PDUFA

Bristol Myers Squibb Completes Acquisition of PureTech's Founded Entity Karuna Therapeutics for $14 Billion

Retrieved on: 
Monday, March 18, 2024
Mental Health, Health, Neurology, Clinical Trials, Pharmaceutical, Biotechnology, BMY, Psychosis, Bristol Myers Squibb, Citigroup, Growth, Schizophrenia, KarXT, Goldman Sachs, EPS, Patient, Neuroscience, Simpson Thacher & Bartlett, Acquisition, BMS, LLP, PRTC, Therapy, GAAP, Covington & Burling, Psychiatry, Bipolar I disorder, PDUFA, Pharmaceutical industry

The full text of the announcement from Bristol Myers Squibb is as follows:

Key Points: 
  • The full text of the announcement from Bristol Myers Squibb is as follows:
    Bristol Myers Squibb Completes Acquisition of Karuna Therapeutics, Strengthening Neuroscience
    PRINCETON, N.J.-- Bristol Myers Squibb (NYSE: BMY) today announced that it has successfully completed its acquisition of Karuna Therapeutics, Inc. (“Karuna”).
  • With the acquisition's completion, Karuna shares have ceased trading on the Nasdaq Global Select Market and Karuna is now a wholly owned subsidiary of Bristol Myers Squibb (“BMS”).
  • “We are excited to expand our neuroscience portfolio as we welcome Karuna to Bristol Myers Squibb,” said Chris Boerner, Ph.D., Chief Executive Officer, Bristol Myers Squibb.
  • Gordon Dyal & Co. and Citi are serving as financial advisors to Bristol Myers Squibb, and Covington & Burling LLP is serving as legal counsel.

U.S. Food and Drug Administration (FDA) Has Accepted the New Drug Application (NDA) for Ensartinib

Retrieved on: 
Wednesday, March 13, 2024
Oncology, FDA, Health, Clinical Trials, Pharmaceutical, Biotechnology, Brain, NSCLC, New Drug Application, Patient, JAMA Oncology, NDA, Anaplastic lymphoma kinase, Crizotinib, Safety, File, Holding, Food, ALK, PDUFA, Pharmaceutical industry

Xcovery Holdings, Inc. , an oncology focused pharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for ensartinib, an Anaplastic Lymphoma Kinase (ALK) inhibitor for the treatment of adult patients with metastatic ALK-positive non-small cell lung cancer (NSCLC).

Key Points: 
  • Xcovery Holdings, Inc. , an oncology focused pharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for ensartinib, an Anaplastic Lymphoma Kinase (ALK) inhibitor for the treatment of adult patients with metastatic ALK-positive non-small cell lung cancer (NSCLC).
  • The filing is based on the results of the eXalt3, a randomized global phase III study designed to evaluate the efficacy and safety of ensartinib vs crizotinib in the first-line treatment of ALK-positive NSCLC.
  • The FDA granted the application Standard Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of December 28, 2024.
  • In the clinical trials, the drug has demonstrated robust and durable responses in ALK-positive NSCLC patients (both systemically and in the brain), with an extensively studied and well manageable safety profile.

Citius Pharmaceuticals Announces FDA Acceptance of the BLA Resubmission of LYMPHIR™ (Denileukin Diftitox) for the Treatment of Adults with Relapsed or Refractory Cutaneous T-Cell Lymphoma

Retrieved on: 
Monday, March 18, 2024
T-cell lymphoma, PDUFA, Symantec Endpoint Protection, Letter, Disease, Patient, CRL, CTCL, Drug development, Safety, FDA, BLA, Food, Pharmaceutical industry

CRANFORD, N.J., March 18, 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 that the U.S. Food and Drug Administration (FDA) has accepted the resubmission of the Company's Biologics License Application (BLA) 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. The FDA has assigned a PDUFA goal date of August 13, 2024.

Key Points: 
  • "The acceptance of the BLA resubmission reflects the completeness of our response to the enhanced product testing and additional controls highlighted by the FDA in their July 2023 CRL.
  • "We believe there remains a critical unmet need for an additional viable treatment option for patients with relapsed or refractory CTCL as current therapies are non-curative.
  • We look forward to the FDA's decision and the potential benefit LYMPHIR may provide patients with relapsed or refractory CTCL," added Mazur.
  • The resubmission follows dialog with the FDA resulting from a Complete Response Letter (CRL) received on July 28, 2023.

