Prescription Drug User Fee Act

Valneva Reports Positive Initial Phase 3 Safety Data in Adolescents for its Single-Shot Chikungunya Vaccine Candidate

Retrieved on: 
Monday, August 28, 2023
Epidemic, Biologics license application, DSMB, Saint-Herblain, Coalition, European Medicines Agency, Valneva SE, CEPI, Clinical trial, Coalition for Epidemic Preparedness Innovations, Licensure, Prescription Drug User Fee Act, EMA, Euronext Paris, Food and Drug Administration Amendments Act of 2007, VLA, Instituto Butantan, PDUFA, Adolescence, Ageing, FDA, Lancet, BLA, Food, Safety, CHIKV, Vaccine, Pharmaceutical industry, Chikungunya

Saint-Herblain (France), August 28, 2023 – Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company, today reported positive initial Phase 3 safety data in adolescents for its single-dose chikungunya virus (CHIKV) vaccine candidate VLA1553.

Key Points: 
  • Saint-Herblain (France), August 28, 2023 – Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company, today reported positive initial Phase 3 safety data in adolescents for its single-dose chikungunya virus (CHIKV) vaccine candidate VLA1553.
  • An independent DSMB has continuously evaluated safety data during the trial and has not identified any safety concerns.
  • Importantly, the initial data suggest a favorable safety profile in seropositive participants, confirming the observations following re-vaccination of individuals in Phase 1 trial VLA1553-1011.
  • If approved, VLA1553 could become the first licensed chikungunya vaccine available to address this unmet medical need.

bluebird bio Confirms That FDA Has Communicated That Advisory Committee Meeting Will Not Be Scheduled for lovo-cel Gene Therapy for Sickle Cell Disease

Retrieved on: 
Wednesday, August 16, 2023
Science, Research, Pharmaceutical, General Health, Health, FDA, Genetics, Other Health, Priority review, SCD, Voe Minas Gerais, History, Regenerative medicine, Prescription Drug User Fee Act, Patient, PDUFA, Sickle cell disease, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BLA, Food, Safety, Pharmaceutical industry, Agency

bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has communicated that an advisory committee meeting will not be scheduled for lovotibeglogene autotemcel (lovo-cel).

Key Points: 
  • bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has communicated that an advisory committee meeting will not be scheduled for lovotibeglogene autotemcel (lovo-cel).
  • Lovo-cel is a potentially transformative one-time gene therapy for individuals living with sickle cell disease (SCD) with a proposed indication for patients ages 12 and older who have a history of vaso-occlusive events (VOEs).
  • “Lovo-cel is the most deeply studied gene therapy in development for sickle cell disease and represents the third lentiviral vector gene therapy that the Agency has reviewed from bluebird—giving us great confidence in the robustness and maturity of our BLA package,” said Andrew Obenshain, chief executive officer, bluebird bio.
  • The FDA previously granted lovo-cel orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation, and rare pediatric disease designation.

Updated Data from TRIDENT-1 Trial Show Durable Efficacy Benefits with Repotrectinib for Patients with Locally Advanced or Metastatic ROS1-Positive Non-Small Cell Lung Cancer

Retrieved on: 
Wednesday, August 16, 2023
Oncology, Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Bristol Myers Squibb, Priority review, Progression-free survival, DOR, New Drug Application, International Association, Lung cancer, Prescription Drug User Fee Act, Hope, TKI, NSCLC, Brain, Immunotherapy, EDT, PFS, BMY, Yonsei University, Phase 1, Patient, NTRK, ROS1, Corr, Therapy, Abstract, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Conference, NYSE, ORR, Food, Pharmaceutical industry, Medical imaging

In this updated analysis, repotrectinib continued to demonstrate durable efficacy in patients with ROS1-positive NSCLC, including intracranial activity, in patients who were TKI-naïve or previously treated with one TKI and no chemotherapy.

Key Points: 
  • In this updated analysis, repotrectinib continued to demonstrate durable efficacy in patients with ROS1-positive NSCLC, including intracranial activity, in patients who were TKI-naïve or previously treated with one TKI and no chemotherapy.
  • In patients with measurable brain metastases at baseline (n=9), intracranial ORR per BICR was 89% and responses were prolonged.
  • At the recommended dose for Phase 2, the safety profile of repotrectinib was manageable and remained consistent with previous reports.
  • Furthermore, the data offer hope for the patients with ROS1-positive non-small cell lung cancer who still face high remaining unmet needs,” said Joseph Fiore, executive director, global program lead, repotrectinib, Bristol Myers Squibb.

