Chugai Pharmaceutical Co. Ltd., of Tokyo, and Roche Holding AG, of Basel, Switzerland

Hemlibra (emicizumab)

Factor IX/X antagonist

Hemophilia A

European Commission approved drug in all age groups for routine prophylaxis of bleeding episodes in severe hemophilia A (congenital factor VIII deficiency, FVIII <1%) without FVIII inhibitors

3/14/19

Cstone Pharmaceuticals Co. Ltd., of Suzhou, China

CS-3003

Histone deacetylase 6 selective inhibitor

Advanced solid tumors, relapsed/refractory multiple myeloma

China's NMPA accepted IND filing for multicenter, dose-escalation phase I study in China and Australia

3/13/19

Cynata Therapeutics Ltd., of Melbourne, Australia

CYP-002

Mesenchymal stem cells

Critical limb ischemia

The Medicines and Healthcare products Regulatory Agency in the U.K. advised the company that its manufacturing and completed preclinical development were acceptable; company needs to run a preclinical biodistribution study before running a 90-patient phase II study, which is scheduled to start in the second half of 2019

3/12/19

Specialized Therapeutics Asia Pte. Ltd., of Singapore

Nerlynx (neratinib)

Binds to the intercellular signaling domain of HER1, HER2, HER3 and epithelial growth factor receptor

Early stage breast cancer

Received marketing authorization from Australia's Therapeutic Goods Administration to commercialize in Australia for the extended adjuvant treatment of adult patients with early stage HER2-overexpressed/amplified breast cancer following adjuvant trastuzumab (Herceptin, Roche Holding AG)-based therapy

3/18/19

Takeda Pharmaceutical Co. Ltd., of Tokyo

Flexbumin

Albumin

Hypovolemia, hypoalbuminemia, burns, adult respiratory distress syndrome, nephrosis, cardiopulmonary bypass surgery and hemolytic disease of the newborn

FDA approved manufacturing at new facility near Covington, Ga.

3/18/19

Vertex Pharmaceuticals Inc., of Boston

Symdeko (tezacaftor/ivacaftor and ivacaftor)

CFTR modulator/stimulator

Cystic fibrosis

Therapeutic Goods Administration of Australia granted registration to the drug for use in patients 12 and older who are homozygous for the F508del mutation or who have at least 1 mutation in the cystic fibrosis transmembrane conductance regulator gene that is responsive to the combination based on in vitro data and/or clinical evidence

3/12/19

Notes

The date indicated refers to the BioWorld Regulatory actions table in which the news item can be found.

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