U.S. FDA Oncologic Drugs Advisory Committee recommends CARVYKTI® (ciltacabtagene autoleucel) for the earlier treatment of patients with relapsed or refractory multiple myeloma

Retrieved on: 
Friday, March 15, 2024
Dexamethasone, Type, Health, Survival, Multiple myeloma, Disease, Bortezomib, Oncology, Patient, Committee, EMA, Lenalidomide, European Medicines Agency, Daratumumab, DPD, Society, Johnson & Johnson, ODAC, Safety, ASCO, Food, Hematology, FDA, CHMP, Immunomodulatory imide drug, PVD, Annual general meeting, PDUFA, Pharmaceutical industry

RARITAN, N.J., March 15, 2024  /PRNewswire/ -- Johnson & Johnson announced today that the U.S. Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) recommends CARVYKTI® (ciltacabtagene autoleucel, cilta-cel) for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent (IMiD) and who are refractory to lenalidomide. The committee reviewed survival and safety data from the Phase 3 CARTITUDE-4 study and voted unanimously in favor of CARVYKTI® (11 to 0) finding the risk-benefit assessment of CARVYKTI® for the proposed indication as favorable. A supplemental Biologics License Application (sBLA) supported by the CARTITUDE-4 study is currently under review by the FDA with a Prescription Drug User Fee Act (PDUFA) date of April 5, 2024. 

Key Points: 
  • "We are pleased with the advisory committee's support for CARVYKTI in earlier lines of treatment based on the CARTITUDE-4 data," said Jordan Schecter, M.D., Vice President, Disease Area Leader, Multiple Myeloma, Johnson & Johnson Innovative Medicine.
  • "As a physician and researcher committed to advancing patient care, the potential of CARVYKTI in earlier lines of therapy represents an important therapeutic option for patients with multiple myeloma."
  • The ODAC is convened upon request of the FDA to review and evaluate safety and efficacy data of human drug products for use in the treatment of oncologic diseases.
  • The committee provides non-binding recommendations based on its evaluation; however, final decisions on approval of the drug are made by the FDA.

Akebia Therapeutics to Report Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Monday, March 11, 2024
Chronic kidney disease, Dialysis, Anemia, Patient, Kidney disease, Akebia, Food, Vadadustat, AKBA, PDUFA

CAMBRIDGE, Mass., March 11, 2024 /PRNewswire/ -- Akebia Therapeutics ® , Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease, today announced plans to report fourth quarter and full year 2023 financial results on March 14, 2024.

Key Points: 
  • CAMBRIDGE, Mass., March 11, 2024 /PRNewswire/ -- Akebia Therapeutics ® , Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease, today announced plans to report fourth quarter and full year 2023 financial results on March 14, 2024.
  • Akebia will not host a conference call due to the proximity to the anticipated March 27, 2024 Prescription Drug User Fee Act (PDUFA) target action date for vadadustat, which is under review by the U.S. Food and Drug Administration as a treatment for anemia due to chronic kidney disease in adult patients on dialysis.

Congressional "Minibus" Includes Historic Funding for MDMA-Assisted Therapy Trials

Retrieved on: 
Monday, March 11, 2024
Diagnosis, Congress, Veteran, Federation, Executive, Research, Public policy, NDA, Breakthrough therapy, Safety, Suicide, FDA, PTSD, PDUFA, Pharmaceutical industry

WASHINGTON, March 11, 2024 /PRNewswire/ -- Healing Breakthrough, the leading federal advocacy group for veteran access to MDMA-assisted therapy (MDMA-AT), celebrates the achievement of its FY24 advocacy goal: securing the first-ever Congressional appropriation for large-scale, nationwide trials of MDMA-AT. This effort was led by a courageous coalition of bipartisan legislators who are committed to combating PTSD and bringing the veteran suicide epidemic to an end.

Key Points: 
  • To combat PTSD and Veteran suicide, Congress directs the VA to fund nationwide trials of FDA-designated "Breakthrough Therapies," including MDMA-Assisted Therapy.
  • WASHINGTON, March 11, 2024 /PRNewswire/ -- Healing Breakthrough , the leading federal advocacy group for veteran access to MDMA-assisted therapy (MDMA-AT), celebrates the achievement of its FY24 advocacy goal: securing the first-ever Congressional appropriation for large-scale, nationwide trials of MDMA-AT.
  • This effort was led by a courageous coalition of bipartisan legislators who are committed to combating PTSD and bringing the veteran suicide epidemic to an end.
  • This historic Congressional appropriation marks a shift from decades of philanthropically funded private research to public funding of the VA's continued research and deployment of this FDA-designated "Breakthrough Therapy."

