PDUFA

U.S. FDA Accepts for Priority Review the Supplemental Biologics License Application for Epcoritamab (EPKINLY®) for Difficult-to-Treat Relapsed or Refractory Follicular Lymphoma

Retrieved on: 
Tuesday, February 27, 2024
Oncology, FDA, Health, Clinical Trials, Pharmaceutical, Biotechnology, ABBV, BTD, ASH, Food, AbbVie, PDUFA, Exposition, Annual general meeting, Safety, Society, Patient, Diagnosis, Priority review, Pharmaceutical industry

The FDA grants Priority Review to investigational therapies that, if approved, may offer significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.

Key Points: 
  • The FDA grants Priority Review to investigational therapies that, if approved, may offer significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.
  • “The acceptance of the epcoritamab application for Priority Review marks an important milestone toward potentially providing a new treatment option to patients affected by R/R follicular lymphoma.
  • The FDA previously granted Breakthrough Therapy Designation (BTD) to epcoritamab for the treatment of adult patients with R/R FL after two or more lines of systemic therapy.
  • The companies will share commercial responsibilities in the U.S. and Japan, with AbbVie responsible for further global commercialization.

Incyte Announces U.S. Food and Drug Administration Grants Priority Review for Axatilimab for the Treatment of Chronic Graft-Versus-Host Disease

Retrieved on: 
Tuesday, February 27, 2024
FDA, Health, Stem Cells, Clinical Trials, Pharmaceutical, Biotechnology, Food, BLA, PDUFA, SNDX, GVHD, Safety, Society, Patient, Priority, Biologics license application, Priority review, Pharmaceutical industry

Incyte (Nasdaq:INCY) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for axatilimab, an anti-CSF-1R antibody, for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy.

Key Points: 
  • Incyte (Nasdaq:INCY) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for axatilimab, an anti-CSF-1R antibody, for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy.
  • The Prescription Drug User Fee Act (PDUFA) date for the FDA decision is August 28, 2024.
  • Axatilimab is being developed by Incyte and Syndax Pharmaceuticals (Nasdaq:SNDX) as part of an exclusive worldwide co-development and co-commercialization license agreement.
  • “Axatilimab’s novel mechanism offers a differentiated treatment approach which may help patients suffering from this devastating disease.

Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Progress

Retrieved on: 
Monday, February 26, 2024
FDA, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, PKD, Personalized medicine, EMA, Research, MAA, Investment, Marketing, Food, Arrhythmogenic cardiomyopathy, Circulation, BLA, PDUFA, Fanconi anemia, Conference, Safety, DCM, European Medicines Agency, Food and Drug Administration, Muscle weakness, Efgartigimod alfa, Pyruvate kinase deficiency, Patient, Disorder, Clinical trial, Medication, ACM, IND, AAV, CGMP, CMC, Priority review, Pharmaceutical industry

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2023.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2023.
  • “I am pleased with the strong results Rocket delivered in 2023, as we closed another successful year of progress across all six disclosed gene therapy programs spanning our AAV cardiovascular and LV hematology portfolios,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma.
  • Results from the study demonstrated the potential of RP-A601 as a gene therapy for patients with PKP2-ACM.
  • As of December 31, 2023, Rocket had cash, cash equivalents and investments of $407.5 million.

Karuna Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Provides General Business Update

Retrieved on: 
Thursday, February 22, 2024
Mental Health, Health, Neurology, Clinical Trials, Pharmaceutical, Biotechnology, Acquisition, PANSS, Blood pressure, KRTX, NEI, FDA, Food, Schizophrenia, PDUFA, Psychiatry, Bristol Myers Squibb, Safety, Psychosis, Therapy, Patient, Congress, Positive, NDA, Pharmaceutical industry

Karuna Therapeutics, Inc. (NASDAQ: KRTX), a biopharmaceutical company driven to discover, develop, and deliver transformative medicines for people living with psychiatric and neurological conditions, today announced financial results for the fourth quarter and full year ended December 31, 2023, and provided a general business update.

Key Points: 
  • Karuna Therapeutics, Inc. (NASDAQ: KRTX), a biopharmaceutical company driven to discover, develop, and deliver transformative medicines for people living with psychiatric and neurological conditions, today announced financial results for the fourth quarter and full year ended December 31, 2023, and provided a general business update.
  • Results from the EMERGENT-2 trial published in The Lancet in December 2023.
  • Announced positive results from the Phase 1b Ambulatory Blood Pressure Monitoring trial in schizophrenia in the fourth quarter of 2023.
  • Presented additional data from the EMERGENT program at the 2023 Neuroscience Education Institute (NEI) Congress and the CNS Summit in the fourth quarter of 2023.

