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Immune Deficiencies

FDA Grants Breakthrough Therapy Designation for X4 Pharmaceuticals’ Mavorixafor for Treatment of WHIM Syndrome

CAMBRIDGE, Mass.–(BUSINESS WIRE)–X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a clinical-stage biopharmaceutical company focused on the development of novel therapeutics for the treatment of rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for mavorixafor (X4P-001) for the treatment of adult patients with WHIM (Warts, Hypogammaglobulinemia, Infections, …

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FDA Grants Regenerative Medicine Advanced Therapy Designation to MB-107 Lentiviral Gene Therapy for X-Linked Severe Combined Immunodeficiency

NEW YORK and MEMPHIS, Tenn., Aug. 22, 2019 (GLOBE NEWSWIRE) — Mustang Bio, Inc. (NASDAQ: MBIO) and St. Jude Children’s Research Hospital have announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted the Regenerative Medicine …

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ADMA Biologics Receives FDA Approval for License Transfers for Bivigam and Nabi-HB

RAMSEY, N.J. and BOCA RATON, Fla., July 08, 2019 (GLOBE NEWSWIRE) — ADMA Biologics, Inc. (NASDAQ: ADMA) (“ADMA” or the “Company”), a vertically integrated commercial biopharmaceutical and specialty immunoglobulin company that manufactures, markets and develops specialty plasma-derived biologics for the treatment of immune deficiencies and the prevention of certain infectious …

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FDA Approves Grifols’ Xembify, 20% Subcutaneous Immunoglobulin for Primary Immunodeficiencies

Barcelona, July 4, 2019.– Grifols (MCE:GRF, MCE:GRF.P NASDAQ:GRFS), a leading global producer of plasma-derived medicines, announced today that Xembify®, its new 20% subcutaneous immunoglobulin, has been approved by the U.S. Food and Drug Administration (FDA). Xembify® is used to treat primary immunodeficiencies. The FDA approval marks the culmination of an important …

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FDA Approves New Product Label for Octapharma’s Octaplas

HOBOKEN, N.J.–(BUSINESS WIRE)–The U.S. Food and Drug Administration (FDA) has approved a revised product label for Octapharma USA’s Octaplas™, Pooled Plasma (Human), Solvent/Detergent Treated Solution for Intravenous Infusion. The new labeling provides the results of a post-marketing study on Octaplas™ that demonstrates the product’s safety, efficacy and tolerability in treating critically ill pediatric …

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FDA Approves ADMA Biologics’ Asceniv, a Novel Intravenous Immune Globulin

RAMSEY, N.J. and BOCA RATON, Fla., April 01, 2019 (GLOBE NEWSWIRE) — ADMA Biologics, Inc. (NASDAQ: ADMA) (“ADMA” or the “Company”), a vertically integrated commercial biopharmaceutical and specialty immunoglobulin company that manufactures, markets and develops plasma-derived biologics for the treatment of immune deficiencies and the prevention of certain infectious diseases, …

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FDA Approves Leadiant’s Revcovi for the Treatment of ADA-SCID in Pediatric and Adult Patients

GAITHERSBURG, Md.–(BUSINESS WIRE)–Leadiant Biosciences, Inc. today announced that the Food and Drug Administration (FDA) has granted approval to Revcovi™ (elapegademase-lvlr) injection in the U.S. Revcovi is a new enzyme replacement therapy (ERT) for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients. “We are gratified …

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Enzyvant and Visikol Collaborate to Develop Innovative Digital Pathology Assay to Support RVT-802 Development

BASEL, Switzerland and WHITEHOUSE STATION, N.J., July 5, 2018 /PRNewswire/ — Enzyvant, a biopharmaceutical company focused on developing innovative treatments for patients with rare diseases, today announced that it has formed a partnership with Visikol, a contract research organization focused on digital pathology and drug discovery. Under the terms of …

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Early Diagnosis Can Save Babies’ Lives: A Guide to Severe Combined Immunodeficiency Disease (SCID)

A new review provides guidance on a deadly, but rare, disease that is potentially curable if identified early. Severe combined immunodeficiency disease (SCID), known as the “bubble boy disease” in the 1970s, is treatable with a stem cell transplant, gene therapy and other treatments if identified at birth or soon …

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FDA Grants Breakthrough Therapy and Regenerative Advanced Therapy Designation to Enzyvant’s RVT-802

BASEL, Switzerland, April 17, 2017 /PRNewswire/ — Enzyvant, a biopharmaceutical company focused on developing innovative treatments for patients with rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted RVT-802, Enzyvant’s investigational tissue-based therapy for the treatment of complete DiGeorge Syndrome (cDGS), Breakthrough Therapy designation as …

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