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

ProductManufacturerTherapeutic Area &
Akcea Therapeutics and Ionis PharmaceuticalsPolyneuropathy caused by hereditary transthyretin-amyloidosis (hATTR) in adults.
ULTOMIRIS® (ravulizumab-cwvz)
Alexion Pharmaceuticals, IncParoxysmal nocturnal hemoglobinuria (PNH) that destroys red blood
cells in adults
GIVLAARI®Alnylam PharmaceuticalsAcute hepatic porphyria (AHP) linked to production of toxic porphyrin
molecules in the bloodstream in adults
ONPATTRO®Alnylam PharmaceuticalsPolyneuropathy caused by hATTR amyloidosis leading to loss of nerve
and organ function
Aimovig®AmgenCGRP receptor blocker to treat chronic migraine
EVENITY®AmgenOsteoporosis in post-menopausal women at high risk of bone fracture
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
Palynziq® (pegvaliase-pqpz) injectionBioMarin Pharmaceutical, Inc.Lowers blood levels of phenylalanine, an amino acid that causes PKU
(phenylketoneuria) leading to brain damage and seizures beginning in infancy
ENHERTU®Daiichi Sankyo & AstraZenecaTreatment of unresectable or metastatic HER2 positive breast cancer
OXERVATE®Dompé U.S. Inc.Recombinant human growth factor to treat neurotrophic keratitis,
a rare condition affecting the eye cornea
Emgality®Eli Lilly and CompanyOnce a month CGRP antibody preventive treatment for adult migraine
Hemlibra®Genentech Inc.Reduce or prevent bleeding episodes in adults and children with
hemophilia A, with or without factor-VIII inhibitors
Polivy™ Genentech, a member of the Roche GroupAntibody drug conjugate for treatment of diffuse large B-cell lymphoma
Global Blood Therapeutics, Inc. Prevents inherited propensity for the hemoglobin polymerization that
causes sickle cell disease
TREMFYA® (guselkumab)Janssen Biotech, Inc.Medium to severe plaque psoriasis in adults
LILETTA®Medicines360IUD to prevent pregnancy through timed release of hormone levonorgestrel
ERVEBO®Merck & Co., IncFirst preventive vaccine against the disease caused
by the Zaire ebolavirus in adults
Zolgensma® (onasemnogene abeparvovec-xioi)
Novartis Gene Therapies One-time gene replacement therapy for children under age 2 with spinal muscular atrophy
Rybelsus®Novo Nordisk GLP-1 agonist treatment to control blood sugar in adults with Type-II diabetes
Andexxa®Portola Pharmaceuticals, Inc.Reversal of medicine-specific, anticoagulant-based,
and life-threatening bleeding events
Dupixent® (dupilumab)
Regeneron and SanofiSingle dose pre-filled pen technology to treat adults and adolescents with
atopic dermatitis (eczema), asthma and rhinosinusitis with nasal polyposis
Cablivi®SanofiNanodrug to treat adult acquired thrombotic thrombocytopenic
purpura (aTTP), a rare blood disorder
TAKHZYRO®Takeda Pharmaceutical Company LimitedTreatment for hereditary angioedema (HAE) in adults