PDUFA

Abeona Therapeutics Reports Full Year 2023 Financial Results and Announces Completion of FDA Inspections

Retrieved on: 
Monday, March 18, 2024
Research, AAV, Advisory Committee, Form FDA 483, PLI, Sale, EIR, University, Trial of the century, Rett syndrome, Concha bullosa, Priority review, Investment, IP, Therapy, Safety, REMS, EB, Food, FDA, BLA, RDEB, PDUFA, Pharmaceutical industry

On March 15, 2024, the Company submitted a response to the FDA, outlining already implemented and ongoing steps toward resolution that follow FDA guidance provided during the audit.

Key Points: 
  • On March 15, 2024, the Company submitted a response to the FDA, outlining already implemented and ongoing steps toward resolution that follow FDA guidance provided during the audit.
  • In January 2024, the FDA completed both a Bioresearch Monitoring (BIMO) inspection of Abeona and the Mid-Cycle Meeting regarding the pz-cel BLA.
  • Following completion of the BIMO inspection, the FDA inspector did not issue any observations or FDA Form 483s.
  • In March 2024, Abeona received a written Establishment Inspection Report (EIR), the formal report from the FDA regarding the BIMO inspection.

Journey Medical Corporation Announces U.S. FDA Acceptance of New Drug Application for DFD-29 for the Treatment of Rosacea

Retrieved on: 
Monday, March 18, 2024
Entrepreneurship, Capsule, Patient, Food and Drug Administration, Rosacea, NDA, Red, Minocycline, Erythema, Food, Marketing, FDA, CEA, Physician, IGA, PDUFA, Pharmaceutical industry

The FDA has set a Prescription Drug User Fee Act (“PDUFA”) goal date of November 4, 2024.

Key Points: 
  • The FDA has set a Prescription Drug User Fee Act (“PDUFA”) goal date of November 4, 2024.
  • The Phase 3 clinical trials achieved all co-primary and secondary endpoints, and subjects completed the 16-week treatment with no significant safety issues.
  • DFD-29 demonstrated statistically significant superiority over both the current standard-of-care treatment, Oracea® 40 mg capsules, and placebo for Investigator’s Global Assessment (IGA) treatment success as well as the reduction in the total inflammatory lesion count in both studies.
  • For the secondary endpoint evaluating erythema (redness) associated with rosacea, DFD-29 showed a statistically significant reduction in Clinician’s Erythema Assessment (CEA) compared to placebo in both clinical trials.”

Autolus Therapeutics Reports Full Year 2023 Financial Results and Business Updates

Retrieved on: 
Thursday, March 14, 2024
Nucleus, Bone marrow, BCMA, BioNTech, B-ALL, Patient, ALL, SLE, Engineering, Minocycline, European Medicines Agency, Therapy, Research, Canadian Aviation Regulations, Flow cytometry, ASH, General counsel, Immunotherapy, SVP, EMA, Legal Affairs, Trial of the century, GMP, SME, BLA, Nature Communications, Development, UCL, Biologics license application, Multiple myeloma, MIA, University College London, Senior, TRBC, GD2, ASCO, FDA, CD19, Conference call, Conference, MAA, EHA, ADS, MHRA, T cell, ACS Chemical Biology, PDUFA, Autoimmune disease, Pharmaceutical industry

“For now, we remain fully focused on preparing for a potential obe-cel launch and successfully transitioning Autolus to a commercial stage company.

Key Points: 
  • “For now, we remain fully focused on preparing for a potential obe-cel launch and successfully transitioning Autolus to a commercial stage company.
  • In February 2024, Autolus promoted Dr. Chris Williams to Chief Business Officer and Alex Driggs to Senior Vice President, Legal Affairs and General Counsel.
  • Dr. Leiderman brings extensive transactional and financial expertise, and Mr. Azelby brings more than 30 years of biopharmaceutical leadership and commercial experience to Autolus’ Board.
  • Financial Results for the Year Ended December 31, 2023

Zai Lab Partner Bristol Myers Squibb Announces Pivotal KRYSTAL-12 Confirmatory Trial Evaluating KRAZATI (adagrasib) Meets Primary Endpoint of Progression-Free Survival for Patients with Pretreated KRASG12C-Mutated Locally Advanced or Metastatic...

