibitor created through joint research between Hokkaido University and Shionogi. SARS-CoV-2 has an enzyme called the main protease (3-CL), which is essential for the replication of the virus. Ensitrelvir suppresses the replication of SARS-CoV-2 by selectively inhibiting the main protease.

Ensitrelvir, known as Xocova® in countries where it is approved, received emergency regulatory approval in Japan in November 2022 and full approval in March 2024 for the treatment of COVID-19 based on results from SCORPIO-SR, a Phase 3 study conducted in Asia, during the Omicron-dominant phase of the pandemic.

In SCORPIO-SR, ensitrelvir showed both clinical symptomatic efficacy (symptom resolution sustained for at least 24 hours) for five typical Omicron-related symptoms (primary endpoint) and antiviral efficacy (key secondary endpoint) in a predominantly vaccinated population of patients with mild-to-moderate SARS-CoV-2 infection, regardless of risk factors. Most adverse events were mild in severity and no deaths were seen in the study. Among the most common treatment-related adverse events were temporary decreases in high-density lipoprotein and increased blood triglycerides, as observed in previous studies. Results from this study were published in JAMA Network Open.

In 2025, Shionogi submitted two new drug applications in Japan for post-exposure prophylaxis of COVID-19 and for COVID-19 treatment in pediatric patients aged six to under 12 years. The pediatric submission is based on a multicenter, randomized, double-blind, placebo-controlled trial of ensitrelvir in mild-to-moderate COVID-19 patients aged 6 to 12 years in Japan. The study confirmed safety and tolerability and found the pharmacokinetics of ensitrelvir in this age group to be similar to that in adults.

Ensitrelvir became available in Singapore for the treatment of COVID-19 via a Special Access Route application in 2023, and it is currently under regulatory review in Taiwan for the treatment of COVID-19. Ensitrelvir is also under regulatory review with the European Medicines Agency for COVID-19 post-exposure prophylaxis and treatment.22

Ensitrelvir is an investigational drug outside of Japan and Singapore. In addition, the brand name Xocova® has not been approved for use outside of Japan and Singapore and pertains only to the approved drug in Japan and Singapore.

FDA Accepts Shionogi’s Ensitrelvir NDA for Review as the First Oral Therapy for the Prevention of COVID-19 Following Exposure

Achieve Life Sciences Announces FDA Acceptance of Cytisinicline New Drug Application for Treatment of Nicotine Dependence for Smoking Cessation

Achieve Life Sciences, Inc. (Nasdaq: ACHV), a late-stage specialty pharmaceutical company focused on the global development and commercialization of cytisinicline for treatment of nicotine dependence, today announced that the U.S. Food and Drug Administration (FDA) has accepted the cytisinicline New Drug Application (NDA) for a new treatment for smoking cessation in adults. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) targeted action date of June 20, 2026.

“The FDA’s acceptance of our NDA validates the totality of our product development program,” said Dr. Cindy Jacobs, President and Chief Medical Officer of Achieve Life Sciences. “Our application is supported by a decade of rigorous research and comprehensive data from thousands of participants. We've built compelling scientific and clinical results as a foundation that, if approved, positions cytisinicline to potentially address a significant medical need. We’re eager to engage constructively with the FDA as we progress through the NDA review process.”

The company’s NDA details a comprehensive clinical development program, with more than 2,000 clinical trial participants contributing to the body of evidence. The ORCA-2 and ORCA-3 Phase 3 trials showed cytisinicline administered for either 6 or 12 weeks, alongside standard behavioral support, demonstrated significantly greater smoking abstinence rates by the end of treatment and in long-term abstinence through week 24 as compared to placebo. In addition, safety data will include over 400 participants who have received at least six months of cumulative cytisinicline exposure and over 200 participants receiving at least 1 year of cumulative cytisinicline exposure, with no new safety concerns as reported by the Data Safety Monitoring Committee.

