Prix Galien USA 2020
Nominees for Best Biotechnology
The biotech sector continues to pull more than its weight in developing new medicines, a trend that, even in the midst of a global pandemic, has drawn vast amounts of new capital to fund pipeline growth in areas ranging from cell and gene therapy to the microbiome. This year has already broken the all-time record for IPO placements and valuations remain high even for those companies still seeking to launch their first marketed product. Still, small biotech must compete for visibility and resources against the global established players in big pharma, particularly for those with a therapeutic focus on medicines outside the current frenzied search for solutions to the coronavirus pandemic.
To mark the Prix Galien USA’s upcoming October 26 “Week of Discovery and Celebration on the Frontiers of Medical Innovation,” distinguished members of the USA Galien Awards Committee will chair three webinars highlighting each of the 23 nominee products they have selected for the best Biotechnology category.
These virtual expert panelist exchanges will offer the opportunity for webinar participants to assess the current state of innovation in biotech, covering key aspects of clinical progress and differentiation; the ways that patient care is now being conducted outside traditional delivery settings; and the next wave of promising breakthrough technologies, including their transformative – and potentially disruptive – effect on health care productivity and outcomes worldwide.

Best BioTech Product Nominees 2020
Product | Company | Therapeutic Area & Indication |
---|---|---|
Tegsedi® | Akcea Therapeutics and Ionis Pharmaceuticals | Polyneuropathy caused by hereditary transthyretin-amyloidosis (hATTR) in adults. Read more |
ULTOMIRIS® (ravulizumab-cwvz) | Alexion Pharmaceuticals, Inc | Paroxysmal nocturnal hemoglobinuria (PNH) that destroys red blood cells in adults Read more |
GIVLAARI® | Alnylam Pharmaceuticals | Acute hepatic porphyria (AHP) linked to production of toxic porphyrin molecules in the bloodstream in adults Read more |
ONPATTRO® | Alnylam Pharmaceuticals | Polyneuropathy caused by hATTR amyloidosis leading to loss of nerve and organ function Read more |
Aimovig® | Amgen | CGRP receptor blocker to treat chronic migraine Read more |
EVENITY® | Amgen | Osteoporosis in post-menopausal women at high risk of bone fracture Read more |
GALAFOLD® | Amicus Therapeutics, Inc. | Oral drug for treatment of Fabry Disease through repair of gene-induced enzyme deficiencies that lead to organ failures, stroke and early death in adults Read more |
Palynziq® (pegvaliase-pqpz) injection | BioMarin Pharmaceutical, Inc. | Lowers blood levels of phenylalanine, an amino acid that causes PKU (phenylketoneuria) leading to brain damage and seizures beginning in infancy Read more |
ENHERTU® | Daiichi Sankyo & AstraZeneca | Treatment of unresectable or metastatic HER2 positive breast cancer Read more |
OXERVATE® | Dompé U.S. Inc. | Recombinant human growth factor to treat neurotrophic keratitis, a rare condition affecting the eye cornea Read more |
Emgality® | Eli Lilly and Company | Once a month CGRP antibody preventive treatment for adult migraine Read more |
Hemlibra® | Genentech Inc. | Reduce or prevent bleeding episodes in adults and children with hemophilia A, with or without factor-VIII inhibitors Read more |
Polivy™ | Genentech, a member of the Roche Group | Antibody drug conjugate for treatment of diffuse large B-cell lymphoma Read more |
Oxbryta® | Global Blood Therapeutics, Inc. | Prevents inherited propensity for the hemoglobin polymerization that causes sickle cell disease Read more |
TREMFYA® (guselkumab) | Janssen Biotech, Inc. | Medium to severe plaque psoriasis in adults Read more |
LILETTA® | Medicines360 | IUD to prevent pregnancy through timed release of hormone levonorgestrel Read more |
ERVEBO® | Merck & Co., Inc | First preventive vaccine against the disease caused by the Zaire ebolavirus in adults Read more |
Zolgensma® (onasemnogene abeparvovec-xioi) | Novartis Gene Therapies | One-time gene replacement therapy for children under age 2 with spinal muscular atrophy Read more |
Rybelsus® | Novo Nordisk | GLP-1 agonist treatment to control blood sugar in adults with Type-II diabetes Read more |
VYNDAQEL® (tafamidis meglumine) | Pfizer Inc. | For cardiomyopathy associated with transthyretin-mediated amyloidosis (ATTR-CM) to reduce cardiovascular mortality and related hospitalizations Read more |
Andexxa® | Portola Pharmaceuticals, Inc. | Reversal of medicine-specific, anticoagulant-based, and life-threatening bleeding events Read more |
Dupixent® (dupilumab) | Regeneron and Sanofi | Single dose pre-filled pen technology to treat adults and adolescents with atopic dermatitis (eczema), asthma and rhinosinusitis with nasal polyposis Read more |
Cablivi® | Sanofi | Nanobody to treat adult acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood disorder Read more |
TAKHZYRO® | Takeda Pharmaceutical Company Limited | Treatment for hereditary angioedema (HAE) in adults Read more |