Biologics license application

BriaCell Initiates Pivotal Phase 3 Study of Bria-IMT™ in Advanced Metastatic Breast Cancer

Retrieved on: 
Tuesday, October 3, 2023
Biologics license application, Communication, Institutional review board, Survival, Immunotherapy, Patient, IRB, TSX, FDA, Therapy, BCT, Safety, Disease, Pharmaceutical industry

Positive completion of the pivotal study, following review by FDA, could lead to full approval of the Bria-IMT™ immune checkpoint inhibitor combination in advanced metastatic breast cancer.

Key Points: 
  • Positive completion of the pivotal study, following review by FDA, could lead to full approval of the Bria-IMT™ immune checkpoint inhibitor combination in advanced metastatic breast cancer.
  • The study will enroll 177 patients in the Bria-IMT™ combination therapy arm and 177 patients in the treatment of physician’s choice arm.
  • We expect frequent and responsive FDA communication under our Fast Track status during our pivotal Phase 3 study.
  • The successful completion of the pivotal study would allow BriaCell to subsequently submit a Biologics License Application and accelerate the path to commercialization.

Ipsen updates on QM-1114 regulatory process

Retrieved on: 
Tuesday, October 3, 2023
IPN, Intellectual property, Partnership, International, Arbitral tribunal, ADR, Symantec Endpoint Protection, Galderma, CMC, Biologics license application, ICC, Tribunal, FDA, Commerce, Practice of law, Ipsen, Euronext

Furthermore, on 28 September 2023, the Arbitral Tribunal of the International Chamber of Commerce (ICC) issued a final decision on arbitration proceedings that Galderma initiated against Ipsen.

Key Points: 
  • Furthermore, on 28 September 2023, the Arbitral Tribunal of the International Chamber of Commerce (ICC) issued a final decision on arbitration proceedings that Galderma initiated against Ipsen.
  • The result of this arbitration is that any regulatory applications for QM-1114 in the partnership territories submitted by Galderma shall be assigned to Ipsen as the owner of the intellectual property and marketing authorization of QM-1114.
  • Galderma remains responsible for development, regulatory filing strategy, manufacturing and commercialization.
  • As such, the Tribunal declared that Galderma has the right to decide on QM-1114’s regulatory strategy.

Avenge Bio Receives FDA Fast Track Designation for AVB-001, a Novel Cell Therapy Leveraging the LOCOcyte™ Immunotherapy Platform

Retrieved on: 
Monday, October 2, 2023
Adenocarcinoma, Biologics license application, Ovary, Biotechnology, Fast Track, Fast track (FDA), The Company, Patient, Trial of the century, FDA, Ovarian cancer, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BLA, Food, Safety, Pharmaceutical industry

"We are extremely pleased to receive the FDA Fast Track designation for AVB-001 based on FDA's review of our preclinical and emerging clinical data.

Key Points: 
  • "We are extremely pleased to receive the FDA Fast Track designation for AVB-001 based on FDA's review of our preclinical and emerging clinical data.
  • The Fast Track designation has been provided for platinum-resistant, refractory ovarian cancer, and acknowledges the potential for AVB-001 to treat this significant unmet medical need," said Michael Heffernan, CEO at Avenge Bio.
  • The Fast Track designation is available not only where treatments do not exist, but also for drugs that demonstrate a potential advantage over available therapies.
  • Once granted Fast Track designation, the FDA increases the frequency of meetings to discuss the development, trial design, clinical data, and submission of the Biologics License Application (BLA).

Rocket Pharmaceuticals Announces FDA Acceptance of Biologics License Application with Priority Review for RP-L201 (marnetegragene autotemcel) for the Treatment of Severe Leukocyte Adhesion Deficiency-I (LAD-I)

Retrieved on: 
Monday, October 2, 2023
Science, Biotechnology, Research, Pharmaceutical, Health, FDA, Genetics, Clinical Trials, Biologics license application, Priority review, University, Pediatrics, Molecule, Pharming, Infection, Survival, Skin, MBA, University of California, Disorder, Medication, Șaeș, Molecular genetics, Patient, Cell, Disease, PDUFA, Mortality, Immunology, Principal, UCLA, Pharmacology, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BLA, Food, Caregiver, Pharmaceutical industry, Microbiology

“Today’s acceptance of the BLA by the FDA marks a significant milestone for Rocket towards our goal of delivering a one-time gene therapy to patients facing the devastating effects of severe LAD-I.

Key Points: 
  • “Today’s acceptance of the BLA by the FDA marks a significant milestone for Rocket towards our goal of delivering a one-time gene therapy to patients facing the devastating effects of severe LAD-I.
  • All primary and secondary endpoints were met, and RP-L201 was very well tolerated in all patients with no treatment related SAEs.
  • “As the Principal Investigator in the U.S., I oversaw treatment of six of the nine LAD-I patients in this trial.
  • All of these children have been in good health with no significant LAD-I-related infections or inflammatory skin lesions since treatment.

FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies

Retrieved on: 
Friday, September 29, 2023
Well, China Biologic Products, CDER, Communication, Drug development, START, Life, Register, CBER, Marketing, Center for Drug Evaluation and Research, New Drug Application, Disability, Center for Biologics Evaluation and Research, ARC, Rare, Adequate Yearly Progress, Federal, Biologics license application, Request for information, Investigational New Drug, Federal Register, Patient, Learning, Effectiveness, FDA, Death, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IND, Support, Education, Food, Safety, Disease, Pharmaceutical industry, Medical device, Research

"These are complex products and we recognize the importance of sponsor communication with the FDA to facilitate development of products for patients with unmet medical needs."

Key Points: 
  • "These are complex products and we recognize the importance of sponsor communication with the FDA to facilitate development of products for patients with unmet medical needs."
  • The FDA has undertaken additional efforts to further enhance and expedite the availability of therapies intended to treat rare diseases.
  • The agency additionally published a docket for stakeholder feedback as part of the Learning and Education to Advance and Empower Rare Disease Drug Developers (LEADER 3D) program under the CDER Accelerating Rare disease Cures (ARC) program .
  • Feedback will be used for the identification of knowledge gaps in rare disease drug development and the development of publicly available resources to inform stakeholders who design and conduct rare disease drug development programs on regulatory considerations surrounding clinical trial design.

Odronextamab BLA for Treatment of Relapsed/Refractory Follicular Lymphoma (FL) and Diffuse Large B-cell Lymphoma (DLBCL) Accepted for FDA Priority Review

Retrieved on: 
Friday, September 29, 2023
Biologics license application, Hematology, CD20, DLBCL, European Medicines Agency, Bone marrow, Marketing, Regeneron Pharmaceuticals, Patient, Priority, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BLA, Society, Food, Safety, Pharmaceutical industry

TARRYTOWN, N.Y., Sept. 29, 2023 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for odronextamab to treat adult patients with relapsed/refractory (R/R) follicular lymphoma (FL) or R/R diffuse large B-cell lymphoma (DLBCL), who have progressed after at least two prior systemic therapies. The target action date for the FDA decision is March 31, 2024. Odronextamab is an investigational CD20xCD3 bispecific antibody designed to bridge CD20 on cancer cells with CD3-expressing T cells to facilitate local T-cell activation and cancer-cell killing.

Key Points: 
  • Odronextamab is an investigational CD20xCD3 bispecific antibody designed to bridge CD20 on cancer cells with CD3-expressing T cells to facilitate local T-cell activation and cancer-cell killing.
  • The BLA was supported by data from a Phase 1 and pivotal Phase 2 trial (ELM-1 and ELM-2).
  • The FDA previously granted odronextamab Orphan Drug Designation and Fast Track Designation for FL and DLBCL.
  • Odronextamab is currently under clinical development, and its safety and efficacy have not been fully evaluated by any regulatory authority.

Outlook Therapeutics® Requests Type A Meeting With FDA

Retrieved on: 
Friday, September 29, 2023
Retina, Biologics license application, Bevacizumab, Symantec Endpoint Protection, AMD, CMC, Patient, Agency, CRL, FDA, Therapy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BLA, Food, Safety, Pharmaceutical industry

In the FDA’s recently issued CRL, although the Agency acknowledged the NORSE TWO pivotal trial met its safety and efficacy endpoints, it concluded it could not approve the BLA during this review cycle due to several CMC issues, open observations from pre-approval manufacturing inspections, and a lack of substantial evidence.

Key Points: 
  • In the FDA’s recently issued CRL, although the Agency acknowledged the NORSE TWO pivotal trial met its safety and efficacy endpoints, it concluded it could not approve the BLA during this review cycle due to several CMC issues, open observations from pre-approval manufacturing inspections, and a lack of substantial evidence.
  • The Company is committed to working with the FDA to address its concerns.
  • “Our belief remains unwavering that the retina community needs an FDA-approved ophthalmic bevacizumab to deliver an alternative on-label bevacizumab option for patients with wet AMD.
  • We look forward to a productive meeting with FDA to discuss the CRL with the goal of obtaining a clear understanding of the items that need to be addressed to resubmit our BLA for ONS-5010 at the earliest opportunity,” said Russell Trenary, President and CEO of Outlook Therapeutics.

BrainStorm Cell Therapeutics Provides Update on FDA Advisory Committee Meeting to Review NurOwn for the Treatment of ALS

Retrieved on: 
Thursday, September 28, 2023
Biologics license application, Therapy, Gene, Brainstorm, Advisory Committee, Research, Doctor of Philosophy, Committee, Regulation of tobacco by the U.S. Food and Drug Administration, ALS, BLA, Food, Pharmaceutical industry

Today the Committee voted that NurOwn did not demonstrate substantial evidence of effectiveness for treatment of mild to moderate ALS.

