Prescription Drug User Fee Act

Valneva Submits Chikungunya Vaccine Marketing Application to EMA and Announces CHMP Accelerated Assessment

Retrieved on: 
Wednesday, October 25, 2023
FDA, Chikungunya, Biologics license application, VLA, PDUFA, Food, CHMP, Priority, Prescription Drug User Fee Act, European Directive on Traditional Herbal Medicinal Products, MD, Health Canada, European Medicines Agency, Regulation of tobacco by the U.S. Food and Drug Administration, Valneva SE, Euronext Paris, Saint-Herblain, CHIKV, Risk, EMA, Public, Fast track (FDA), BLA, Lancet, Pharmaceutical industry

Saint-Herblain (France), October 25, 2023 – Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company, today announces the submission of a marketing application with the European Medicines Agency (EMA) for approval of the Company’s single-shot chikungunya vaccine candidate, VLA1553.

Key Points: 
  • Saint-Herblain (France), October 25, 2023 – Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company, today announces the submission of a marketing application with the European Medicines Agency (EMA) for approval of the Company’s single-shot chikungunya vaccine candidate, VLA1553.
  • Valneva was also granted accelerated assessment1 for the application by EMA’s Committee for Medicinal Products for Human Use (CHMP) based on the vaccine candidate’s “major interest for public health and therapeutic innovation”.
  • VLA1553 is currently the first and only chikungunya vaccine candidate worldwide for which regulatory review processes are underway.
  • Juan Carlos Jaramillo, MD, Chief Medical Officer of Valneva, commented, “We welcome EMA’s accelerated assessment and will work closely with them to bring this vaccine to market.

US Food and Drug Administration accepts for review AstraZeneca’s Supplemental Biologics License Application for self-administration of FLUMIST® QUADRIVALENT (Influenza Vaccine Live, Intranasal)

Retrieved on: 
Tuesday, October 24, 2023
Oncology, FDA, Health, Pharmaceutical, Cardiology, Biotechnology, Allergy, Child, AstraZeneca, PDUFA, Heart, Nursing, History, Prescription Drug User Fee Act, MD, Vaccine, Wheeze, US Foods, Aspirin, Diabetes, University School, Fever, Vaccination, Caregiver, Egg, Immune system, Risk, Safety, Feinberg School of Medicine, Amantadine, Rimantadine, Nasal administration, Patient, Parent, Nose, Pediatrics, Infection

AstraZeneca’s Supplemental Biologics License Application (sBLA) for the approval of a self- or caregiver-administered option for FLUMIST® QUADRIVALENT (Influenza Vaccine Live, Intranasal), a needle-free nasal spray, has been accepted for review by the US Food and Drug Administration (FDA).

Key Points: 
  • AstraZeneca’s Supplemental Biologics License Application (sBLA) for the approval of a self- or caregiver-administered option for FLUMIST® QUADRIVALENT (Influenza Vaccine Live, Intranasal), a needle-free nasal spray, has been accepted for review by the US Food and Drug Administration (FDA).
  • FLUMIST QUADRIVALENT, which is sprayed into the nose, has extensive data demonstrating comparable effectiveness and acceptable safety relative to other flu vaccines.
  • If approved at that time, FLUMIST QUADRIVALENT is anticipated to be available for self-administration in the US for the 2024/2025 flu season.
  • FLUMIST QUADRIVALENT now has the potential to be the first and only self-administered flu vaccine, which could revolutionize flu vaccination.

Mirum Pharmaceuticals Announces New PDUFA Date for LIVMARLI for the Treatment of Cholestatic Pruritus in Progressive Familial Intrahepatic Cholestasis

Retrieved on: 
Tuesday, October 17, 2023
Health, FDA, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Prescription Drug User Fee Act, PFIC, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, PDUFA, Shanda, Cholestatic pruritus, Patient, Medication, Pharmaceutical industry, Generic drug

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has extended the review of the Supplemental New Drug Application (sNDA) for LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC).

