Hypogammaglobulinemia

X4 Pharmaceuticals Reports First-Quarter 2024 Financial Results and Provides Corporate Updates

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星期二, 五月 7, 2024
Birth, FDA, Capsule, Myelokathexis, Research and development, Priority review, Safety, WHIM syndrome, Food, Security (finance), Conference call, WHIM, Research, Conference, Wart, Neutrophil, CIS, Patient, Society, EMA, Hypogammaglobulinemia, Infection, G-CSF, Interim, Immune system, Lymphocyte, Clinical trial, ID, European Medicines Agency, Pharmaceutical industry

BOSTON, May 07, 2024 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today reported financial results for the first quarter ended March 31, 2024 and highlighted key recent and upcoming expected milestones.

Key Points: 
  • ET
    BOSTON, May 07, 2024 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today reported financial results for the first quarter ended March 31, 2024 and highlighted key recent and upcoming expected milestones.
  • “Concurrent with this approval, we received a Rare Pediatric Disease Priority Review Voucher, which we intend to monetize.
  • X4 currently expects to seek approval from the European Medicines Agency (EMA) for mavorixafor in WHIM syndrome in late 2024/early 2025.
  • The live webcast will be accessible through the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com.

PANTHERx® Rare Partnering with X4 Pharmaceuticals Inc. for the Distribution of XOLREMDI™ (mavorixafor), the First FDA-Approved Therapy Indicated for Use in Patients with WHIM Syndrome

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星期一, 四月 29, 2024
FDA, Bone marrow, Myelokathexis, CXCR4, WHIM syndrome, Food, WHIM, Pharmacy, Wart, CXCL12, Patient, Hypogammaglobulinemia, Primary immunodeficiency, Infection, Pharmaceutical industry

WHIM syndrome is a rare, combined primary immunodeficiency and chronic neutropenic disorder caused by reduced mobilization of white blood cells into the peripheral circulation due to CXCR4/CXCL12 pathway dysfunction.

Key Points: 
  • WHIM syndrome is a rare, combined primary immunodeficiency and chronic neutropenic disorder caused by reduced mobilization of white blood cells into the peripheral circulation due to CXCR4/CXCL12 pathway dysfunction.
  • By targeting the underlying cause of WHIM syndrome, treatment with XOLREMDI results in the increased mobilization of immune cells from the bone marrow.
  • "We are very pleased to be working with X4 Pharmaceuticals for the distribution of XOLREMDI in the U.S.," said Rob Snyder, CEO of PANTHERx Rare Pharmacy.
  • "Up until now, there has been no treatment specifically indicated in people with WHIM syndrome.

Orphan designation: Mavorixafor Treatment of WHIM syndrome, 25/07/2019 Positive

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星期二, 四月 9, 2024
Eurostat, Diagnosis, Civil service commission, WHIM, WHIM syndrome, Myelokathexis, Pneumonia, Bone marrow, CXCR4, Risk, Patient, Committee, Knowledge, Hypogammaglobulinemia, Antibody, Regulation, Viral, EMA, IRIS, Wart, Otitis, Protein, Lymphocyte, European, Infection, COMP, Immune system, European Commission, Neutrophil, Blood, Safety, Consultant, Marketing, Movement, Pharmaceutical industry

Orphan designation: Mavorixafor Treatment of WHIM syndrome, 25/07/2019 Positive

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Orphan designation: Mavorixafor Treatment of WHIM syndrome, 25/07/2019 Positive

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

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星期五, 三月 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.

Analyses of Kite’s Yescarta® CAR T-Cell Therapy Support Curative Potential in Patients With Non-Hodgkin Lymphomas

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星期二, 十二月 12, 2023
Oncology, Health, Hospitals, Clinical Trials, Pharmaceutical, Biotechnology, Arm, Safety, Senior, Hope, Hypogammaglobulinemia, Ageing, ASH, University, EFS, B-cell lymphoma, Patient, Abstract, PFS, DOR, Follicular lymphoma, Lymphoma, DSS, Subgroup analysis, Standard of care, ORR, MD, Survival, Death, HR, Doctor of Philosophy, MD Anderson Cancer Center, Cancer, SOC, OS, Knowledge, Society, Confidence interval, Pharmaceutical industry, Axicabtagene ciloleucel

In this post hoc analysis of ZUMA-1, with up to six years of follow-up, the five-year long-term lymphoma-related event-free survival (LREFS) was used as a measure to explore Yescarta’s curative potential.

