Company Product Description Indication Status

Axovant Gene Therapies Ltd., of New York


Gene therapy

GM1 gangliosidosis

FDA granted orphan designation

Levo Therapeutics Inc., of Chicago


Intranasal carbetocin

Prader-Willi syndrome

FDA granted fast track designation

Orchard Therapeutics Ltd., of London


Gene therapy

Metachromatic leukodystrophy

EMA's Committee for Medicinal Products for Human Use granted accelerated assessment

Pfizer Inc., of New York

Abrilada (adalimumab-afzb)

Biosimilar of TNF-alpha inhibitor Humira

Rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, adults Crohn's disease, ulcerative colitis, plaque psoriasis

Approved by FDA; Pfizer plans to launch in 2023

Protalix Biotherapeutics Inc., of Carmiel, Israel, and Chiesi Farmaceutici SpA, of Parma, Italy

Pegunigalsidase alfa (PRX-102)

Plant cent culture-expressed recombinant alpha-galactosidase-A enzyme

Fabry disease

Completed type B pre-BLA meeting with FDA and reached alignment on accelerated approval pathway; BLA to include data from completed phase I/II trials and from ongoing phase III Bridge trial; BLA submission expected by April 2020

Seelos Therapeutics Inc., of New York


Intranasal racemic ketamine

Acute suicidal ideation and behavior in major depressive disorder

FDA granted fast track designation


For more information about individual companies and/or products, see Cortellis.

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