Key Points: 
  • Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has extended the review of the Supplemental New Drug Application (sNDA) for LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC).
  • The new Prescription Drug User Fee Act (PDUFA) date is March 13, 2024.
  • The FDA extended the PDUFA date to allow time for a full review of a submission provided in response to an FDA information request, which the FDA deemed a major amendment.
  • The submission comprised further analysis of certain clinical information that was previously provided to the FDA by the company.

Basilea reports new data for ceftobiprole (Zevtera®) presented at US IDWeek Congress 2023

Retrieved on: 
Tuesday, October 17, 2023
Efficacy, Staphylococcus, Methicillin-resistant Staphylococcus aureus, Ambrose, Abstract, Treatment, Clinical, Circulatory system, MRSA, Biomedical Advanced Research and Development Authority, BARDA, Enterococcus faecalis, Infection, ASPR, MSSA, IDSA, Isolates, Laboratory, Liolios Xirolivaditis, Staphylococcus aureus, PK/PD models, SAB, ABSSSI, CABP, Bronchopneumonia, Administration for Strategic Preparedness and Response, Streptococcus pneumoniae, Prescription Drug User Fee Act, Duke University School of Medicine, Infectious Diseases Society of America, Randomness, Development, Administration, Safety, Patient, PDUFA, HHS, Pharmaceutical industry, Vaccine, Ceftobiprole, Litherland, Enterococcus, Allschwil, Streptococcus, Bloodstream infections

Dr. Marc Engelhardt, Chief Medical Officer of Basilea, stated: “The data presented at IDWeek provide further evidence for the potent antimicrobial activity of ceftobiprole against MRSA and other clinically relevant pathogens.

Key Points: 
  • Dr. Marc Engelhardt, Chief Medical Officer of Basilea, stated: “The data presented at IDWeek provide further evidence for the potent antimicrobial activity of ceftobiprole against MRSA and other clinically relevant pathogens.
  • It also provides results of pharmacokinetic-pharmacodynamic modeling supporting the dosing regimens to treat severe bacterial infections from the successful clinical phase 3 studies.
  • Abstract #1946 – In Vitro Activity of Ceftobiprole against Staphylococcus aureus Bacteremia Isolates from the United States (2018–2020) – L. Duncan, M. Castanheira, J. I.
  • Smart, M. E. Jones, P. G. Ambrose, K. Litherland
    Abstract #2561 – Population Pharmacokinetic Analyses for Ceftobiprole Using Data from Phase 1 and 3 Studies – A. P. Cammarata, K. Litherland, M. C. Safir, S. M. Bhavnani, M. Saulay, J. I.

TAGRISSO® Plus Chemotherapy Granted Priority Review in the US for Patients with EGFR-Mutated Advanced Lung Cancer

Retrieved on: 
Monday, October 16, 2023
Oncology, FDA, Health, Clinical Trials, General Health, Pharmaceutical, Cardiomyopathy, Priority review, HIV disease progression rates, Lung cancer, Food, Death, NSCLC, Standard of care, Risk, Safety, Prescription Drug User Fee Act, Heart, AstraZeneca, LVEF, Criticism of the Food and Drug Administration, Wireless ambulatory ECG, Electrolyte, QT interval, Progression-free survival, Patient, Pharmaceutical industry, Medical imaging

Importantly, a clinically meaningful PFS benefit was observed across all prespecified subgroups, including patients with central nervous system metastasis.

Key Points: 
  • Importantly, a clinically meaningful PFS benefit was observed across all prespecified subgroups, including patients with central nervous system metastasis.
  • At the time of this analysis, the overall survival (OS) data were immature, however, a favorable trend was observed for TAGRISSO plus chemotherapy.
  • The safety profile of TAGRISSO plus chemotherapy was generally manageable and consistent with the established profiles of the individual medicines.
  • AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death.