FDA Grants Priority Review to Merck's Application for KEYTRUDA® (pembrolizumab) Plus Chemotherapy as Treatment for Primary Advanced or Recurrent Endometrial Carcinoma

Retrieved on: 
Tuesday, February 20, 2024
Oncology, FDA, Health, General Health, Pharmaceutical, Biotechnology, MSD, Carboplatin, MRK, Society of Gynecologic Oncology, Food, PDUFA, Risk, Cancer, Gynecologic Oncology (journal), Death, Paclitaxel, SGO, Patient, Pharmaceutical industry

The FDA has set a Prescription Drug User Fee Act (PDUFA), or target action, date of June 21, 2024.

Key Points: 
  • The FDA has set a Prescription Drug User Fee Act (PDUFA), or target action, date of June 21, 2024.
  • The sBLA is based on data from the Phase 3 NRG-GY018 trial.
  • Health authorities in Israel, Canada, Australia, Singapore and Brazil will review this application as part of Project Orbis.
  • In endometrial cancer, Merck is evaluating KEYTRUDA in the first-line setting for advanced or recurrent disease that is dMMR (KEYNOTE-C93/ENGOT-en15/GOG-3064) and in the adjuvant setting (KEYNOTE-B21/ENGOT-en11/GOG-3053).

U.S. Food and Drug Administration (FDA) Accepts Supplemental New Drug Application for KRAZATI® (adagrasib) in Combination with Cetuximab as a Targeted Treatment Option for Patients with Previously Treated KRAS G12C-Mutated Locally Advanced or...

Retrieved on: 
Tuesday, February 20, 2024
Oncology, FDA, Health, Clinical Trials, Pharmaceutical, Biotechnology, BMY, Progression-free survival, Cetuximab, Physician, Food, Shanda, PDUFA, CRC, Bristol Myers Squibb, Safety, Patient, Pharmaceutical industry

The FDA assigned a Prescription Drug User Fee Act (PDUFA) goal date of June 21, 2024.

Key Points: 
  • The FDA assigned a Prescription Drug User Fee Act (PDUFA) goal date of June 21, 2024.
  • “The acceptance of this filing for KRAZATI in combination with cetuximab is a positive step toward providing a potential new option for patients and their physicians.
  • The secondary endpoints for the pooled cohorts included duration of response, progression-free survival, overall survival and safety.
  • The safety profile for KRAZATI plus cetuximab was manageable and consistent with previous reports, and with the known safety profile of each drug individually.

Datopotamab Deruxtecan Biologics License Application Accepted in the U.S. for Patients with Previously Treated Advanced Nonsquamous Non-Small Cell Lung Cancer

Retrieved on: 
Monday, February 19, 2024
Oncology, Health, FDA, Clinical Trials, Pharmaceutical, Biotechnology, NSCLC, Breast cancer, Progression-free survival, PFS, AstraZeneca, Docetaxel, Doctor of Philosophy, MD, News, Food, BLA, PDUFA, Lung, Daiichi Sankyo, ADC, OS, IHC, HER2, Lung cancer, DXD, Patient, Congress, Pharmaceutical industry

Daiichi Sankyo (TSE: 4568) and AstraZeneca’s (LSE/STO/Nasdaq: AZN) Biologics License Application (BLA) for datopotamab deruxtecan (Dato-DXd) has been accepted in the U.S. for the treatment of adult patients with locally advanced or metastatic nonsquamous non-small cell lung cancer (NSCLC) who have received prior systemic therapy.

Key Points: 
  • Daiichi Sankyo (TSE: 4568) and AstraZeneca’s (LSE/STO/Nasdaq: AZN) Biologics License Application (BLA) for datopotamab deruxtecan (Dato-DXd) has been accepted in the U.S. for the treatment of adult patients with locally advanced or metastatic nonsquamous non-small cell lung cancer (NSCLC) who have received prior systemic therapy.
  • Datopotamab deruxtecan is a specifically engineered TROP2 directed DXd antibody drug conjugate (ADC) being jointly developed by Daiichi Sankyo and AstraZeneca.
  • In patients with nonsquamous NSCLC, datopotamab deruxtecan showed a clinically meaningful PFS benefit and a numerically favorable OS trend.
  • Additional regulatory submissions for datopotamab deruxtecan in lung and breast cancer are underway globally.

