About Steen Bjerre

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So far Steen Bjerre has created 183 blog entries.

First patient dosed in APeX-S trial


BioCryst Pharmaceuticals, Inc. has dosed the first patient in APeX-S, a long-term safety trial evaluating two dosage strengths of BCX7353 administered orally once-daily as a preventive treatment in patients with HAE. “Initiation of the APeX-S trial is an important milestone to support filing and approval of BCX7353, and furthers our core strategy of bringing a [...]

EMA grants accelerated assessment for Lanadelumab


The European Medicines Agency (EMA) has granted an accelerated assessment for Lanadelumab (SHP643), an investigational treatment being evaluated for the prevention of angioedema attacks in patients 12 years and older with HAE. “The EMA decision, coupled with the U.S. FDA’s recent Priority Review designation for Lanadelumab, reinforces Shire’s dedication to advancing new treatment options for [...]

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. [...]