“Smoking is the leading cause of preventable death and disease, claiming the lives of nearly half a million Americans each year, and costing the American economy more than an estimated $600 billion a year,” said Rick Stewart, Chief Executive Officer of Achieve Life Sciences. “The FDA's acceptance of our NDA filing underscores nicotine dependence as an important public health need that demands action. We're energized by this milestone and remain laser-focused on our mission, working toward commercial readiness for the second half of 2026, pending FDA approval. Each year, an estimated 15 million Americans attempt to quit smoking, and we're committed to providing those who are ready to quit a new tool so they can break free from nicotine dependence.”

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

Achieve Life Sciences Announces FDA Acceptance of Cytisinicline New Drug Application for Treatment of Nicotine Dependence for Smoking Cessation

Bridgebio Pharma Announces U.S. Food and Drug Administration (FDA) Acceptance of New Drug Application (NDA) for Acoramidis for the Treatment of Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the Company’s New Drug Application (NDA) for acoramidis, an investigational drug for the treatment of ATTR-CM. The application was based on positive results from ATTRibute-CM, the Company’s Phase 3 study designed to evaluate the efficacy and safety of acoramidis, an investigational, next-generation, orally-administered, highly potent, small molecule stabilizer of transthyretin (TTR). The FDA has set an action date of November 29, 2024 under the PDUFA. The FDA also notified the Company that it is not currently planning to hold an advisory committee meeting to discuss the application.

 

“The FDA’s acceptance of our NDA submission for review reinforces our belief in acoramidis and its potential to make an important contribution to the care of patients with ATTR-CM,” said Jonathan Fox, MD, PhD, President and Chief Medical Officer of BridgeBio Cardiorenal. “We look forward to the upcoming review process and the potential for approval in the United States. Similarly, with the European Marketing Authorization Application accepted and with plans to extend our submissions to other countries and regions, we are committed to making acoramidis available to patients.”

In July 2023, BridgeBio announced positive results from ATTRibute-CM, reporting a highly statistically significant result, demonstrated by a Win Ratio of 1.8 (p<0.0001) on the primary endpoint (a hierarchical analysis prioritizing in order: ACM, then frequency of CVH, then change from baseline in N-terminal prohormone of brain natriuretic peptide (NT-proBNP), then change from baseline in 6-minute walk distance (6MWD)). Acoramidis was well-tolerated, with no safety signals of potential clinical concern identified. BridgeBio has also presented analyses from ATTRibute-CM at the European Society of Cardiology Congress 2023 and at the American Heart Association Scientific Sessions 2023.

“As part of our mission, we seek to improve the lives of patients with amyloidosis by providing support to them and their caregivers throughout their journey. There is a need for more treatment options that can help fill the significant unmet need that exists for patients today. We are excited by BridgeBio’s recent NDA acceptance from the FDA, which we hope moves us one step closer to having acoramidis available as a treatment for the ATTR-CM community,” said Isabelle Lousada, president and CEO of the Amyloidosis Research Consortium, a global nonprofit organization dedicated to advancements in amyloidosis.

The Company also received acceptance of its Marketing Authorization Application with the European Medicines Agency and is preparing for additional global regulatory submissions.

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

Bridgebio Pharma Announces U.S. Food and Drug Administration (FDA) Acceptance of New Drug Application (NDA) for Acoramidis for the Treatment of Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Lykos Therapeutics Announces FDA Acceptance and Priority Review of New Drug Application for MDMA-Assisted Therapy for PTSD

Lykos Therapeutics (formerly MAPS Public Benefit Corporation) ("Lykos"), a company dedicated to transforming mental healthcare, announced that the U.S. Food and Drug Administration ("FDA") has accepted its new drug application ("NDA") for midomafetamine capsules ("MDMA") used in combination with psychological intervention, which includes psychotherapy (talk therapy) and other supportive services provided by a qualified healthcare provider for individuals with post-traumatic stress disorder ("PTSD"). The FDA has granted the application priority review and has assigned a Prescription Drug User Fee Act ("PDUFA") target action date of August 11, 2024. If approved, this would be the first MDMA-assisted therapy and psychedelic-assisted therapy.

