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So far Steen Bjerre has created 217 blog entries.

Health Canada authorizes TAKHZYRO


Shire plc and Shire Pharma Canada ULC (Shire Canada) announces that following priority review, Health Canada has authorized TAKHZYRO (lanadelumab injection) for routine prevention of attacks of HAE in adolescents and adults (12 years of age and older). "The burden HAE patients and their families face every day can’t be ignored,” said Jacquie Badiou, President, HAE Canada. “Our [...]

Additional clinical trial data for prophylaxis of HAE requested


Pharming Group N.V. has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding the supplemental Biologics License Application (sBLA) for Ruconest [C1 Esterase Inhibitor (recombinant)] to expand the current indication to include prophylaxis in patients with HAE. In November 2017, following feedback from FDA on two completed trials of [...]

KalVista Reports First Quarter Results


From the KalVista Pharmaceuticals, Inc. financial results for the fiscal first quarter ended July 31, 2018: “Our recent financings provide significant additional capital for late-stage development of KVD900, our oral plasma kallikrein inhibitor for potential treatment of on-demand acute attacks in patients with HAE,” said Andrew Crockett, CEO. Additionally, we remain committed to our work [...]

BioCryst reports positive results in trial


BioCryst Pharmaceuticals, Inc. announces the initial results from the ZENITH-1 trial showing that a single 750 mg oral dose of BCX7353 was well tolerated and superior to placebo (p<0.05) against the majority of efficacy endpoints evaluated in HAE patients suffering an acute attack. BCX7353 is a novel oral plasma kallikrein inhibitor being developed for both [...]

Shire acquires plasma collection company


Shire plc has acquired sanaplasma AG, a source plasma collection company headquartered in Switzerland. The acquisition is expected to increase Shire’s access to plasma in the longer term and add to its European plasma collection network, complementing existing core capabilities in plasma supply and manufacturing. Plasma is essential to the manufacture of immunoglobulin therapies that help [...]

Orphan Drug Designation for Novel Gene Therapy Candidate


The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ADVM-053, a preclinical gene therapy candidate being investigated as a potential single‑administration treatment which has the potential to provide sustained levels of the C1 esterase inhibitor (“C1EI”) protein. “We are pleased to receive the Orphan Drug Designation for ADVM-053 from the [...]



Following priority review, the U.S. Food and Drug Administration (FDA) has approved TAKHZYRO (lanadelumab-flyo) injection, for prophylaxis to prevent attacks of HAE in patients 12 years of age and older. “TAKHZYRO provides the HAE community with a new option for the prevention of HAE attacks,” said Anthony J. Castaldo, President, U.S. HAEA. “We are grateful [...]

U.S. FDA Grants Fast Track Designation for BioCryst’s BCX7353


The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for BioCryst Pharmaceuticals, Inc.'s BCX7353 for the prevention of angioedema attacks in patients with HAE. Fast Track Designation provides opportunities for frequent interactions with the FDA during development of a product candidate and provides the opportunity for priority review if supported by clinical [...]

Clinical Progress Across Gene Therapy Pipeline


Adverum Biotechnologies, Inc. updates its next-generation ADVM-053 targeting HAE. “We are excited to be able to share positive progress in our lead gene therapy program and to share our continued commitment to improving the quality of life for patients with unmet medical needs,” said Leone Patterson, interim president and CEO of Adverum Biotechnologies. “We are [...]

Pharming’s Financial Results for first half of 2018


On the presentation of the Pharming Group N.V. financial report for the six months ended 30 June 2018 CEO Sijmen de Vries said: “We are delighted with the further progress we have made expanding the reach of Ruconest, allowing more patients to access the clinical benefits of our product. We have continued net profitability in the second [...]