Servier Receives Regulatory Filing Acceptances from FDA and EMA for Vorasidenib in the Treatment of IDH-Mutant Diffuse Glioma

Retrieved on: 
Wednesday, February 21, 2024
Orbis, Classification, PFS, European Commission, EMA, Traffic barrier, Progression-free survival, Confidence interval, Committee, Enzyme, MAA, Research and development, ASCO, Malignancy, Physician, FDA, PDUFA, Society, Glioma, TGR, Microsoft Access, Safety, INDIGO, Prognosis, Civil service commission, HR, CHMP, SNO, IDH, Patient, Medicine, BIRC, Public, Disease, Diagnosis, Mutation, Development, NDA, Priority review, Pharmaceutical industry

BOSTON and SURESNES, France, Feb. 21, 2024 /PRNewswire/ -- Servier, a global leader in oncology focused on delivering meaningful therapeutic progress for the patients it serves, today announced the FDA filing acceptance and priority review for a New Drug Application (NDA) for vorasidenib, as well as the EMA granting accelerated assessment for the vorasidenib Marketing Authorization Application (MAA). This innovative targeted therapy is an oral, selective, highly brain-penetrant dual inhibitor of mutant isocitrate dehydrogenase 1 and 2 (IDH1/2) enzymes for the treatment of IDH-mutant diffuse glioma. If approved, vorasidenib would become a first-in-class targeted therapy for patients with IDH-mutant gliomas and would mark Servier's sixth approval across IDH-mutant cancers. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of August 20, 2024, while the European Commission approval is anticipated in the second half of 2024.

Key Points: 
  • This innovative targeted therapy is an oral, selective, highly brain-penetrant dual inhibitor of mutant isocitrate dehydrogenase 1 and 2 (IDH1/2) enzymes for the treatment of IDH-mutant diffuse glioma.
  • If approved, vorasidenib would become a first-in-class targeted therapy for patients with IDH-mutant gliomas and would mark Servier's sixth approval across IDH-mutant cancers.
  • "In the realm of glioma treatment, innovation has been stagnant for nearly a quarter-century, posing challenges for patients who, post-surgery, may opt to defer treatment due to concerns around potential toxic side effects.
  • "This promising outcome brings hope to patients grappling with IDH-mutant diffuse gliomas, offering a potential breakthrough for those eagerly awaiting a new therapeutic option."

Citius Pharmaceuticals, Inc. Reports Fiscal First Quarter 2024 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, February 14, 2024
TenX, Shareholder, Catheter, Accounting, Research, FDA, Food, Hemorrhoid, BLA, Calendar, PDUFA, FIRST, G&A, Pharmaceutical industry

CRANFORD, N.J., Feb. 14, 2024 /PRNewswire/ -- Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products today reported business and financial results for the fiscal first quarter 2024 ended December 31, 2023.

Key Points: 
  • "We had a strong first quarter of fiscal 2024 during which we achieved multiple mission critical goals.
  • FIRST QUARTER 2024 Financial Results:
    As of December 31, 2023, the Company had $20.3 million in cash and cash equivalents.
  • R&D expenses were $2.6 million for the first quarter ended December 31, 2023, compared to $3.4 million for the first quarter ended December 31, 2022.
  • G&A expenses were $3.7 million for the first quarter ended December 31, 2023, compared to $2.6 million for the first quarter ended December 31, 2022.

Citius Pharmaceuticals Resubmits the Biologics License Application of LYMPHIR™ (Denileukin Diftitox) for the Treatment of Adults with Relapsed or Refractory Cutaneous T-Cell Lymphoma

Retrieved on: 
Wednesday, February 14, 2024
Symantec Endpoint Protection, FDA, Food, Safety, BLA, CRL, PDUFA, CTCL, Letter, Patient

CRANFORD, N.J., Feb. 13, 2024 /PRNewswire/ -- Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products today announced the resubmission of the Company's Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for LYMPHIR™ (denileukin diftitox), an IL-2-based immunotherapy for the treatment of patients with relapsed or refractory cutaneous T-cell lymphoma (CTCL) after at least one prior systemic therapy.

Key Points: 
  • The resubmission follows ongoing engagement with the FDA resulting from a Complete Response Letter (CRL) received on July 28, 2023.
  • Citius believes it has addressed enhanced product testing and additional manufacturing controls noted in the letter.
  • There were no safety or efficacy issues cited and no additional trials required.
  • Based on Center for Drug Evaluation and Research timelines, FDA acceptance of the completed resubmission package and issuance of a Prescription Drug User Fee Act (PDUFA) date is expected within 30 days of resubmission.