Key Points: 
  • In this post hoc analysis of ZUMA-1, with up to six years of follow-up, the five-year long-term lymphoma-related event-free survival (LREFS) was used as a measure to explore Yescarta’s curative potential.
  • Yescarta had long-term LREFS in a substantial proportion of patients, with a five-year rate of 34% (57% among patients who achieved a CR).
  • Among patients who had a CR to therapy, the leading risks of death were reasons other than progression or adverse events after month 24 post-infusion.
  • “We are encouraged by these data, particularly the continued durable response and long-term survival in these patient populations that speak to the potentially curative benefit of this therapy.

X4 Pharmaceuticals Reports Third-Quarter 2023 Financial Results and Provides Corporate Updates

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星期四, 十一月 9, 2023
Priority review, FDA, Ageing, PDUFA, Addition, Rare, Physician, Christophe, SG, MD, Conference, Organization, Doctor of Philosophy, PRV, Primary immunodeficiency, R, Society, Neutropenia, ASH, Wart, WHIM, Disease, ID, WHIM syndrome, Immune system, Research and development, Line, File, Security (finance), New Drug Application, NDA, Safety, Conference call, Patient, Diagnosis, State Administrative Expenses, Pharmaceutical industry, Rare-earth element, Hypogammaglobulinemia, Infection, Myelokathexis

BOSTON, Nov. 09, 2023 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today reported financial results for the third quarter ended September 30, 2023 and highlighted key recent and upcoming expected milestones.

Key Points: 
  • ET
    BOSTON, Nov. 09, 2023 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today reported financial results for the third quarter ended September 30, 2023 and highlighted key recent and upcoming expected milestones.
  • The FDA has notified X4 that it does not currently plan to hold an advisory committee meeting to review the filing.
  • Cash, Cash Equivalents, Restricted Cash, and Short-Term Marketable Securities: X4 had $142.7 million in cash, cash equivalents, restricted cash, and marketable securities as of September 30, 2023.
  • The live webcast will be accessible through the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com .

X4 Pharmaceuticals Announces FDA Acceptance with Priority Review of U.S. NDA for Mavorixafor in WHIM Syndrome

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星期二, 十月 31, 2023
Priority review, FDA, Ageing, PDUFA, Orphan, Rare, RPD, CXCR4, PRV, Primary immunodeficiency, Wart, WHIM, WHIM syndrome, Immune system, New Drug Application, NDA, Pharmaceutical industry, Hypogammaglobulinemia, Infection, Myelokathexis

BOSTON, Oct. 31, 2023 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced that the United States Food and Drug Administration (FDA) has accepted for filing the company’s New Drug Application (NDA) for once-daily, oral mavorixafor to treat individuals aged 12 and older with WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome, a rare, primary immunodeficiency. The FDA granted Priority Review of the mavorixafor NDA, establishing a goal of six months review from the date of acceptance and assigning a Prescription Drug User Fee Act (PDUFA) target action date of April 30, 2024.

Key Points: 
  • The FDA granted Priority Review of the mavorixafor NDA, establishing a goal of six months review from the date of acceptance and assigning a Prescription Drug User Fee Act (PDUFA) target action date of April 30, 2024.
  • “The FDA’s acceptance of our mavorixafor NDA with priority review represents yet another significant step forward towards a potential treatment for those with WHIM syndrome, a rare disease for which there are currently no approved therapies,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals.
  • Upon FDA approval of a product with RPD designation, the sponsor can receive a Priority Review Voucher that can be used to obtain priority review for a subsequent application or sold to another drug sponsor.
  • The NDA is supported by the results of the global, pivotal, 4WHIM Phase 3 clinical trial of once-daily, oral mavorixafor in individuals with WHIM syndrome.