"Securing priority review for our investigational MDMA-assisted therapy is a significant accomplishment and underscores the urgent unmet need for new innovation in the treatment of PTSD," said Amy Emerson, chief executive officer of Lykos Therapeutics. "We remain focused on working with the FDA through the review process and preparing for a controlled launch with an emphasis on quality should this potential treatment be approved."

The NDA submission included results from numerous studies including two randomized, double-blind, placebo-controlled Phase 3 studies (MAPP1 and MAPP2) evaluating the efficacy and safety of MDMA used in combination with psychological intervention versus placebo with therapy in participants diagnosed with severe or moderate to severe PTSD, respectively. Both MAPP1 and MAPP2 studies met their primary and secondary endpoints and were published in Nature Medicine. 1, 2 The primary endpoint for both studies was to assess changes in PTSD symptom severity as measured by the change from baseline in Clinician-Administered PTSD Scale for DSM-5 ("CAPS-5"). The key secondary endpoint of both studies was to assess improvement in functional impairment associated with PTSD as measured by the change from baseline in the Sheehan Disability Scale ("SDS"). No serious adverse events were reported in the MDMA group in either study.

The FDA grants priority review 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. 

MDMA-assisted therapy has not been approved by any regulatory agency. The safety and efficacy of MDMA-assisted therapy has not been established for the treatment of PTSD. Investigational MDMA-assisted therapy is also being studied in other indications.

About MDMA-Assisted Therapy

MDMA (3,4-methylenedioxymethamphetamine) is not new to mental health professionals. MDMA is an entactogen— a class of psychoactive compounds that are differentiated from classic psychedelics (i.e., psilocybin, mescaline and LSD) and are defined based on their mechanism and known effects of increasing self-awareness leading to introspection and personal reflection.27,,28 In the 1970's and early 1980's MDMA was used in conjunction with talk therapy by mental health providers to enhance patients' access, processing, and communication of difficult emotions and experiences.29 In 1985, the U.S. Drug Enforcement Administration ("DEA") made MDMA a Schedule I drug under the Controlled Substances Act preventing it from being used for recreational or medical use. 30 Since then, research has shown the unique properties of MDMA allow it to act as a powerful catalyst to support psychotherapy by helping diminish the brain's fear response allowing people to access and process painful memories without being overwhelmed. 31 However, additional clinical trials would be needed to secure regulatory review and potential approval.

Lykos, with longstanding roots in advocacy for psychedelic medicine, pioneered the first randomized, double-blind, placebo controlled clinical trials evaluating the efficacy and safety of MDMA-assisted therapy as an investigational modality using midomafetamine (MDMA) in combination with psychological intervention to treat PTSD.

With a growing body of evidence supporting the potential medical use of MDMA, in 2017 the FDA granted the company's investigational MDMA-assisted therapy Breakthrough Therapy designation, a process designed to expedite the development and review of drugs intended to treat serious conditions for which preliminary scientific evidence indicates that it may demonstrate a substantial improvement over available therapies. If approved by the FDA, the U.S. Drug Enforcement Administration ("DEA") would be required to reschedule MDMA making it available for prescription medical use.

Ref: https://en.wikipedia.org/wiki/MDMA

Lykos Therapeutics Announces FDA Acceptance and Priority Review of New Drug Application for MDMA-Assisted Therapy for PTSD

Cidara Therapeutics Announces FDA Acceptance for Priority Review of New Drug Application for Rezafungin for the Treatment of Candidemia and Invasive Candidiasis

Cidara Therapeutics, Inc.  , a biotechnology company developing long-acting therapeutics designed to improve the standard of care for patients facing serious diseases,  announced   the  FDA acceptance for filing and granted Priority Review to its New Drug Application (NDA) for rezafungin for the treatment of candidemia and invasive candidiasis. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of March 22, 2023 enabled by rezafungin’s designation as a Qualified Infectious Disease Product (QIDP) and has indicated that it is currently planning to hold an advisory committee meeting to discuss the application. QIDP designation is reserved for antibacterial and antifungal drug candidates intended to treat serious or life-threatening infections. Rezafungin is a novel, once-weekly echinocandin antifungal being developed for the treatment of candidemia and invasive candidiasis, as well as for the prophylaxis of invasive fungal infections in patients undergoing allogeneic blood and marrow transplant.