Wugen Begins First-in-Human Phase 1 Clinical Trial of WU-NK-101, its Lead Allogeneic Memory Natural Killer (NK) Cell Therapy for Patients with Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML)

Retrieved on: 
Tuesday, August 15, 2023
Research, FDA, Genetics, Clinical Trials, Biotechnology, Health, Pharmaceutical, Science, Oncology, Orphan Drug Act of 1983, Acute leukemia, CIML, Marketing, ODD, Clinical trial, Pharmacokinetics, Prescription Drug User Fee Act, NK, Cell, AML, Patient, American Society of Hematology, TME, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Phenotype, Memory, News, Society, Food, Safety, Disease, Pharmaceutical industry, Vaccine, Blood

Additionally, Wugen announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to WU-NK-101 for the treatment of AML.

Key Points: 
  • Additionally, Wugen announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to WU-NK-101 for the treatment of AML.
  • WU-NK-101 is Wugen’s lead memory natural killer (NK) cell therapy product and is comprised of cells optimized for anti-cancer function.
  • WU-NK-101 is produced with the company’s proprietary MonetaTM manufacturing platform, which enables robust generation of off-the-shelf memory NK cell-based therapies.
  • “Early clinical studies with memory NK cells demonstrated impressive response rates in patients with AML, even those with a high disease burden.

Venatorx Pharmaceuticals Announces FDA Acceptance and Priority Review of New Drug Application for Cefepime-Taniborbactam to Treat Complicated Urinary Tract Infections (cUTI), including Pyelonephritis, in Adults

Retrieved on: 
Tuesday, August 15, 2023
Health, FDA, Infectious Diseases, Clinical Trials, Research, Science, Pharmaceutical, Priority review, Senior, Pyelonephritis, Medicine, Infection, New Drug Application, NDA, Cutis verticis gyrata, Prescription Drug User Fee Act, Cutis, Medication, Urinary tract infection, Genetically modified bacteria, Patient, Test, PDUFA, Viral, Meropenem, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Safety, Pharmaceutical industry

The FDA previously granted Qualified Infectious Disease Product and Fast Track designations to cefepime-taniborbactam.

Key Points: 
  • The FDA previously granted Qualified Infectious Disease Product and Fast Track designations to cefepime-taniborbactam.
  • The FDA has granted Priority Review of the NDA with a Prescription Drug User Fee Act (PDUFA) target action date of February 22, 2024.
  • “The NDA acceptance represents the culmination of unwavering dedication, scientific excellence, and the collaborative efforts of our talented team, partners, and clinical investigators,” said Christopher J. Burns, Ph.D., Chief Executive Officer of Venatorx.
  • “Patients with cUTIs, including pyelonephritis, and their healthcare providers should have a new treatment option when confronted with infections due to these antibacterial resistant infections.”

Formosa Pharmaceuticals Announces Licensing Agreement with Eyenovia, Inc., for the Commercialization of APP13007 for the Treatment of Inflammation and Pain Following Ocular Surgery

Retrieved on: 
Wednesday, August 16, 2023
PDUFA, Pain, Geography of Taiwan, FDA, Prescription Drug User Fee Act, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Inflammation, Food, Pharmaceutical industry

The total deal package, including upfront payment and development and sales milestones, has a value of $86 million dollars, with additional considerations throughout the term of the agreement.

Key Points: 
  • The total deal package, including upfront payment and development and sales milestones, has a value of $86 million dollars, with additional considerations throughout the term of the agreement.
  • APP13007's active ingredient is the superpotent corticosteroid, clobetasol propionate, and is derived from Formosa Pharma's proprietary APNT™ nanoparticle formulation platform.
  • "We are pleased to have entered into this agreement with our development partner, Formosa Pharmaceuticals, to acquire the U.S. commercial rights to APP13007.
  • If approved, APP13007 would be an attractive new treatment option for the aftereffects of ocular surgery, most notably inflammation and pain," stated Michael Rowe, Chief Executive Officer of Eyenovia.

Everest Medicines' Partner Venatorx Receives FDA Acceptance and Priority Review of New Drug Application for Cefepime-Taniborbactam to Treat Complicated Urinary Tract Infections (cUTI)

Retrieved on: 
Tuesday, August 15, 2023
Priority review, Pyelonephritis, Public, Infection, New Drug Application, NDA, Tuberculosis verrucosa cutis, Prescription Drug User Fee Act, AMR, Trial of the century, Urinary tract infection, MDR, HKEX, Patient, Test, PDUFA, Meropenem, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Safety, Pharmaceutical industry

The FDA has granted Priority Review of the NDA with a Prescription Drug User Fee Act (PDUFA) target action date of Feb. 22, 2024.

