Wiskott–Aldrich syndrome

Key Proteo Submits De Novo Application to FDA for its First Newborn Screening Kit

Retrieved on: 
Friday, March 29, 2024
Disease, WD, XLA, Infant, Patient, Classification, Mortality, Wiskott–Aldrich syndrome, RUO, IVD, Trial of the century, Proteomics, X-linked agammaglobulinemia, WAS, Hypogammaglobulinemia, FDA, DSM-IV codes, Food, Pharmaceutical industry

SEATTLE, March 29, 2024 /PRNewswire/ -- Key Proteo, a pioneering proteomics diagnostics company specializing in the enhanced early detection of rare but treatable genetic disorders, today announced that it has submitted a de novo classification request to the U.S. Food and Drug Administration (FDA) for its first in vitro diagnostic Key Proteo Newborn Screening Kit. The novel proteomics-based screening panel addresses a critical, unmet need to aid in the early identification of four treatable rare genetic disorders that current newborn screening programs do not typically test for, including Wilson Disease (WD), Wiskott-Aldrich Syndrome (WAS), X-linked Agammaglobulinemia (XLA) and Adenosine Deaminase Deficiency (ADA). Early screening has the potential to enable timely intervention that can help change the clinical trajectory and relieve the burden of disease for patients and their families afflicted by these potentially devastating disorders.

Key Points: 
  • The submission of Key Proteo's Newborn Screening Kit 1 comes after screening over 22,000 newborn samples in an ongoing pilot study in Washington state.
  • Subsequently, Key Proteo successfully completed a clinical study that included 3,294 newborn samples analyzed at 3 sites across North America.
  • The in vitro diagnostic Key Proteo Newborn Screening Kit 1 will only be available for commercialization once the FDA has completed its process.
  • Currently, Key Proteo Newborn Screening Kit 1 is available as an RUO offering.

Key Proteo Announces Clinical Laboratory Expansion Plans Including New CLIA-Certified Laboratory

Retrieved on: 
Thursday, March 7, 2024
Wilson's disease, Growth, Wiskott–Aldrich syndrome, Adenosine deaminase, Classification, DSM-IV codes, Research, IVD, Proteomics, Pharmaceutical industry

SEATTLE, March 7, 2024 /PRNewswire/ -- Key Proteo, a pioneering proteomics diagnostics company specializing in the early identification of rare but treatable genetic disorders, announced it has initiated plans to build a new CLIA-certified laboratory as part of its initial commercialization and growth strategy to enhance newborn screening programs. The new larger facility and expanded scope of operations will allow Key Proteo to serve unaddressed newborn screening needs among public health laboratories, reference laboratories, hospital laboratories and other providers associated with neonatal care, while maintaining dedicated lab operations for ongoing clinical research and other collaborative opportunities.

Key Points: 
  • The new larger facility and expanded scope of operations will allow Key Proteo to serve unaddressed newborn screening needs among public health laboratories, reference laboratories, hospital laboratories and other providers associated with neonatal care, while maintaining dedicated lab operations for ongoing clinical research and other collaborative opportunities.
  • Based in Seattle, the new 5,000-square-foot facility will include the addition of a state-of-the art CLIA-certified clinical laboratory alongside Key Proteo's corporate headquarters and dedicated clinical research facilities.
  • Buildout of the lab has commenced, and, upon launch, Key Proteo's first newborn screening panel will target four treatable genetic orders including Wilson's disease, Wiskott-Aldrich syndrome, X-linked a-y-globulinemia and adenosine deaminase (ADA) deficiency.
  • In parallel path, Key Proteo is pursuing FDA De Novo classification of an in vitro diagnostic (IVD) test kit for the same four disorders.

Progress update on pilot for academic and non-profit developers of advanced therapy medicines

Retrieved on: 
Friday, February 9, 2024
European, Wiskott–Aldrich syndrome, Agency, Bone marrow, EMA, Hospital, Marketing, Patient, Medicine, Transplant, Pharmaceutical industry

In December 2021, it was granted eligibility to PRIME, EMA’s scheme to support the development of medicines that target an unmet medical need.

Key Points: 
  • In December 2021, it was granted eligibility to PRIME, EMA’s scheme to support the development of medicines that target an unmet medical need.
  • In a second round which finished in December 2023 a total of 11 candidates had expressed their interest to join the pilot.
  • Non-profit academic developers are a major contributor to the development of ATMPs, but experience has shown that navigating regulatory requirements can be challenging.
  • Upon completion, a report will be published on the lessons learnt from this initiative and the areas for improvement in support to academic developers.