Merck Announces Pivotal KEYNOTE-671 Trial Meets Dual Primary Endpoint of Overall Survival (OS) in Resectable Stage II, IIIA or IIIB Non-Small Cell Lung Cancer (NSCLC)

Retrieved on: 
Tuesday, October 10, 2023
Oncology, Health, FDA, General Health, Clinical Trials, Pharmaceutical, Biotechnology, MSD, Congress, MRK, NYSE, Lung cancer, Food, Research, NSCLC, MPR, SCLC, OS, Prescription Drug User Fee Act, Society, American Society of Clinical Oncology, Survival, Disease, ESMO, Merck & Co., EFS, Carcinoma, Head, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, European Society for Medical Oncology, ASCO, PCR, PDUFA, Rollcage Stage II, Patient, Pharmaceutical industry, Merck

The safety profile of KEYTRUDA was consistent with that observed in previously reported studies; no new safety signals were identified.

Key Points: 
  • The safety profile of KEYTRUDA was consistent with that observed in previously reported studies; no new safety signals were identified.
  • “This is a significant milestone in the treatment of resectable non-small cell lung cancer, as it represents the first Phase 3 study to show a statistically significant overall survival benefit for these patients with stage II, IIIA or IIIB (T3-4N2) non-small cell lung cancer.
  • Merck has an extensive clinical development program in lung cancer and is advancing multiple registration-enabling studies, with research directed at earlier stages of disease and novel combinations.
  • Key studies in earlier stages of NSCLC and small cell lung cancer (SCLC) include KEYNOTE-671, KEYNOTE-091, KEYNOTE-867, KEYLYNK-012, KEYVIBE-006 and KEYLYNK-013.

Alnylam to Present Additional Data for Patisiran in Patients With the Cardiomyopathy of ATTR Amyloidosis, Including 24-Month Results From the APOLLO-B Phase 3 Study, at the Heart Failure Society of America Annual Scientific Meeting 2023

Retrieved on: 
Friday, October 6, 2023
Health, Other Health, Research, Science, Pharmaceutical, Cardiology, Biotechnology, ATTR, Cardiomyopathy, Premedication, Food, IRR, OLE, Heart Failure Society of America, Shanda, Nyctalopia, Risk, Therapy, Amyloidosis, Safety, RNA interference, Vitamin A deficiency, Abdominal pain, Patisiran, Nausea, Headache, FDA, Health status of Asian Americans, Polyneuropathy, Paracetamol, Vitamin A, Periodic breathing, ASM, Back pain, Patient, Medication, New Drug Application, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, RDA, APOLLO, PDUFA, EAP, Serum, Prescription Drug User Fee Act, Quality of life, Pharmaceutical industry, Dietary supplement

Data from an interim analysis of the ongoing open-label extension (OLE) period of the APOLLO-B Phase 3 study of patisiran in patients with the cardiomyopathy of ATTR amyloidosis through 24 months will be presented.

Key Points: 
  • Data from an interim analysis of the ongoing open-label extension (OLE) period of the APOLLO-B Phase 3 study of patisiran in patients with the cardiomyopathy of ATTR amyloidosis through 24 months will be presented.
  • Patisiran is the established name for ONPATTRO®, which is approved by the U.S. FDA for the treatment of the polyneuropathy of hereditary ATTR amyloidosis in adults.
  • In a controlled clinical study, 19% of ONPATTRO-treated patients experienced IRRs, compared to 9% of placebo-treated patients.
  • Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness).

Theratechnologies Receives January 2024 PDUFA Goal Date for Tesamorelin F8 Formulation sBLA

Retrieved on: 
Wednesday, October 4, 2023
Connective tissue, Lipodystrophy, HIV, Prescription Drug User Fee Act, PDUFA, TSX, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, News, Food, F4, Medical device, Pharmaceutical industry

MONTREAL, Oct. 04, 2023 (GLOBE NEWSWIRE) -- Theratechnologies Inc. (“Theratechnologies” or the “Company”) (TSX: TH) (NASDAQ: THTX), a biopharmaceutical company focused on the development and commercialization of innovative therapies, today announced that the U.S. Food and Drug Administration (FDA) has assigned a Prescription Drug User Fee Act (PDUFA) goal date of January 22, 2024 to the Company’s supplemental Biologics License Application (sBLA) of the F8 formulation of tesamorelin.

