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. |