Key Points: 
  • The FDA has granted Priority Review of the NDA with a Prescription Drug User Fee Act (PDUFA) target action date of Feb. 22, 2024.
  • "We congratulate our partner for achieving this important milestone and bringing cefepime-taniborbactam a step closer to reaching patients confronted with antibacterial resistant infections.
  • "In Asia, the challenges we are facing from MDR infections are more urgent and severe.
  • The company is also actively developing other important antibacterial drug candidates for combating the increasing MDR infections in China and the rest of Asia.

Valneva Announces PDUFA Date Extension for Chikungunya Virus Vaccine Candidate

Retrieved on: 
Monday, August 14, 2023
Biologics license application, Prescription Drug User Fee Act, PRV, Saint-Herblain, European Medicines Agency, Valneva SE, Chikungunya, EMA, Euronext Paris, VLA, PDUFA, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BLA, Food, Pharmaceutical industry

The FDA extended the PDUFA date to allow sufficient time to align and agree on the phase 4 program necessary under the accelerated approval pathway.

Key Points: 
  • The FDA extended the PDUFA date to allow sufficient time to align and agree on the phase 4 program necessary under the accelerated approval pathway.
  • Valneva is committed to working with the FDA in its ongoing review of the BLA, and to potentially delivering the world’s first chikungunya vaccine.
  • This PDUFA extension does not impact Valneva’s current regulatory submission in Canada or its planned submission with the European Medicines Agency (EMA).
  • We are continuing to work closely and collaboratively with the FDA, and we believe it may be possible to obtain an approval before the new PDUFA date.”

ARS Reports Second Quarter 2023 Financial Results and Provides Business Updates

Retrieved on: 
Thursday, August 10, 2023
Patent, Prescription Drug User Fee Act, Allergy, ARS, Marketing, Anxiety, Advisory Committee, European Medicines Agency, New Drug Application, Adrenaline, NDA, G&A, Hives, U.S. FDA, Tobacco Products Scientific Advisory Committee, Investment, Patient, Anaphylaxis, SAN, PDUFA, Nasal administration, R, MAA, Research, FDA, Regulation of tobacco by the U.S. Food and Drug Administration, Food, Caregiver, D, Pharmaceutical industry, Fine chemical

SAN DIEGO, Aug. 10, 2023 (GLOBE NEWSWIRE) -- ARS Pharmaceuticals, Inc. (Nasdaq: SPRY), a biopharmaceutical company dedicated to empowering at-risk patients and caregivers to better protect themselves from severe allergic reactions that could lead to anaphylaxis, today reported business updates and financial results for the second quarter of 2023.

Key Points: 
  • Topline data from the company’s ongoing Phase 2 randomized, placebo-controlled study of neffy in urticaria patients are expected by the fourth quarter of 2023.
  • R&D Expenses: Research and development (R&D) expenses were $7.3 million for the quarter ended June 30, 2023.
  • G&A Expenses: General and administrative (G&A) expenses were $13.3 million for the quarter ended June 30, 2023.
  • Net Loss: Net loss was $17.4 million for the quarter ended June 30, 2023.

U.S. FDA Accepts Astellas' sNDA for CRESEMBA® (isavuconazonium sulfate) in Children

Retrieved on: 
Thursday, August 10, 2023
Prescription Drug User Fee Act, Head, New Drug Application, Pharmacokinetics, Research, Doctor of Philosophy, Shanda, MD, Patient, Azole, Physician, Blood, Cancer, Disease, PDUFA, Ageing, Trial of the century, Immunodeficiency, Fungal infection, FDA, Death, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Isavuconazonium, Food, Safety, Pharmaceutical industry, Astellas Pharma

NORTHBROOK, Ill., Aug. 10, 2023 /PRNewswire/ -- Astellas Pharma US, Inc. (President: Mark Reisenauer, "Astellas") announced that the U.S. Food and Drug Administration (FDA) has accepted the company's supplemental New Drug Application (sNDA) for CRESEMBA® (isavuconazonium sulfate), a prodrug of isavuconazole, an azole antifungal drug, seeking approval for the treatment of invasive aspergillosis (IA) or invasive mucormycosis (IM) in pediatric patients aged one to 17 years old. Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of December 9, 2023.

Key Points: 
  • Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of December 9, 2023.
  • CRESEMBA is already approved by the FDA for the treatment of IA and IM in adults.
  • If approved for pediatric use, it may offer children significant advances in treatment or may provide a treatment where no adequate therapy exists.
  • "This sNDA acceptance by the FDA brings Astellas one step closer to helping pediatric patients by potentially having a new treatment option available for IA and IM for a younger patient population, if approved."