Key Points: 
  • Today the Committee voted that NurOwn did not demonstrate substantial evidence of effectiveness for treatment of mild to moderate ALS.
  • "The Committee's vote was a sad outcome for the ALS community, who have too few options to help manage this merciless and deadly disease," said Stacy Lindborg, PhD, co-CEO of BrainStorm.
  • We truly did our best to make the NurOwn data clear to the FDA Advisory Committee.
  • Unfortunately, had more time and opportunity been allowed, many remaining questions posed by Advisory Committee members could have been sufficiently addressed."

U.S. FDA Approves Subcutaneous Administration of Takeda's ENTYVIO® (vedolizumab) for Maintenance Therapy in Moderately to Severely Active Ulcerative Colitis

Retrieved on: 
Wednesday, September 27, 2023
Research, Nursing, FDA, Clinical Trials, Practice Management, Biotechnology, Health, Pharmaceutical, Science, Biologics license application, Mayo, Maintenance, TNF, Icahn School of Medicine at Mount Sinai, Week, SC, TAK, Head, Mount Sinai, Ulcerative colitis, Azathioprine, UC, Patient, Physician, Gastroenterology, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Safety, Disease, Pharmaceutical industry, Takeda

Additionally, a Biologics License Application for an investigational SC administration of ENTYVIO for the treatment of adults with moderately to severely active Crohn’s disease is currently under review by the FDA.

Key Points: 
  • Additionally, a Biologics License Application for an investigational SC administration of ENTYVIO for the treatment of adults with moderately to severely active Crohn’s disease is currently under review by the FDA.
  • “With the FDA approval of subcutaneous ENTYVIO, patients and physicians who want ENTYVIO’s clinical profile along with flexibility of administration now have two choices for maintenance treatment for adults with moderate to severe ulcerative colitis,” said Brandon Monk, senior vice president, head, U.S. Gastroenterology Business Unit, Takeda.
  • ENTYVIO is the only FDA-approved biologic for maintenance therapy in ulcerative colitis offering the option of either intravenous or subcutaneous administration.”
    The approval of this new route of administration for ENTYVIO is based on the VISIBLE 1 study (SC UC Trial).
  • “The VISIBLE 1 trial demonstrated that ENTYVIO SC can provide physicians with an additional administration option for achieving remission in their moderate to severe ulcerative colitis patients.

Pancreatic Cancer Research Gains Momentum Amidst Soaring Young-Onset Cases

Retrieved on: 
Tuesday, September 26, 2023
Gastrointestinal cancer, MDT, Breakthrough therapy, MBA, GH, Congress, Incidence, Helmy Eltoukhy, Patient, JAMA Network Open, Health, NYSE, Polyp, News, Disease, Oncolytics Biotech, Research, Attention, Biologics license application, MRD, ONC, Pancreatic Cancer Action Network, Data, JD, TSX, FDA, Lancet, GI, Pancreatic cancer, Human, CAD, NSCLC, Biomarker, NRG1, Colorectal cancer, CR, United States Congress, ORR, Clinical trial, FOLFIRINOX, ASCO, American Society of Clinical Oncology, Physician, EUS, Annual general meeting, Poster, Recurrence, Society, FTD, Breast cancer, Pharmaceutical industry, Medical imaging, Meru Networks, Nvidia, Medtronic

VANCOUVER, British Columbia, Sept. 26, 2023 /PRNewswire/ -- USA News Group - The seriousness of pancreatic cancer continues to grow, with increasing alarm since The Lancet published an analysis back in April 2023, showing a rising incidence of pancreatic cancer among individuals younger than 55 years. Another study published in JAMA Network Open showed that early-onset cancer in people younger than 50 rose over the last decade, with gastrointestinal cancers seeing the fastest increase. This has led to increased calls from pancreatic cancer organizations such as Pancreatic Cancer Action Network (PanCAN) for more action in the space, including advocating that the US Congress invest more in cancer research funding now. Among the ongoing efforts that are drawing more attention in the pancreatic cancer space have been the developments coming from Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), Merus N.V. (NASDAQ: MRUS), Guardant Health, Inc. (NASDAQ: GH), Medtronic plc (NYSE: MDT), and even NVIDIA Corporation (NASDAQ: NVDA).

Key Points: 
  • This has led to increased calls from pancreatic cancer organizations such as Pancreatic Cancer Action Network (PanCAN) for more action in the space, including advocating that the US Congress invest more in cancer research funding now.
  • "By teaming up with PanCAN, we believe we may be able to expedite development and provide pancreatic cancer patients with a bespoke immunotherapeutic treatment option."
  • Pelareorep delivered the company a big win near the end of 2022, when the FDA rewarded it with a Fast Track Designation (FTD) for treating advanced or metastatic pancreatic cancer.
  • "With a five-year survival rate of 12%, pancreatic cancer patients cannot afford to wait for new treatment options," said Julie Fleshman, JD, MBA, President and CEO of PanCAN.