ADMA Biologics Announces FDA Approval for Room Temperature Storage of ASCENIV™ & BIVIGAM®

Retrieved on: 
Tuesday, December 13, 2022
IVIG, Food and Drug Administration, Respiratory syncytial virus, RSV, Protein, PIDD, Temperature, Antibody, Bacteria, Wiskott–Aldrich syndrome, FDA, Marketing, 8-K, Immunity, Hepatitis B virus, Blas, Infection, European Patent Office, ADMA, Plasma, Disease, Immunodeficiency, Virus, Risk, Patient, Pharmaceutical industry, Vaccine, PI

RAMSEY, N.J. and BOCA RATON, Fla., Dec. 13, 2022 (GLOBE NEWSWIRE) -- ADMA Biologics, Inc. (NASDAQ: ADMA) (“ADMA” or the “Company”), an end-to-end commercial biopharmaceutical company dedicated to manufacturing, marketing and developing specialty plasma-derived biologics, today announced the United States Food and Drug Administration’s (“FDA”) approval for its supplemental Biologics License Applications (BLAs) for both ASCENIV and BIVIGAM to now include room temperature (25°C) storage conditions for up to 4 weeks during the first 24 months of the 36-month approved shelf life. The room temperature approval applies to all existing ASCENIV and BIVIGAM lots currently in the commercial supply chain as well as to future production of ASCENIV and BIVIGAM.

Key Points: 
  • The room temperature approval applies to all existing ASCENIV and BIVIGAM lots currently in the commercial supply chain as well as to future production of ASCENIV and BIVIGAM.
  • ADMA Biologics is an end-to-end commercial biopharmaceutical company dedicated to manufacturing, marketing and developing specialty plasma-derived biologics for the treatment of immunodeficient patients at risk for infection and others at risk for certain infectious diseases.
  • ADMA manufactures its immune globulin products at its FDA-licensed plasma fractionation and purification facility located in Boca Raton, Florida.
  • These forward-looking statements also include, but are not limited to, the anticipated benefits and significance of the FDA’s room temperature storage approval.

ADMA Biologics Announces Third Quarter 2022 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, November 9, 2022
Wiskott–Aldrich syndrome, Increase, Transplant, Risk, Sale, Infection, Virus, Company, Conference, San Jose BioCenter, 8-K, Review, Immunity, Forward-looking statement, Plasma, Morgan Stanley, Marketing, RSV, Environment, IVIG, SEC, Immunodeficiency, Collection, U.S. Securities and Exchange Commission, Research, Patient, Private Securities Litigation Reform Act, Protein, PIDD, Hepatitis B virus, Nasdaq, Food and Drug Administration, Antibody, European Patent Office, Bacteria, Growth, FDA, Income, GLOBE, Respiratory syncytial virus, Sex linkage, PIN, Investment, Federation of Clinical Immunology Societies, Publication, Disease, General manager, ADMA, Urinary tract infection, Iron ore, Pharmaceutical industry, Video game, Vaccine, Medical device, Reinsurance, Air cargo, PI

ET

Key Points: 
  • ET
    RAMSEY, N.J. and BOCA RATON, Fla., Nov. 09, 2022 (GLOBE NEWSWIRE) -- ADMA Biologics, Inc. (Nasdaq: ADMA) (ADMA or the Company), an end-to-end commercial biopharmaceutical company dedicated to manufacturing, marketing, and developing specialty plasma-derived biologics, today announced its third quarter 2022 financial results and provided a business update.
  • Third Quarter 2022 and Recent Achievements:
    Significant Revenue Growth: Achieved third quarter 2022 total revenues of $41.1 million, as compared to $20.7 million during the third quarter of 2021, an increase of $20.4 million, or approximately 99%.
  • 2022 & Long-Term Financial Guidance:
    2022 Financial Guidance: Enabled by the strong year-to-date execution, ADMA anticipates total 2022 revenues will reach approximately $145 million.
  • Third Quarter 2022 Financial Results:
    Total revenues for the third quarter ended September 30, 2022 were $41.1 million, as compared to $20.7 million during the third quarter of 2021, an increase of $20.4 million, or approximately 99%.

ADMA Biologics Announces Second Quarter 2022 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, August 10, 2022
Shift, U.S. Securities and Exchange Commission, GLOBE, SEC, Patent, Method, Marketing, Patient, PIN, European Patent Office, Infection, Urinary tract infection, Collection, Disease, Metapneumovirus, Sex linkage, Risk, Private Securities Litigation Reform Act, Environment, Respiratory syncytial virus, Increase, Conference, Immunity, San Jose BioCenter, Forward-looking statement, Growth, Income, General manager, ADMA, 8-K, Lists of diseases, IVIG, Morgan Stanley, Coronavirus, Virus, FDA, Antibody, Immunodeficiency, RSV, PIDD, Immunoglobulin therapy, Plasma, Company, Bacteria, Wiskott–Aldrich syndrome, United States Patent and Trademark Office, Protein, Hepatitis B virus, Food and Drug Administration, Adoption, Video game, Renewable energy, Vaccine, Medical device, Tutoring, PI

