Company

Product

Description

Indication

Status

Aridis Pharmaceuticals Inc., of San Jose, Calif.

AR-501

Inhaled formulation of gallium citrate with broad-spectrum anti-infective activity

Cystic fibrosis

FDA granted both fast track and qualified infectious disease product designations; agency also cleared an IND to start a phase I/IIa trial next month

Chugai Pharmaceutical Co. Ltd., of Tokyo

Hemlibra (emicizumab)

Bispecific antibody binding factor IXa and factor X

Hemophilia A

Approved by Taiwan Food and Drug Administration for routine prophylaxis of bleeding episodes in patients with factor VIII inhibitors

Novartis AG, of Basel, Switzerland

Zolgensma (AVXS-101)

Gene replacement therapy

Spinal muscular atrophy type 1

FDA accepted the BLA and granted priority review; regulatory action anticipated in May 2019

Proqr Therapeutics NV, of Leiden, the Netherlands

QR-421a

RNA-based oligonucleotide

Usher syndrome type 2 and nonsyndromic retinitis pigmentosa due to mutations in exon 13 of USH2A gene

FDA cleared the IND for phase I/II STELLAR trial; preliminary data expected in mid-2019

Scholar Rock Holding Corp., of Cambridge, Mass.

SRK-015

Selective inhibitor of myostatin

Spinal muscular atrophy

EMA's Committee for Orphan Medicinal Products adopted a positive opinion for orphan designation


Notes

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