Center for Biologics Evaluation and Research

Sarah McGarry, M.D., Senior FDA Official in CDER and CBER, Joins Greenleaf Health

Retrieved on: 
Wednesday, November 8, 2023
FDA, COVID-19, Public Policy, Consulting, Pharmaceutical, Professional Services, Medical Devices, Infectious Diseases, Government, Science, Biotechnology, AIDS, Other Science, Health, Research, Georgetown University School of Medicine, Bioinformatics, CBER, Center for Biologics Evaluation and Research, Policy, Food, Infection, Duke University, SARS-CoV-2, Center for Drug Evaluation and Research, OND, HIV, CDER, Ritonavir, Senior, Marketing, Internal medicine, China Biologic Products, Associate, Food and Drug Administration, Pharmaceutical industry

Greenleaf Health, Inc. (Greenleaf), a leading consulting firm for companies regulated by the Food and Drug Administration (FDA), is pleased to announce that Sarah McGarry, M.D., has joined as Senior Vice President, Drug and Biological Products.

Key Points: 
  • Greenleaf Health, Inc. (Greenleaf), a leading consulting firm for companies regulated by the Food and Drug Administration (FDA), is pleased to announce that Sarah McGarry, M.D., has joined as Senior Vice President, Drug and Biological Products.
  • Throughout her 18-year FDA service, Sarah held multiple leadership, review, and advisory positions within the Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER).
  • Her knowledge of the FDA review process and regulatory policy will enrich the strategic and scientific guidance that Greenleaf provides to clients.
  • from Georgetown University Medical School and completed a residency in internal medicine and a fellowship in infectious diseases at Duke University.

FDA Issues Approvable Letter for SeaStar Medical’s Selective Cytopheretic Device for Pediatric Patients

Retrieved on: 
Monday, October 30, 2023
FDA, Standard of care, Center for Biologics Evaluation and Research, HDE, Humanitarian Device Exemption, Food, SCD, Mortality, MD, Acute kidney injury, Inflammation, AKI, Hepatorenal syndrome, Dialysis, Cardiorenal syndrome, SeaStar, Intensive care medicine, CKRT, Letter, Neutrophil, Hospice and palliative medicine, Monocyte, Sepsis, Patient, Label, Infection, ICU, Pharmaceutical industry, Medical device, Research

As announced in early October 2023, SeaStar Medical expected the FDA to issue this approvable letter within a month of such announcement.

Key Points: 
  • As announced in early October 2023, SeaStar Medical expected the FDA to issue this approvable letter within a month of such announcement.
  • The issuance by the FDA of an Approvable Letter is a standard step in the approval process of a Humanitarian Device Exemption (HDE) application.
  • The Approvable Letter indicates that SeaStar Medical’s HDE application substantially meets the requirements for an Approval Order and outlines remaining administrative steps that must be finalized before the HDE can be active for commercialization.
  • The SCD-PED-01 (weight range ≥15 kg) and PED-02 (weight range ≥10 kg) studies demonstrated 75% and 83% reductions in mortality, respectively.

Immatics Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for ACTengine® IMA203 TCR-T Monotherapy

Retrieved on: 
Tuesday, October 24, 2023
FDA, Uveal melanoma, Regenerative Medicine Advanced Therapy, Center for Biologics Evaluation and Research, FDA Center for Devices and Radiological Health, Ovarian cancer, Disease, PRAME, Synovial sarcoma, Cancer, 21st Century Cures Act, Patient, BLA, Pharmaceutical industry, Research

IMA203 is a TCR-T cell therapy targeting PRAME, a protein frequently expressed in a large variety of solid tumors.

Key Points: 
  • IMA203 is a TCR-T cell therapy targeting PRAME, a protein frequently expressed in a large variety of solid tumors.
  • “The FDA RMAT designation for multiple indications underscores the broad potential of IMA203 and the benefits it may provide for advanced-stage solid tumor patients.
  • This is an important regulatory milestone and a recognition of our clinical development progress for this program,” said Cedrik Britten, Chief Medical Officer of Immatics.
  • As of Sep 30, 2023, the U.S. FDA has received at least 238 requests for RMAT designations and granted 922.

FDA Grants SeaStar Medical’s Selective Cytopheretic Device Breakthrough Device Designation for Hepatorenal Syndrome

Retrieved on: 
Wednesday, October 18, 2023
FDA, Center for Biologics Evaluation and Research, Food, SCD, Mortality, MD, Liver transplantation, Liver, Acute kidney injury, University, AKI, Society, Hepatorenal syndrome, Cardiorenal syndrome, NASH, COVID-19, Fatty liver disease, Immunomodulation, Kidney, Cirrhosis, Hospital, Sepsis, Patient, Food and Drug Administration, Prognosis, ICU, Pharmaceutical industry, Research

This is the third Breakthrough Device Designation granted by the FDA to SeaStar Medical for the SCD device, and is expected to expedite the clinical development and regulatory review of the SCD for use in this patient population.

