Hereditary Angioedema

FDA Accepts BioCryst’s NDA for Oral, Once Daily Berotralstat to Prevent Hereditary Angioedema Attacks

RESEARCH TRIANGLE PARK, N.C., Feb. 18, 2020 (GLOBE NEWSWIRE) — BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) announced that the U.S. Food and Drug Administration (FDA) has accepted and filed its new drug application (NDA) for the approval of oral, once daily berotralstat (BCX7353) for the prevention of hereditary angioedema (HAE) attacks. The Prescription …

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REGENXBIO Expands Pipeline Using NAV Vectors to Deliver Therapeutic Antibodies for the Treatment of Hereditary Angioedema and Neurodegenerative Diseases

ROCKVILLE, Md., July 24, 2019 /PRNewswire/ — REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, today announced an expansion of its pipeline to include novel treatments for hereditary angioedema (HAE) and …

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BioCryst’s Oral BCX7353 Meets Primary Endpoint in Phase 3 Trial for Prevention of Hereditary Angioedema Attacks

RESEARCH TRIANGLE PARK, N.C., May 21, 2019 (GLOBE NEWSWIRE) — BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the randomized (n=121), double-blind, placebo-controlled, Phase 3 APeX-2 trial of once-daily, oral BCX7353 for the prevention of hereditary angioedema (HAE) attacks achieved its primary endpoint for both dose levels (110 mg and 150 mg), …

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Shire Announces Publication of Complete Results of Phase 3 Study of Lanadelumab as Preventive Treatment for Hereditary Angioedema

Dublin, Ireland – 27 November 2018 – Shire plc (LSE: SHP, NASDAQ: SHPG), the leading global biotechnology company focused on rare diseases, today announced the Journal of the American Medical Association (JAMA) publication of complete results from the Phase 3 HELP Study™, a randomised, placebo-controlled trial evaluating the efficacy and safety of subcutaneously …

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FDA Approves Shire’s Takhzyro, a First-of-its-Kind mAb Preventive Treatment for Hereditary Angioedema

Dublin, Ireland – August 23, 2018 – Shire plc (LSE: SHP, NASDAQ: SHPG), the leading global biotechnology company focused on rare diseases, today announced that following priority review, the U.S. Food and Drug Administration (FDA) has approved TAKHZYRO™ (lanadelumab-flyo) injection, for prophylaxis to prevent attacks of hereditary angioedema (HAE) in patients 12 …

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CSL Behring’s Haegarda Receives Orphan-Drug Exclusivity for Prevention of Hereditary Angioedema Attacks

KING OF PRUSSIA, Pa., July 21, 2017 /PRNewswire/ — Global biotherapeutics leader CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has granted the Company seven years of orphan-drug exclusivity for HAEGARDA® (C1 Esterase Inhibitor Subcutaneous [Human]), the first and only subcutaneous treatment option for prevention of …

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FDA Approves CSL Behring’s Haegarda for Prevention of Hereditary Angioedema Attacks

Global biotherapeutics leader CSL Behring today announced that the U.S. Food and Drug Administration (FDA) has approved HAEGARDA® (C1 Esterase Inhibitor Subcutaneous [Human]), the first and only subcutaneous therapy indicated for routine prophylaxis to prevent hereditary angioedema (HAE) attacks in adolescent and adult patients. HAE is a rare, genetic, and …

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Shire’s Investigational Hereditary Angioedema Drug Demonstrates Positive Results in Late-Stage Trial

Lexington, MA – May 18, 2017 – Shire plc (LSE: SHP, NASDAQ: SHPG), the global leader in rare diseases, announces positive topline Phase 3 results for the HELP™ study, a global, multi-center, randomized, double-blind placebo-controlled parallel group trial that evaluated the efficacy and safety of subcutaneously administered lanadelumab versus placebo …

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CSL Behring’s CSL830 Prevents Hereditary Angioedema Attacks in Late-Stage Study

Median reduction in hereditary angioedema (HAE) attack rate relative to placebo was 95% at the highest dose studied Data show up to 40% of HAE patients were free from attacks throughout the study In the clinical study, CSL830 met the primary endpoint for preventing HAE attacks Global biotherapeutics leader CSL …

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FDA Accepts CSL’s BLA for First Subcutaneous Prophylactic Therapy to Prevent HAE Attacks

KING OF PRUSSIA, Pa., Aug. 30, 2016 /PRNewswire/ — CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has accepted for review the company’s Biologics License Application (BLA) for its low-volume subcutaneous (SC) C1-Esterase Inhibitor (C1-INH) Human replacement therapy, CSL830, as prophylaxis to prevent Hereditary Angioedema (HAE) …

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