Key Points: 
  • MONTREAL, Oct. 04, 2023 (GLOBE NEWSWIRE) -- Theratechnologies Inc. (“Theratechnologies” or the “Company”) (TSX: TH) (NASDAQ: THTX), a biopharmaceutical company focused on the development and commercialization of innovative therapies, today announced that the U.S. Food and Drug Administration (FDA) has assigned a Prescription Drug User Fee Act (PDUFA) goal date of January 22, 2024 to the Company’s supplemental Biologics License Application (sBLA) of the F8 formulation of tesamorelin.
  • Tesamorelin is the only medication approved in the U.S. for the reduction of excess abdominal fat in adults with HIV who have lipodystrophy.
  • Once approved, the F8 formulation is set to replace the current F4 formulation, which is sold in the U.S. under the trade name EGRIFTA SV®.
  • The proposed proprietary name for the F8 formulation of tesamorelin, EGRIFTA MDVTM, is already under review by the FDA.

Basilea announces FDA acceptance of New Drug Application for antibiotic ceftobiprole

Retrieved on: 
Monday, October 2, 2023
SAB, ASPR, US Foods, Prescription Drug User Fee Act, Partnership, Biomedical Advanced Research and Development Authority, HHS, QIDP, Bronchopneumonia, Administration for Strategic Preparedness and Response, LR, NDA, CABP, Ceftobiprole, ABSSSI, GAIN, Administration, Development, Patient, PDUFA, Infection, Fungal infection, FDA, Safety, BARDA, TARGET, Pharmaceutical industry, Allschwil, New Drug Application

Prescription Drug User Fee Act (PDUFA) goal date set for April 03, 2024

Key Points: 
  • Prescription Drug User Fee Act (PDUFA) goal date set for April 03, 2024
    Ad hoc announcement pursuant to Art.
  • 53 LR
    Basilea Pharmaceutica Ltd, Allschwil (SIX: BSLN), a commercial-stage biopharmaceutical company committed to meeting the needs of patients with severe bacterial or fungal infections, announced today that the US Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) for the antibiotic ceftobiprole, which was submitted to the FDA on August 3rd this year.
  • The FDA has set April 03, 2024, as the Prescription Drug User Fee Act (PDUFA) goal date.
  • Basilea is planning to commercialize ceftobiprole in the US through a partner and intends to enter into such a partnership prior to the PDUFA goal date.

HUTCHMED Announces Submission of New Drug Application for Fruquintinib for Previously Treated Metastatic Colorectal Cancer in Japan

Retrieved on: 
Friday, September 29, 2023
Priority review, CRC, Progression-free survival, TAK, European Medicines Agency, JAMA, NDA, Ministry of Health, Labour and Welfare, Woman, EMA, American Medical Association, Ministry, Incidence, HKEX, Journal, Prescription Drug User Fee Act, Patient, PDUFA, Federal Ministry of Health (Germany), BSC, Mortality, MAA, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Pharmaceutical industry, Takeda, Health, Lancet

Fruquintinib is a selective inhibitor of vascular endothelial growth factor receptors (“VEGFR”) -1, -2 and -3, which play a pivotal role in blocking tumor angiogenesis.

Key Points: 
  • Fruquintinib is a selective inhibitor of vascular endothelial growth factor receptors (“VEGFR”) -1, -2 and -3, which play a pivotal role in blocking tumor angiogenesis.
  • The FRESCO-2 and FRESCO clinical trials compared fruquintinib plus best supportive care (“BSC”) with placebo plus BSC in patients with previously treated metastatic CRC.
  • The U.S. Food and Drug Administration (“FDA”) granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November 30, 2023.
  • A Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) was validated and accepted for regulatory review in June 2023.