CHESHIRE, Conn. & PARIS — Alexion Pharma France and Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) today received the 2009 Prix Galien France for Soliris(R) (eculizumab) in the category of medicines for rare diseases. The award recognizes the scientific innovation represented by the complement-inhibition technology of Soliris, and the impact the drug...
treatment News
INDIANAPOLIS — Eli Lilly and Company (NYSE: LLY) announced today that the European Medicines Agency’s (EMEA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for the use of ALIMTA(R) (pemetrexed for injection) as monotherapy for the maintenance treatment of locally advanced or metastatic non-small cell...
CAMBRIDGE, Mass. – Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that pivotal trial results from the ILLUMINATE-A Phase 3 study of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – for the treatment of primary hyperoxaluria type 1 (PH1), were published online in...
CAMBRIDGE, Mass. – Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced that pivotal trial results from the ILLUMINATE-A Phase 3 study of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – for the treatment of primary hyperoxaluria type 1...
SUZHOU, China – Alphamab Oncology (stock code: 9966.HK) announced that U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug application (IND) to initiate an open label, multi-center phase II pivotal clinical study (clinical trial No.: KN046-205) in the United States to evaluate the efficacy, safety and tolerability of KN046...
In the midst of a global pandemic, it’s easy to forget that there are other terrible diseases in the world, ones that we know very little about. Consider amyotrophic lateral sclerosis — ALS. Also known as Lou Gehrig’s disease, ALS is an illness whose outcome is never in doubt. The...
DUBLIN — Amarin Corporation plc (NASDAQ:AMRN) today announced encouraging top line results from an exploratory Phase 2a multi-centre, dose-ranging, cross-over clinical study of EN101 in patients with myasthenia gravis, a chronic autoimmune disease characterized by muscle weakness which can be life-threatening. Thomas Lynch, Chairman and Chief Executive Officer of Amarin,...
Ridgeland, MS — Amicus Therapeutics (FOLD) has commenced the U.S. registration Phase 3 trial with its investigational drug, Amigal(TM) (migalastat hydrochloride) for the treatment of Fabry disease. The Company has reached agreement with the FDA on the key protocol design elements of the pivotal trial, including the use of the...
DUBLIN, Ireland, and Boston MA, December 23, 2020, Amryt (Nasdaq: AMYT, AIM: AMYT), a global, commercial-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from serious and life-threatening rare diseases, is pleased to announce today that the U.S. Food and Drug Administration (“FDA”) has granted orphan...
AMSTERDAM — Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that the European Medicines Agency has granted Orphan Drug Designation to AMT’s gene therapy product AMT-021 for the treatment of acute intermittent porphyria (AIP). Orphan Drug Designation for AIP entitles AMT to...