The Committee for Medicinal Products for Human Use (CHMP) of the European Medicine Agency (EMA) has recommended approval of Ruconest for the treatment of acute angioedema attacks in children with HAE. This marketing authorization would expand the age range of Pharming Group N.V.‘s lead product, a recombinant analogue of the human C1 esterase inhibitor produced [...]
New data from the APeX-2 trial shows that 150 mg of oral, once-daily berotralstat (BCX7353) for prophylaxis of HAE attacks reduced patients’ monthly use of standard of care (SoC) on-demand medicine by 53.6 percent compared to placebo, and reduced the number of HAE attacks requiring acute SoC treatment by 49.2 percent compared to placebo. Additional [...]
The US Food and Drug Administration (FDA) has approved Pharming Group N.V.’s Prior Approval Supplement to add the new Netherlands production facility of starting material to the Biologics License Application (BLA) to support its lead product Ruconest. With the addition of this new facility for US supplies as well, Pharming can continue to expand sales [...]
At the release of the financial results for the fiscal third quarter ending 31 January 2020 KalVista Pharmaceuticals, Inc. CEO Andrew Crockett said the ‘work to bring multiple best-in-class oral therapies to patients with HAE is advancing well’: “First, we look forward to delivering data in the second quarter of this year from our Phase 2 [...]
Announcing the financial results for the full year 2019, BioCryst Pharmaceuticals, Inc. President and CEO Jon Stonehouse says that “2020 is off to a strong start, with NDAs accepted and approvals lined up later this year in the U.S. and Japan”: “We are hearing increasing excitement from both HAE patients and physicians about the availability of an [...]
BioMarin Pharmaceutical Inc., a global biotechnology company that develops and commercializes therapies for people with serious and life-threatening rare diseases and medical conditions, has announced its financial results for 2019. From the Key Program Highlights: On 14 November 2019, BioMarin introduced its third gene therapy candidate, BMN 331, for the treatment of HAE. The company [...]
