Company Product Description Indication Status

Axovant Gene Therapies Ltd., of New York

AXO-AAV-GM1

Gene therapy

GM1 gangliosidosis

FDA granted orphan designation

Levo Therapeutics Inc., of Chicago

LV-101

Intranasal carbetocin

Prader-Willi syndrome

FDA granted fast track designation

Orchard Therapeutics Ltd., of London

OTL-200

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

SLS-002

Intranasal racemic ketamine

Acute suicidal ideation and behavior in major depressive disorder

FDA granted fast track designation

Notes

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

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