Phathom Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
Thursday, March 7, 2024
Esophagus, Hercules, Pharmacy, Quarter, Research, Heartburn, DUAL, Regulation, Forecasting, FDA, Pain, Table, PDUFA, Vomiting, Inflammation, GERD, Ratio, PPI, GAAP, Business, Webcast, Enzyme inhibitor, Patient, Express Scripts, Helicobacter pylori, Clinical trial, Vonoprazan/amoxicillin, PBM, G&A, Eosinophilic esophagitis, Tablet, NDA, Prescription, Vonoprazan/amoxicillin/clarithromycin, Pharmaceutical industry

Net loss: Net loss for the fourth quarter 2023 was $79.6 million, compared to $55.0 million for fourth quarter 2022.

Key Points: 
  • Net loss: Net loss for the fourth quarter 2023 was $79.6 million, compared to $55.0 million for fourth quarter 2022.
  • Fourth quarter 2023 net loss included a non-cash charge related to stock-based compensation of $24.6 million compared to $6.7 million for fourth quarter 2022.
  • Phathom will host a conference call and webcast to discuss its fourth quarter and full year 2023 financial results and business highlights today, March 7, 2024, at 8:30 a.m.
  • Phathom believes the presentation of these non-GAAP financial measures provides useful information to management and investors regarding Phathom's results of operations.

argenx Delivers on Promise to Transform Patient Expectations in Autoimmunity at American Academy of Neurology 2024 Annual Meeting

Retrieved on: 
Thursday, March 7, 2024
OCS, Neonatal Fc receptor, Autoimmune disease, Continuity, ADAPT, CIDP, Alfa Romeo, FDA, Immunoglobulin therapy, Quality of life, NXT, PDUFA, Euronext, Risk, AAN, Clinical trial, Immunoglobulin G, GMG, Abstract, Efficacy, American Academy, Pharmaceutical industry

“We are opening a new chapter for the VYVGART portfolio,” said Luc Truyen, M.D., Ph.D., Chief Medical Officer at argenx.

Key Points: 
  • “We are opening a new chapter for the VYVGART portfolio,” said Luc Truyen, M.D., Ph.D., Chief Medical Officer at argenx.
  • These positive data from the ADHERE study have been submitted to the FDA for potential approval of VYVGART Hytrulo in CIDP with a PDUFA target action date of June 21, 2024.
  • Achievement of MSE enables significant quality of life improvements: ADAPT/ADAPT+ demonstrate that >40% of patients achieve minimal symptom expression (MSE) across both studies.
  • Patients achieving MSE experience quality of life outcomes comparable to healthy populations, suggesting MSE could be a primary goal of gMG treatment.

Applied Therapeutics Reports Fourth Quarter and Year-end 2023 Financial Results

Retrieved on: 
Wednesday, March 6, 2024
Janus, Sorbitol, Marketing, EMA, Doctor of Philosophy, New Drug Application, MAA, Interim analysis, P-value, FDA, Food, Growth, Treatment, INSPIRE, Priority review, PDUFA, Chairperson, Therapy, CHMP, European Medicines Agency, Patient, Private placement, PRV, NDA, Ageing, Pharmaceutical industry

The FDA also noted that it is planning to hold an advisory committee meeting to discuss the application.

Key Points: 
  • The FDA also noted that it is planning to hold an advisory committee meeting to discuss the application.
  • Govorestat was previously granted Pediatric Rare Disease designation and will qualify for a Priority Review Voucher (PRV) upon approval.
  • The Company expects a decision by the EMA in the fourth quarter of 2024.
  • Full study results will be presented at an upcoming medical conference, along with results of the Diabetic Peripheral Neuropathy sub-study, which are still being analyzed.

Aquestive Therapeutics Reports Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
Tuesday, March 5, 2024
PD, Research, FDA, ATM, Food, Growth, Anaphylaxis, Loss, PDUFA, Attention deficit hyperactivity disorder, Emergency, Safety, Therapy, Allergy, Patent, Diazepam, Patient, Serdexmethylphenidate/dexmethylphenidate, Lennox–Gastaut syndrome, Net, ADHD, Adrenaline, Clutch (eggs), Trial of the century, NDA, Riluzole, Seizure, Pharmaceutical industry

Aquestive received positive feedback in October 2023 from the U.S. Food and Drug Administration (FDA) on the Company's pivotal Phase 3 Pharmacokinetic (PK) clinical protocol for Anaphylm.

Key Points: 
  • Aquestive received positive feedback in October 2023 from the U.S. Food and Drug Administration (FDA) on the Company's pivotal Phase 3 Pharmacokinetic (PK) clinical protocol for Anaphylm.
  • Aquestive commenced dosing in December 2023 in the Phase 3 pivotal PK clinical study of Anaphylm.
  • Non-GAAP adjusted EBITDA loss was $2.8 million in the fourth quarter 2023, compared to a $9.6 million loss in the fourth quarter 2022.
  • During the fourth quarter 2023, the Company accessed capital net proceeds of $3.7 million under its "At-the-Market" (ATM) facility.