“Today’s announcement is an important step forward for patients fighting difficult-to-treat and often deadly candidemia and invasive candidiasis, and represents a critical milestone for Cidara’s rezafungin development program,” said Jeff Stein, President, and CEO of Cidara Therapeutics. “The data generated across our Phase 2 and Phase 3 trials demonstrated that rezafungin could transform the current standard of care for the treatment of invasive Candida infections, and we are excited that rezafungin could potentially be the first new drug approved for this indication in over a decade.”

The NDA submission for rezafungin was supported by positive clinical data from the global ReSTORE Phase 3 and STRIVE Phase 2 clinical trials. Rezafungin dosed once-weekly demonstrated statistical non-inferiority versus caspofungin, the current standard of care, dosed once-daily, meeting the primary endpoints for both the FDA and the European Medicines Agency (EMA).

Cidara retains the rights to rezafungin in Japan and has licensed the commercial rights to Melinta Therapeutics in the U.S. and Mundipharma Medical in all other geographies.

About Rezafungin

Rezafungin is a novel once-weekly echinocandin being developed for both the treatment and prevention of serious fungal infections, such as candidemia and invasive candidiasis. The structure and properties of rezafungin are specifically designed to improve upon a clinically validated mechanism intended to enhance its efficacy and safety potential for patients. Cidara has completed a Phase 3 clinical trial with rezafungin for the first-line treatment of candidemia and/or invasive candidiasis (ReSTORE trial) and is currently conducting a second Phase 3 clinical trial of rezafungin for the prevention of invasive fungal disease in patients undergoing allogeneic blood and marrow transplantation (ReSPECT trial). Rezafungin has been designated a QIDP with Fast Track status by the FDA, and has been granted Orphan Drug Designation for its use in the treatment of invasive candidiasis in both the U.S. and EU.

Marinus Announces FDA Acceptance for New Drug Application for Ganaxolone in CDKL5 Deficiency Disorder

       

Marinus Pharmaceuticals, Inc.  announced  the U.S. FDA acceptance for filing the company’s New Drug Application (NDA) for the use of ganaxolone in the treatment of seizures associated with CDKL5 deficiency disorder (CDD), a rare, genetic epilepsy. The NDA was granted Priority Review designation and the FDA assigned a Prescription Drug User Fee Act (PDUFA) action date of March 20, 2022. Priority Review designation is given to an investigational medicine that, if approved, would be a significant improvement in the safety or effectiveness of the treatment of a serious condition and accelerates the timing of the FDA review of the application compared to a standard review.

“The FDA’s acceptance of our NDA submission is an important step toward potentially bringing the first approved therapy specifically for treatment of seizures associated with CDD—a devastating disorder with high unmet medical need—to families and healthcare providers,” said Scott Braunstein, M.D., Chief Executive Officer of Marinus Pharmaceuticals. “We believe that ganaxolone has the potential to provide meaningful clinical benefit for patients and we look forward to working closely with the FDA during the review process.”

Ganaxolone received orphan drug designation and Rare Pediatric Disease (RPD) designation for CDD in June 2017 and July 2020, respectively. If the NDA is approved, Marinus is eligible to receive a RPD Priority Review Voucher that may be sold or transferred.

In its acceptance letter, the FDA indicated that it is not currently planning to hold an advisory committee meeting to discuss the application.

The acceptance of the NDA for filing enables the company to draw $30 million of additional cash under its May 11, 2021 credit financing agreement with Oaktree Capital Management, L.P., subject to the satisfaction of certain customary conditions described in the credit agreement. If the NDA is approved by December 31, 2022, the company may draw an additional $30 million under the agreement, subject to the satisfaction of certain customary conditions described in the credit agreement.