Key Points: 
  • This is the third Breakthrough Device Designation granted by the FDA to SeaStar Medical for the SCD device, and is expected to expedite the clinical development and regulatory review of the SCD for use in this patient population.
  • An investigator-initiated pilot study conducted at the University of Michigan assessed treatment with the SCD in two patients with type 1 hepatorenal syndrome.
  • “We are grateful for FDA’s decision to award Breakthrough Device Designation to the SCD in hepatorenal syndrome, which follows similar awards for adult AKI in 2022 and for cardiorenal syndrome last month,” said Eric Schlorff, SeaStar Medical CEO.
  • “The SCD now has three Breakthrough Device Designations in total granted by the FDA’s Center for Biologics Evaluation and Research (CBER).

Nuwellis Provides Regulatory Update on SeaStar Medical’s Selective Cytopheretic Device Use in Pediatric Acute Kidney Injury Under a Humanitarian Device Exemption

Retrieved on: 
Tuesday, October 10, 2023
Humanitarian Device Exemption, Language, Food, SCD, Acute kidney injury, AKI, Sepsis, Risk, CKD, Center for Biologics Evaluation and Research, Mortality, Safety, FDA, SeaStar, Chronic kidney disease, CKRT, Marketing, Letter, ICU, Agency, HDE, Physician, Pharmaceutical industry, Nursing, Research

The correspondence indicates that an Approvable Letter is expected to be issued within a month.

Key Points: 
  • The correspondence indicates that an Approvable Letter is expected to be issued within a month.
  • In December of 2022, Nuwellis and SeaStar entered into a U.S. license and distribution agreement for SeaStar Medical’s SCD for pediatric AKI.
  • Under this agreement, Nuwellis will market and distribute the SCD through its direct salesforce to nephologists and intensive care physicians who are trained in pediatric extracorporeal therapy.
  • Children who survive an AKI episode are at risk for long-term conditions, including chronic kidney disease (CKD).1

Rett Syndrome Research Trust's Genetic Medicines Summit Sets the Stage for Accelerated Therapeutic Development

Retrieved on: 
Wednesday, October 11, 2023
Office of Rare Diseases Research, Food, Doctor of Philosophy, Biomarker, National Center for Advancing Translational Sciences, RSRT, Critical Path Institute, RNA, Harvard University, MD, DNA, Therapy, Summit, Center for Biologics Evaluation and Research, MPH, Roger Hawkins (drummer), IND, MacNamara, JD, Biotechnology, P.C, Rare, United States Department of the Army, MECP2, EEG, FDA, Broad Institute, Hyman, MIT, RNA editing, Clinical trial, Wuhan Institute of Biological Products, Bangladesh Technical Education Board, Regulation of tobacco by the U.S. Food and Drug Administration, COA, Rett syndrome, Pharmaceutical industry, Medical imaging, Rett, Research

TRUMBULL, Conn., Oct. 11, 2023 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust (RSRT) is delighted to announce the successful conclusion of the inaugural Rett Syndrome Genetic Medicines Summit, held September 13 – 15 in Boston, Massachusetts. This landmark event gathered distinguished scientists, clinicians, regulatory experts, and over 40 leading biopharma companies. The goal of the Summit was to foster dialogue among scientific, clinical, industry, and regulatory stakeholders to facilitate collaboration, identify best practices, and refine expectations to expedite pre-clinical and clinical development programs focused on genetic medicines for children and adults with Rett syndrome.

Key Points: 
  • The Rett Syndrome Research Trust (RSRT) is delighted to announce the successful conclusion of the inaugural Rett Syndrome Genetic Medicines Summit, held September 13 – 15 in Boston, Massachusetts.
  • TRUMBULL, Conn., Oct. 11, 2023 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust (RSRT) is delighted to announce the successful conclusion of the inaugural Rett Syndrome Genetic Medicines Summit, held September 13 – 15 in Boston, Massachusetts.
  • RSRT's Rett Syndrome Genetic Medicines Summit underscores the organization's unwavering commitment to curing Rett syndrome.
  • RSRT remains steadfast in its mission to accelerate the development of genetic therapies and ultimately find a cure for Rett syndrome.

Novavax 2023-2024 COVID-19 Vaccine Now Authorized and Recommended for Use in the U.S.

Retrieved on: 
Tuesday, October 3, 2023
EUA, Heart, Pregnancy, Pharmacy, Caregiver, Tell, Arthralgia, Vaccination, Chills, Rite Aid, Infection, Fever, COVID-19, Center for Biologics Evaluation and Research, Coronavirus, Skin, Drug testing welfare recipients, Woman, COVID, Inflammation, Fact sheet, CVS Pharmacy, Government, CDC, Myalgia, Vomiting, Formula, Allergy, Physician, Child, XBB, Korea Disease Control and Prevention Agency, Immune system, USE, VAERS, Nausea, NVAX, European Medicines Agency, Ageing, FDA, Headache, Death, Regulation of tobacco by the U.S. Food and Drug Administration, FD, Food, World Health Organization, Fatigue, Pericarditis, Vaccine, Pharmaceutical industry, Birth control, Medical device, Mobile phone, Novavax COVID-19 vaccine, Hypoesthesia, Adjuvant, Novavax

In the coming days, individuals in the U.S. can go to pharmacies, physicians' offices, clinics and various government entities to receive an updated Novavax vaccine."

