Tag Archives: Marc Dunoyer

Ultomiris Receives FDA Approval as First Long-Acting Treatment for AQP4 Ab+ NMOSD, Redefining Patient Care

(IN BRIEF) Ultomiris (ravulizumab-cwvz) has gained approval from the FDA as the inaugural long-acting C5 complement inhibitor for treating adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD). The approval, based on the CHAMPION-NMOSD Phase III trial’s … Read the full press release

Voydeya Recommended for EU Marketing Authorization as PNH Treatment Add-On

(IN BRIEF) Voydeya (danicopan), a first-in-class oral Factor D inhibitor, has been recommended for marketing authorization in the European Union (EU) as an add-on to ravulizumab or eculizumab for adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who experience residual haemolytic … Read the full press release

Acoramidis Shows Positive Results in Japanese Trial for Treating Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

(IN BRIEF) In a Phase III trial conducted in Japan, acoramidis, an investigational treatment for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), demonstrated promising high-level results consistent with the global BridgeBio Pharma, Inc. ATTRibute-CM Phase III trial. The trial in Japan aimed to … Read the full press release

Japan Approves Voydeya (danicopan), an Innovative Treatment for Paroxysmal Nocturnal Haemoglobinuria (PNH)

(IN BRIEF) The Japanese Ministry of Health, Labour and Welfare (MHLW) has granted approval for Voydeya (danicopan), a groundbreaking oral Factor D inhibitor, for the treatment of paroxysmal nocturnal haemoglobinuria (PNH). Voydeya is indicated in Japan for use in combination … Read the full press release

AstraZeneca Teams Up with Cellectis to Accelerate Gene Therapy Development

(IN BRIEF) AstraZeneca has entered into a collaboration and investment agreement with biotech firm Cellectis to fast-track the development of cutting-edge therapeutics in areas such as oncology, immunology, and rare diseases. This partnership will leverage Cellectis’ gene editing technologies and … Read the full press release

AstraZeneca: Soliris Gains Approval in China for Rare Neuromyelitis Optica Spectrum Disorder (NMOSD) Treatment

(IN BRIEF) Soliris (eculizumab) has received approval in China for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive (Ab+). This makes Soliris the first and only complement inhibitor approved for NMOSD … Read the full press release

Transthyretin amyloid cardiomyopathy: Alexion granted an exclusive worldwide licence to develop, manufacture and commercialise NI006 as part of an agreement with Neurimmune AG

Investigational human monoclonal antibody in Phase Ib development for the treatment of transthyretin amyloid cardiomyopathy, a systemic, progressive and fatal condition (PRESS RELEASE) CAMBRIDGE, 7-Jan-2022 — /EuropaWire/ — AstraZeneca (LON: AZN), a British-Swedish multinational pharmaceutical and biotechnology company, has announced … Read the full press release

AL Amyloidosis: Alexion, AstraZeneca Rare Disease, takes over Caelum Biosciences, Inc.

(PRESS RELEASE) CAMBRIDGE, 29-Sep-2021 — /EuropaWire/ — AstraZeneca (LON: AZN), a British-Swedish multinational pharmaceutical and biotechnology company, has announced its rare diseases arm Alexion has exercised its option to acquire all remaining equity in Caelum Biosciences, Inc., a Clinical Stage … Read the full press release