The NDA is supported by data from the Marigold study, a Phase 3, double-blind placebo-controlled trial in 101 patients. Patients treated with ganaxolone showed a 30.7% median reduction in 28-day major motor seizure frequency, compared to a 6.9% reduction for those receiving placebo, achieving the trial’s primary endpoint (p=0.0036). Patients in the open-label extension study treated with ganaxolone for at least 12 months (n=48) experienced a median 49.6% reduction in major motor seizure frequency. In the Marigold trial, ganaxolone was generally well-tolerated and showed a safety profile consistent with previous clinical trials, with the most frequent adverse event being somnolence. 

Marinus has established an Expanded Access Program (EAP) (NCT04678479) for patients in the U.S. who may be eligible to receive access to ganaxolone during the review of the NDA. Additional information about Marinus’ EAP is available here.

More...

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

 

Obseva Announces U.S. FDA Acceptance of New Drug Application for Linzagolix

Obseva SA   announced   the New Drug Application (NDA) for linzagolix for the management of heavy menstrual bleeding associated with uterine fibroids in premenopausal women has been accepted for review by the United States Food and Drug Administration (FDA). The submission is based on data from the two Phase 3 PRIMROSE trials. Linzagolix has a differentiated profile and if approved, would be the first and only GnRH receptor antagonist with flexible dosing options for uterine fibroids, including a low dose option to address the needs of women who cannot or do not want to take hormones.1,4 The FDA set a target action date of September 13, 2022 for this NDA under the Prescription Drug User Fee Act (PDUFA).

“Today marks an important milestone not only in the linzagolix clinical development process, but for Obseva as a company, and most importantly, the millions of women living with uterine fibroids throughout the US. Linzagolix is a significant innovation in the field of women’s health – an area that is consistently underinvested in – and we are incredibly excited about the potential of bringing this important treatment to market” said Brian O’Callaghan, CEO of Obseva. “We are encouraged by our positive Phase 3 PRIMROSE results. If approved, we believe linzagolix will address a significant unmet need in offering a more individualized treatment option for a broader range of women.”

The Phase 3 PRIMROSE trials of linzagolix (PRIMROSE 1: US; n=574 and PRIMROSE 2: Europe and US; n=535) investigated the efficacy and safety of two dosing regimens, 100mg once daily and 200mg once daily, alone or in combination with hormonal ABT (1 mg estradiol and 0.5 mg norethisterone acetate) for the treatment of heavy menstrual bleeding associated with uterine fibroids. The NDA submission comprises positive 24-week treatment results from both studies, as well as supportive results from Week 52 and the 76-week post-treatment follow-up.

“Uterine fibroids can have a devastating impact on women’s day-to-day life. With its unique dosing options, linzagolix has the potential to significantly advance medical options for women,” stated Elizabeth Garner, MD, MPH, Chief Medical Officer of Obseva. “A dosing option without hormonal ABT would be welcomed by the significant number of women who either have contraindications to or a personal preference to avoid the use of estrogen-based therapies, while also providing a dosing option for women in whom hormonal ABT is indicated.”

The linzagolix marketing authorization application (MAA) was validated by the European Medicine Agency (EMA) with an approval recommendation from the Committee for Medicinal Products for Human Use (CHMP) expected in Q4 2021. Obseva announced previously that the company has entered into a partnership with Syneos Health to support commercialization of linzagolix in the US and EU.

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

https://www.drugs.com/nda/linzagolix_211122.html

Blueprint Medicines Announces FDA Acceptance of New Drug Application for Avapritinib for the Treatment of PDGFRA Exon 18 Mutant GIST and Fourth-Line GIST

Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced that the U.S. Food and Drug Administration (FDA) has accepted the company's New Drug Application (NDA) for avapritinib for the treatment of adult patients with PDGFRA Exon 18 mutant gastrointestinal stromal tumors (GIST), regardless of prior therapy, and fourth-line GIST. The FDA granted Priority Review and set an action date of February 14, 2020 under the Prescription Drug User Fee Act (PDUFA). At this time, the FDA is not planning to hold an advisory committee meeting to discuss this application. Avapritinib, an investigational therapy, is a potent and highly selective KIT and PDGFRA inhibitor for patients with advanced GIST.