Takeda Announces Approval of CUVITRU™ Subcutaneous Immunoglobulin in Japan for Patients with Agammaglobulinemia or Hypogammaglobulinemia

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星期一, 九月 25, 2023
Biotechnology, General Health, Infectious Diseases, Health, Pharmaceutical, Infection, Hypogammaglobulinemia, Antibody, TAK, Clinical trial, Immunodeficiency, Pharmacokinetics, PDT, SCIG, Research, SID, Disorder, Sepsis, Plasma, Patient, Standard of care, Risk, PID, Primary immunodeficiency, Diagnosis, Ageing, Pneumonia, Safety, Pharmaceutical industry, Takeda

The approval marks Takeda’s first subcutaneous immunoglobulin (SCIG) therapy for patients in Japan.

Key Points: 
  • The approval marks Takeda’s first subcutaneous immunoglobulin (SCIG) therapy for patients in Japan.
  • Results from the clinical trial in 17 patients in Japan confirmed its efficacy and safety profile1.
  • The most frequently reported adverse reactions were injection site swelling in four patients (23.5%) and injection site erythema in three patients (17.6%)1 during CUVITRU treatment.
  • “We are delighted that CUVITRU, approved in more than 30 countries worldwide, has now been approved in Japan as our first subcutaneous immunoglobulin treatment for agammaglobulinemia or hypogammaglobulinemia,” said Naoyoshi Hirota, regional head of research & development for Takeda’s Plasma-Derived Therapies Business Unit in Japan.

X4 Pharmaceuticals Announces Submission of New Drug Application (NDA) to U.S. FDA for Mavorixafor in WHIM Syndrome

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星期二, 九月 5, 2023
European Hematology Association, File, WHIM, Federation of Clinical Immunology Societies, NDA, CIS, WHIM syndrome, Immune system, EHA, Primary immunodeficiency, Wart, Ageing, Rare, FDA, Pharmaceutical industry, Infection, Myelokathexis, Hypogammaglobulinemia

“The submission of our first NDA is a significant milestone in X4's journey to transform the care of those living with rare immunodeficiencies,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals.

Key Points: 
  • “The submission of our first NDA is a significant milestone in X4's journey to transform the care of those living with rare immunodeficiencies,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals.
  • “We’re excited that this submission moves us one step closer to introducing what could be the first approved product in the U.S. for those with WHIM syndrome.
  • X4 has requested priority review for the application which, if granted, would provide a target FDA review period of six months from the application acceptance for filing date.
  • The NDA submission is supported by the results of the global, pivotal, 4WHIM Phase 3 clinical trial of once-daily, oral mavorixafor in individuals with WHIM syndrome.

Alpine Immune Sciences Announces Initiation of Dosing in 240 mg IgA Nephropathy Cohort of Povetacicept Clinical Study (RUBY-3)

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星期三, 八月 30, 2023
Science, Other Science, Biotechnology, Research, Pharmaceutical, Health, FDA, Clinical Trials, Hypersensitivity, Alpine, Immunoglobulin A, IgA nephropathy, ALPN, Immunoglobulin G, Hypogammaglobulinemia, SMC, Autoimmunity, Safety, Inflammation, Vaccine

Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for autoimmune and inflammatory diseases, announced the successful initiation of the second IgA nephropathy (IgAN) dose cohort in RUBY-3, a phase 1b/2a study in autoimmune glomerulonephritis.

Key Points: 
  • Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for autoimmune and inflammatory diseases, announced the successful initiation of the second IgA nephropathy (IgAN) dose cohort in RUBY-3, a phase 1b/2a study in autoimmune glomerulonephritis.
  • Safety Monitoring Committee (SMC) review has determined that repeat dosing of povetacicept in the first IgAN dose cohort (80 mg subcutaneously once every 4 weeks) has been safe and well-tolerated to date, with no serious or severe adverse events, no events of hypogammaglobulinemia (IgG
  • Dose escalation has been endorsed, and enrollment of the second IgAN dose cohort (240 mg subcutaneously every 4 weeks) has been initiated.
  • “We look forward to providing more detailed clinical updates later this year in appropriate scientific forums.”