Key Points: 
  • In the coming days, individuals in the U.S. can go to pharmacies, physicians' offices, clinics and various government entities to receive an updated Novavax vaccine."
  • What should you mention to your vaccination provider before you or your child get the Novavax COVID-19 Vaccine, Adjuvanted?
  • A person should not get the Novavax COVID-19 Vaccine, Adjuvanted if they had:
    a severe allergic reaction after a previous dose of any Novavax COVID-19 Vaccine, Adjuvanted
    What are the risks of the Novavax COVID-19 Vaccine, Adjuvanted?
  • Women who are vaccinated with the Novavax COVID-19 Vaccine, Adjuvanted during pregnancy are encouraged to enroll in the registry by visiting https://c-viper.pregistry.com .

SeaStar Medical Provides Regulatory Update Regarding Selective Cytopheretic Device Use in Pediatric Acute Kidney Injury Under a Humanitarian Device Exemption

Retrieved on: 
Tuesday, October 3, 2023
Letter, Chronic kidney disease, Neutrophil, Marketing, Infection, HDE, SCD, Center for Biologics Evaluation and Research, Language, AKI, Sepsis, Acute kidney injury, CKRT, Monocyte, Patient, Agency, Risk, Humanitarian Device Exemption, Mortality, ICU, Septic, FDA, Food, Safety, Pharmaceutical industry, Research

DENVER, Oct. 03, 2023 (GLOBE NEWSWIRE) -- SeaStar Medical Holding Corporation (Nasdaq: ICU), a medical device company developing proprietary solutions to reduce the consequences of dysregulated immune responses including hyperinflammation on vital organs, announces receipt of a correspondence from the U.S. Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) indicating that the Agency considers the Selective Cytopheretic Device (SCD) Pediatric (SCD-PED) to be approvable under a Humanitarian Device Exemption (HDE) for use in children weighing 10 kilograms or more with acute kidney injury (AKI) and sepsis or a septic condition requiring continuous kidney replacement therapy (CKRT) in the hospital intensive care unit (ICU).

Key Points: 
  • The correspondence further indicates that an Approvable Letter, which is a standard part of CBER’s approval process, is expected to be issued within a month.
  • The Approvable Letter will outline conditions, including language for safety, probable benefit and labeling for intended use, which will be required for formal marketing approval.
  • “The correspondence provides a clear path forward in making this much needed therapy accessible to critically ill children suffering with AKI and sepsis,” said Eric Schlorff, SeaStar Medical CEO.
  • The SCD-PED-01 (weight range ≥15 kg) and PED-02 (weight range ≥10 kg) studies demonstrated 75% and 83% reductions in mortality, respectively.

Novavax 2023-2024 COVID-19 Vaccine Now Authorized and Recommended for Use in the U.S.

Retrieved on: 
Tuesday, October 3, 2023
EUA, Heart, Pregnancy, Pharmacy, Caregiver, Tell, Arthralgia, Vaccination, Chills, Rite Aid, Infection, Fever, COVID-19, Center for Biologics Evaluation and Research, Coronavirus, Skin, Drug testing welfare recipients, Woman, COVID, Inflammation, Fact sheet, CVS Pharmacy, Government, CDC, Myalgia, Vomiting, Formula, Allergy, Physician, Child, XBB, Korea Disease Control and Prevention Agency, Immune system, USE, VAERS, Nausea, NVAX, European Medicines Agency, Ageing, FDA, Headache, Death, Regulation of tobacco by the U.S. Food and Drug Administration, FD, Food, World Health Organization, Fatigue, Pericarditis, Vaccine, Pharmaceutical industry, Birth control, Medical device, Mobile phone, Novavax COVID-19 vaccine, Hypoesthesia, Adjuvant, Novavax

In the coming days, individuals in the U.S. can go to pharmacies, physicians' offices, clinics and various government entities to receive an updated Novavax vaccine."

Key Points: 
  • In the coming days, individuals in the U.S. can go to pharmacies, physicians' offices, clinics and various government entities to receive an updated Novavax vaccine."
  • What should you mention to your vaccination provider before you or your child get the Novavax COVID-19 Vaccine, Adjuvanted?
  • A person should not get the Novavax COVID-19 Vaccine, Adjuvanted if they had:
    a severe allergic reaction after a previous dose of any Novavax COVID-19 Vaccine, Adjuvanted
    What are the risks of the Novavax COVID-19 Vaccine, Adjuvanted?
  • Women who are vaccinated with the Novavax COVID-19 Vaccine, Adjuvanted during pregnancy are encouraged to enroll in the registry by visiting https://c-viper.pregistry.com .

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.