ET

Key Points: 
  • ET
    RAMSEY, N.J. and BOCA RATON, Fla., Aug. 10, 2022 (GLOBE NEWSWIRE) -- ADMA Biologics, Inc. (Nasdaq: ADMA) (ADMA or the Company), an end-to-end commercial biopharmaceutical company dedicated to manufacturing, marketing, and developing specialty plasma-derived biologics, today announced its second quarter 2022 financial results and provided a business update.
  • Second Quarter 2022 and Recent Achievements:
    Significant Revenue Growth: Achieved second quarter 2022 total revenues of $33.9 million, as compared to $17.8 million during the second quarter of 2021, an increase of $16.1 million, or approximately 90%.
  • 2022 & Long-Term Financial Guidance:
    2022 Financial Guidance: Enabled by the strong start to the year, ADMA anticipates total 2022 revenues will exceed $130 million.
  • Second Quarter 2022 Financial Results:
    Total revenues for the second quarter ended June 30, 2022 were $33.9 million, as compared to $17.8 million during the second quarter of 2021, an increase of $16.1 million, or approximately 90%.

ADMA Biologics Announces First Quarter 2022 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, May 11, 2022
Wiskott–Aldrich syndrome, ADMA, Disease, Sex linkage, Infection, U.S. Securities and Exchange Commission, Antibody, PIDD, Plasma, Virus, IVIG, Adoption, Private Securities Litigation Reform Act, SEC, Collection, Physician, Income, Marketing, Food and Drug Administration, Respiratory syncytial virus, Growth, GLOBE, Company, General manager, Bacteria, Immunodeficiency, FDA, Hepatitis B virus, Lists of diseases, Protein, RSV, European Patent Office, Risk, ID, Nasdaq, Environment, Patient, Forward-looking statement, Immunity, 8-K, San Jose BioCenter, Morgan Stanley, Video game, Risk management, Pharmaceutical industry, Security (finance), Medical device, Vaccine, PI

RAMSEY, N.J. and BOCA RATON, Fla., May 11, 2022 (GLOBE NEWSWIRE) -- ADMA Biologics, Inc. (Nasdaq: ADMA) (“ADMA” or the “Company”), an end-to-end commercial biopharmaceutical company dedicated to manufacturing, marketing and developing specialty plasma-derived biologics, today announced its first quarter 2022 financial results and provided a business update.  

Key Points: 
  • First Quarter 2022 Total Revenues Were Approximately $29.1 Million, an 81% Increase Over First Quarter 2021
    RAMSEY, N.J. and BOCA RATON, Fla., May 11, 2022 (GLOBE NEWSWIRE) -- ADMA Biologics, Inc. (Nasdaq: ADMA) (ADMA or the Company), an end-to-end commercial biopharmaceutical company dedicated to manufacturing, marketing and developing specialty plasma-derived biologics, today announced its first quarter 2022 financial results and provided a business update.
  • We are very encouraged with our first quarter financial results, particularly when considering some of the non-recurring dynamics that occurred during the quarter, said Brian Lenz, ADMAs Chief Financial Officer, and General Manager, ADMA BioCenters.
  • First Quarter 2022 and Recent Achievements:
    Executed Financially: Achieved first quarter 2022 total revenues of $29.1 million, as compared to $16.0 million during the first quarter of 2021, an increase of $13.1 million, or approximately 81%.
  • 2022 & Long-Term Financial Guidance:
    2022 Financial Guidance: Enabled by the strong start to the year, ADMA increases 2022 total revenue guidance to $130 million or more, upwardly revised from $125 million.

ADMA Biologics Announces FDA Approval of Extended Shelf Life for ASCENIV™ & BIVIGAM® from 24 to 36 Months

Retrieved on: 
Friday, March 25, 2022
IG, Company, Private Securities Litigation Reform Act, Risk, Hepatitis B virus, Virus, Plasma, NASDAQ, Marketing, Forward-looking statement, Respiratory syncytial virus, Food and Drug Administration, Compliance, Caregiver, Patient, Wiskott–Aldrich syndrome, IVIG, Bacteria, Antibody, Immunity, European Patent Office, ADMA, U.S. Securities and Exchange Commission, Immunodeficiency, PIDD, Sex linkage, GLOBE, 8-K, Physician, Infection, RSV, Lists of diseases, FDA, Protein, Disease, Decision-making, Health insurance, Vaccine, PI

RAMSEY, N.J. and BOCA RATON, Fla., March 25, 2022 (GLOBE NEWSWIRE) -- ADMA Biologics, Inc. (NASDAQ: ADMA) (“ADMA”), an end-to-end commercial biopharmaceutical company dedicated to manufacturing, marketing and developing specialty plasma-derived biologics, announced the United States Food and Drug Administration’s (“FDA”) approval to extend the expiration dating from 24 to 36 months for both its ASCENIV and BIVIGAM immune globulin (“IG”) drug product stored at 2-8°C. The expiration date extension applies to all existing ASCENIV and BIVIGAM lots currently in the commercial supply chain as well as to future production of ASCENIV and BIVIGAM in all vial sizes, production scales as well as internal and external fill-finished drug product.