Retrieved on: 
Monday, April 1, 2024
Oncology, Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Lung cancer, BMY, NMPA, Development, NSCLC, Cetuximab, Progression-free survival, New Drug Application, Patient, Neoplasm, Shanda, NDA, Cancer, Bristol Myers Squibb, Pancreatic cancer, CRC, Safety, PFS, FDA, PDUFA, Pharmaceutical industry

The study remains ongoing to assess the additional key secondary endpoint of overall survival.

Key Points: 
  • The study remains ongoing to assess the additional key secondary endpoint of overall survival.
  • Results of the confirmatory trial showed that KRAZATI demonstrated a statistically significant and clinically meaningful benefit in PFS and ORR compared to standard-of-care chemotherapy as a second-line or later treatment for these patients.
  • KRAZATI had no new safety signals and the safety data was consistent with the known safety profile.
  • Zai Lab thanks the patients and investigators involved in the KRYSTAL-12 clinical trial.

Global Cell Counting Market to Hit $15.72 Billion by 2029, Fueled by Advancements in Cell Biology and Therapy - ResearchAndMarkets.com

Retrieved on: 
Friday, March 29, 2024
Research, Technology, University, Genetics, Artificial Intelligence, Education, Science, Nanotechnology, Biotechnology, Stem Cells, Other Technology, Health, Other Science, Automation, Quality control, Interdisciplinarity, Acute lymphoblastic leukemia, Growth, Prevalence, Application, Novartis, Patient, Conditional sentence, Cell biology, Lung, Concha bullosa, Hospital, Artificial intelligence, National Cancer Institute, AI, Regenerative medicine, Duchenne muscular dystrophy, Therapy, DEB, Miniaturization, Sarepta Therapeutics, CGT, FDA, DMD, Tisagenlecleucel, PDUFA, Pharmaceutical industry

The global cell counting market was valued at US$9.98 billion in 2023 and is expected to reach US$15.72 billion by 2029.

Key Points: 
  • The global cell counting market was valued at US$9.98 billion in 2023 and is expected to reach US$15.72 billion by 2029.
  • Advancements in cell biology, biotechnology, and medical research are driving demand for precise cell counting, especially with the rise of personalized medicine and regenerative therapies.
  • PDUFA dates for therapies could translate into an order book boost for the cell counting players in the market, resulting in market growth.
  • The global cell counting market is consolidated, with top five players holding approximately 70% share of the market.

Datopotamab Deruxtecan Biologics License Application Accepted in the U.S. for Patients with Previously Treated Metastatic HR Positive, HER2 Negative Breast Cancer

Retrieved on: 
Tuesday, April 2, 2024
Oncology, Health, FDA, Clinical Trials, General Health, Pharmaceutical, OS, HER2, IHC, HR, Neutropenia, Congress, DXD, AstraZeneca, NSCLC, Progression-free survival, Patient, Stomatitis, Lung, Doctor of Philosophy, MD, Breast cancer, Anemia, PFS, Fatigue, ADC, Food, BLA, Daiichi Sankyo, PDUFA, Pharmaceutical industry

Datopotamab deruxtecan is a specifically engineered TROP2 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo and being jointly developed by Daiichi Sankyo and AstraZeneca.

Key Points: 
  • Datopotamab deruxtecan is a specifically engineered TROP2 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo and being jointly developed by Daiichi Sankyo and AstraZeneca.
  • The safety profile of datopotamab deruxtecan was consistent with that observed in other ongoing trials with no new safety concerns identified.
  • “The FDA’s acceptance of the BLA brings us closer to providing patients with previously treated HR positive, HER2 negative breast cancer an alternative option to conventional chemotherapy earlier in the metastatic setting,” said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo.
  • Additional regulatory submissions for datopotamab deruxtecan in lung and breast cancer are underway globally.

Bristol Myers Squibb Announces Pivotal KRYSTAL-12 Confirmatory Trial Evaluating KRAZATI (adagrasib) Meets Primary Endpoint of Progression-Free Survival for Patients with Pretreated KRASG12C-Mutated Locally Advanced or Metastatic Non-Small Cell Lung…

Retrieved on: 
Thursday, March 28, 2024
Oncology, FDA, Health, Clinical Trials, Pharmaceutical, Biotechnology, Civil service commission, BMY, NSCLC, Cetuximab, Progression-free survival, Patient, Neoplasm, Shanda, Health care, News, Bristol Myers Squibb, MHRA, Pancreatic cancer, CRC, Safety, European Commission, PFS, PDUFA, Medication, Pharmaceutical industry

The study remains ongoing to assess the additional key secondary endpoint of overall survival.

