About Steen Bjerre

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So far Steen Bjerre has created 152 blog entries.

FDA accepts BLA for Lanadelumab


The U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) and granted priority review for Lanadelumab (SHP643), an investigational treatment being evaluated for the prevention of angioedema attacks in patients 12 years and older with HAE. “Every day, patients living with HAE struggle to manage their disease — not knowing when [...]

Shire recognizes Rare Disease Day 2018


Shire plc announces its continuing global education and awareness efforts on Rare Disease Day 2018, to deliver on its commitment to champion those impacted by rare disease, including patients, families, and caregivers. The company is helping to raise awareness about the impact and prevalence of rare diseases, while also listening to and working with an [...]

Accelerating time to diagnosis for children with rare diseases


Shire plc, Microsoft and EURORDIS-Rare Diseases Europe has formed a strategic alliance to address the diagnostic challenge for patients living with a rare disease. The long road to diagnosis is one of the most important issues affecting the health, longevity, and well-being for rare disease patients and their families. The Global Commission to End the [...]

FDA Accepts sBLA for Cinryze for Pediatric HAE Use


The U.S. Food and Drug Administration (FDA) has accepted the Cinryze (C1 esterase inhibitor [human]) supplemental Biologics License Application (sBLA) to expand the currently approved indication to include children aged six years and older with HAE. The filing has received Priority Review designation from the FDA, which means Cinryze has an accelerated review target of [...]

Cinryze Manufacturing Process Transfer Approved


The U.S. Food and Drug Administration (FDA) has granted approval for the technology transfer of Cinryze drug product manufacturing process to the Shire manufacturing site in Vienna, Austria. “With the FDA’s approval of the tech transfer, we are pleased to strengthen our supply reliability for adult and adolescent HAE patients who count on Cinryze to [...]

BioCryst and Idera announces merger


BioCryst Pharmaceuticals, Inc. and Idera Pharmaceuticals, Inc. have signed a definitive merger agreement to form a new enterprise focused on the development and commercialization of medicines to serve more patients suffering from rare diseases.  The combined company will be renamed upon closing and will be led by Vincent Milano, CEO of Idera, who will also serve [...]

FDA Acceptance for Review of sBLA for Ruconest


The U.S. Food and Drug Administration (FDA) has accepted for review Pharming Group N.V.'s supplemental Biologics License Application (sBLA) for Ruconest [Recombinant Human C1 Esterase Inhibitor/ conestat alfa] for routine prophylaxis to prevent attacks in adult and adolescent patients with HAE. The FDA has indicated that the sBLA is sufficiently complete to permit a substantive [...]

Verseon presents HAE program


Verseon has presented preclinical results in its HAE program at the 2018 Biotech Showcase in San Francisco, USA. The data show that Verseon’s plasma kallikrein inhibitors are well-suited as oral treatments for this rare genetic disease. To treat HAE, Verseon is developing oral small-molecule inhibitors of plasma kallikrein, a serine protease central to the HAE pathway. [...]

KalVista Commences Clinical Trial


KalVista Pharmaceuticals, Inc. will be initiating a Phase 1 trial for KVD 900, the second candidate in the oral HAE portfolio. The trial commenced in December 2017, in line with KalVista’s previously stated 2017 objectives. Says Andrew Crockett, CEO of KalVista: “2018 will be an exciting year for our HAE portfolio, with our second oral [...]

Adverum accelerates development of pipeline of gene therapies


Adverum Biotechnologies, Inc., a clinical-stage gene therapy company targeting unmet medical needs in serious rare and ocular diseases, reviews recent progress and provides an outlook for 2018: “In 2017, our newly-assembled team achieved our stated goal of transforming Adverum into a clinical-stage company,” said Amber Salzman, Ph.D., president and CEO of Adverum Biotechnologies. “We are [...]