Key Points: 
  • The approval represents an important milestone as it pertains to the culmination of remediation initiatives enacted since ADMA acquired the Boca Raton, FL manufacturing facility in 2017.
  • The new 36-month dating for ASCENIV and BIVIGAM now puts ADMAs IG portfolio on a level playing field with competitor product offerings.
  • The newly approved 36-month dating for ASCENIV and BIVIGAM is immediately effective and product is available to U.S. healthcare providers and patients.
  • ADMA manufactures its immune globulin products at its FDA-licensed plasma fractionation and purification facility located in Boca Raton, Florida.

Protecting People with Cancer from COVID-19: New Recommendations from Cancer Guidelines Organization

Retrieved on: 
Tuesday, January 4, 2022
Immunodeficiency, Caregiver, HCT, Face, Policy, AB, COVID-19, Roswell Park Comprehensive Cancer Center, Hospital, Vaccine, NCCN, Food, RNA transfection, DiGeorge syndrome, Health, Review, Research, Education, Patient, Journal of the National Comprehensive Cancer Network, Vaccination, Stem cell, Cancer, Infection, National Comprehensive Cancer Network, Transplant, Food and Drug Administration, Twitter, Wiskott–Aldrich syndrome, ASH, Society, HIV, Decision-making, Medicine, FDA, Lymphatic system, Pre-exposure prophylaxis, Centers for Disease Control and Prevention, Center, Risk, CAR, Haematopoietic system, Death, CDC, Pharmaceutical industry, Medical device, MD

PLYMOUTH MEETING, Pa., Jan. 4, 2022 /PRNewswire/ -- Today, the National Comprehensive Cancer Network® (NCCN®) published significant updates to the expert consensus recommendations on vaccination and pre-exposure prophylaxis of COVID-19 in people with cancer. The NCCN Advisory Committee on COVID-19 Vaccination and Pre-exposure Prophylaxis meets frequently to review all available research and provide evidence-based best practices for keeping people with cancer as safe as possible during the COVID-19 pandemic. The updated guidance—available at NCCN.org/covid-19—includes information on the preventive use of human monoclonal antibodies in addition to the following principals:

Key Points: 
  • Vaccine delays in patients with cancer should also include those recommended for the general public (e.g.
  • Thankfully, we now have additional tools to help people in active treatment for cancer, solid organ transplant recipients, engineered cellular therapy (e.g.
  • The National Comprehensive Cancer Network ( NCCN ) is a not-for-profit alliance of leading cancer centers devoted to patient care, research, and education.
  • The NCCN Guidelines for Patients provide expert cancer treatment information to inform and empower patients and caregivers, through support from the NCCN Foundation .

Advancements in reproductive medicine available at Shady Grove Fertility (SGF) can reduce the inheritance of genetic diseases like breast cancer

Retrieved on: 
Thursday, October 14, 2021
Genetic testing, K Street, Miss, Wiskott–Aldrich syndrome, Miss America, Breast cancer, Awareness, ASRM, Risk, Cancer, Fertility, Physician, Corporation, Degenerative disease, Child, Population, Infertility, Woman, Insurance, Cystic fibrosis, Education, Miss USA, SGF, Fertility preservation, Disease, Genetic counseling, Patient, National Breast Cancer Coalition, Tay–Sachs disease, IVF, PGT, Allyn, Rose, Directorate of Research and Civil Affairs, Partnership, Hope, Cancer survivor, Sex linkage, Vaccine, Birth control, Animal, Pharmaceutical industry

Individuals and couples with known genetic diseases can turn to Shady Grove Fertility (SGF) to undergo in vitro fertilization (IVF) with preimplantation genetic testing for monogenic/single gene defects(PGT-M) to reduce the risk of passing genetic mutations to future offspring.

Key Points: 
  • Individuals and couples with known genetic diseases can turn to Shady Grove Fertility (SGF) to undergo in vitro fertilization (IVF) with preimplantation genetic testing for monogenic/single gene defects(PGT-M) to reduce the risk of passing genetic mutations to future offspring.
  • Additionally, patients at risk for dominant gene mutations, such as breast cancer or inherited forms of colon cancer, can have individualized testing for these diseases.
  • While Rose is not a carrier for the breast cancer gene, she is a carrier of a rare X-linked genetic mutation called Wiskott-Aldrich syndrome.
  • We are so fortunate to live in a world where we can now reduce the inheritance of deadly genetic diseases via IVF.