Key Points: 
  • The study remains ongoing to assess the additional key secondary endpoint of overall survival.
  • Results of the confirmatory trial showed that KRAZATI demonstrated a statistically significant and clinically meaningful benefit in PFS and ORR compared to standard-of-care chemotherapy as a second-line or later treatment for these patients.
  • “Today’s news is an important reinforcement of the power of a targeted therapy for patients with locally advanced or metastatic KRASG12C-mutated lung cancer.
  • Bristol Myers Squibb thanks the patients and investigators involved in the KRYSTAL-12 clinical trial.

Results from Italfarmaco Pivotal Phase 3 EPIDYS Study of Givinostat in Duchenne Muscular Dystrophy (DMD) Published in The Lancet Neurology

Retrieved on: 
Tuesday, March 19, 2024
Science, Biotechnology, Research, Pharmaceutical, Health, Hospitals, Genetics, Clinical Trials, VL, Civil service commission, Givinostat, Neurology, Catholic higher education, Diarrhea, MAA, The Lancet, Magnetic resonance imaging, Safety, Abdominal pain, NDA, Manuscript, Rehabilitation, Lancet Neurology, University, OLE, EMA, Degenerative disease, Doctor of Philosophy, MD, European Medicines Agency, Duchenne muscular dystrophy, European Commission, NSAA, FDA, DMD, Pediatrics, PDUFA, Pharmaceutical industry

Italfarmaco S.p.A. announced today that the full results from the Company’s pivotal phase 3 EPIDYS clinical trial with givinostat in ambulant boys 6 years of age and older with Duchenne muscular dystrophy (DMD) have been published in The Lancet Neurology.

Key Points: 
  • Italfarmaco S.p.A. announced today that the full results from the Company’s pivotal phase 3 EPIDYS clinical trial with givinostat in ambulant boys 6 years of age and older with Duchenne muscular dystrophy (DMD) have been published in The Lancet Neurology.
  • The full manuscript, titled, “Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomized, double-blind, placebo-controlled, phase 3 trial,” is published on The Lancet Neurology website .
  • Participants attended study site visits every 12 weeks for 72 weeks and were evaluated for all endpoints within the study protocol.
  • Upon completion of the 18-month double-blind period, EPIDYS study participants were eligible to enroll in an open-label extension study (OLE study) to receive givinostat on an ongoing basis.

Merck Announces Positive Data on V116, an Investigational, 21-Valent Pneumococcal Conjugate Vaccine Specifically Designed for Adults, Demonstrated Immune Responses in Adults

Retrieved on: 
Tuesday, March 19, 2024
Health, General Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, MSD, Emory University, Antigen, Streptococcus, International Society, HIV, Risk, Global health, Patient, Cohort, Hospital, Merck & Co., Aes, OPA, Epidemiology, Training school (United States), PCV20, Immunoglobulin G, Attenuated vaccine, MRK, Pediatrics, CDC, Vaccination, PPSV23, Community-acquired pneumonia, Food, FDA, PDUFA, Medicine, Vaccine

V116 also elicited higher immune responses than the studied comparators for the serotypes unique to V116 in all STRIDE studies presented at the meeting.

Key Points: 
  • V116 also elicited higher immune responses than the studied comparators for the serotypes unique to V116 in all STRIDE studies presented at the meeting.
  • If approved, V116 would be the first pneumococcal conjugate vaccine specifically designed for adults.
  • Participants were enrolled based on previous pneumococcal vaccination with PPSV23, PCV15, PCV13 (pneumococcal 13-valent conjugate vaccine), PPSV23+PCV13, PCV13+PPSV23 or PCV15+PPSV23, and received either V116, PCV15 or PPSV23.
  • V116 elicited comparable immune responses to the comparator, PCV15+PPSV23, for all 13 shared serotypes and higher immune responses for the eight serotypes covered only by V116.

Bristol Myers Squibb Completes Acquisition of Karuna Therapeutics, Strengthening Neuroscience Portfolio

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

Bristol Myers Squibb (NYSE: BMY) today announced that it has successfully completed its acquisition of Karuna Therapeutics, Inc. (“Karuna”).

Key Points: 
  • 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.
  • Bristol Myers Squibb expects to offset the operational expenses of the transaction through continued disciplined resource allocation, cost efficiencies and portfolio prioritization.