"Patients with PDGFRA Exon 18 mutant GIST and fourth-line GIST are in need of new treatment options that address the underlying drivers of the disease," said Andy Boral, M.D., Ph.D., Chief Medical Officer at Blueprint Medicines. "The FDA's acceptance of our application for Priority Review brings us closer to our goal of delivering avapritinib to patients with GIST, and we look forward to working closely with the FDAduring the review process."

The FDA's acceptance of the NDA indicates the application is sufficiently complete to permit a substantive review. A Priority Review designation accelerates the FDA's review time from 12 months to a goal of eight months from the NDA submission date, and is granted to drugs that may offer a significant improvement in the safety or effectiveness of the treatment, prevention or diagnosis of a serious condition. Previously, the FDA granted avapritinib Breakthrough Therapy Designation for the treatment of patients with unresectable or metastatic GIST harboring the PDGFRα D842V mutation.

In July 2019, the European Medicines Agency validated Blueprint Medicines' Marketing Authorization Application for avapritinib in adult patients with PDGFRα D842V mutant GIST, regardless of prior therapy, and fourth-line GIST.

About GIST

GIST is a sarcoma, or tumor of bone or connective tissue, of the gastrointestinal (GI) tract. Tumors arise from cells in the wall of the GI tract and occur most often in the stomach or small intestine. Most patients are diagnosed between the ages of 50 to 80, and diagnosis is typically triggered by GI bleeding, incidental findings during surgery or imaging and, in rare cases, tumor rupture or GI obstruction.

Most GIST cases are caused by a spectrum of clinically relevant mutations that force the KIT or PDGFRA protein kinases into an increasingly active state. Because currently available therapies primarily bind to the inactive protein conformations, certain primary and secondary mutations typically lead to treatment resistance and disease progression.

In unresectable or metastatic GIST, clinical benefits from existing treatments can vary by mutation type. Mutational testing is critical to tailor therapy to the underlying disease driver and is recommended in expert guidelines. Currently, there are no approved therapies for patients with KIT-driven GIST whose disease progresses beyond imatinib, sunitinib and regorafenib. In patients with metastatic PDGFRα D842V-driven GIST, progression occurs in a median of approximately three to four months with available therapy.

https://pubchem.ncbi.nlm.nih.gov/compound/Avapritinib#section=2D-Structure

Eton Pharmaceuticals Announces FDA Acceptance of New Drug Application for ET-105

In continuation of my update on  lamotrigine

Eton Pharmaceuticals, Inc (Nasdaq: ETON), a specialty pharmaceutical company focused on developing and commercializing innovative drug products, today announced that Aucta Pharmaceuticals' New Drug Application for ET-105, an innovative formulation of lamotrigine which Eton acquired the U.S. marketing rights to in June 2019, has been accepted for review by the U.S. Food and Drug Administration (FDA). The FDA has assigned the application a Prescription Drug User Fee Act (PDUFA) target action date of March 17, 2020.

“The NDA acceptance of ET-105 marks an important milestone for Eton as this strengthens our growing pipeline of near-launch products. We are very excited about the potential for ET-105 to address a significant unmet need in this large and growing market,” said Sean Brynjelsen, Chief Executive Officer of Eton Pharmaceuticals. “Our team looks forward to working with Aucta and the FDA over the coming months as we prepare for a potential commercial launch in the first half of 2020.”

ET-105 is a patent-pending formulation of lamotrigine, for which Aucta is seeking approval as an adjunct therapy for partial seizures, primary generalized tonic-clonic seizures, and generalized seizures of Lennox-Gastaut syndrome in patients two years of age and older. Lamotrigine is one of the most widely used anti-epilepsy medications in the U.S. with sales exceeding $700 million and 1 billion tablets annually but is only FDA-approved in tablet formulations. ET-105’s innovative formula will be delivered to patients as an oral liquid and has been developed specifically to address the significant unmet need in patients with dysphagia and pediatric patients requiring precision dosing at levels below the currently available tablet strengths.