Regulatory
uniQure N.V. announced that the planned enrollment of 56 patients has been achieved in the HOPE-B pivotal trial of etranacogene dezaparvovec, an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B.
GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation and Rare Pediatric Disease Designation to GTX-102 for the treatment of Angelman Syndrome, a serious, debilitating rare neurogenetic disorder that affects approximately 1 in 15,000 people worldwide.
Santhera Pharmaceuticals reports first half-year results as of June 30, 2019, and provides an update on its pipeline and strategic focus.
Esperion announced that, on August 27, 2019, the Compensation Committee of Esperion’s Board of Directors granted non-qualified stock options to purchase an aggregate of 39,500 shares of its common stock and 35,388 restricted stock units to eighteen new colleagues under Esperion’s 2017 Inducement Equity Incentive Plan.
Akari Therapeutics, Plc announced that the U.S. Food and Drug Administration has granted orphan drug designation for nomacopan for the treatment of hematopoietic stem cell transplantation-associated thrombotic microangiopathy.
Vivoryon Therapeutics AG announced its financial results for the first six months of 2019 ending June 30.
Zensun USA, Inc., a wholly-owned subsidiary of Zensun Sci & Tech Co. Ltd., Shanghai, announced that the investigation of NEUCARDIN, its Recombinant human neuregulin-1 fragment, has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for the potential treatment of Chronic Heart Failure (CHF).
The first half of the year has been characterized by continued work to develop the company’s innovative projects in two treatment areas, both of which have significant medical needs because the current treatment options are less effective or non-existent.
Promore Pharma AB today announced that the company was granted a patent in Japan for the usage of LL-37 for treating chronic wounds.
Pharmaceutical manufacturing license and GMP compliance of the Rentschler Fill Solutions plant reconfirmed by the Austrian Agency for Health and Food Safety (AGES)
PRESS RELEASES