Bjerre

About Steen Bjerre

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So far Steen Bjerre has created 222 blog entries.

Orphan Drug Designation for Novel Gene Therapy Candidate

2018-08-26T18:00:19+00:00August 26, 2018|HAEi News|

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 [...]

US FDA approves TAKHZYRO

2018-08-25T15:01:55+00:00August 24, 2018|HAEi News|

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

2018-08-07T07:30:25+00:00August 7, 2018|HAEi News|

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

2018-08-03T09:45:08+00:00August 3, 2018|HAEi News|

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

2018-07-27T11:38:20+00:00July 27, 2018|HAEi News|

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 [...]

APeX-1 Clinical Trial Results for BCX7353

2018-07-26T16:24:09+00:00July 26, 2018|HAEi News|

Results from the Phase 2, APeX-1 clinical trial of BCX7353 for the prevention of attacks in patients with HAE are published online in the July 26th issue of The New England Journal of Medicine. The APeX-1 trial was a double blind, randomized, parallel group, placebo-controlled Phase 2 dose ranging trial comparing the safety and efficacy [...]

The HAEi Magazine ‘Global Perspectives’ out now

2018-07-11T20:15:16+00:00July 11, 2018|HAEi News|

It is July – and it is time for the second 2018 issue of the HAEi magazine ’Global Perspectives’. This time the magazine is truly covering the world as we focus on the record-breaking 2018 HAE Global Conference that took place in Vienna, Austria 17-20 May. Indeed, the fourth HAE global Conference set new records [...]

Agreement with PMDA on Clinical Trial and Regulatory Requirements

2019-05-07T12:56:15+00:00June 25, 2018|HAEi News, Uncategorized|

BioCryst Pharmaceuticals, Inc. has reached agreement on the design of a Phase 3 trial and regulatory requirements for marketing authorization of BCX7353 for HAE with the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan. The Phase 3 trial design agreed upon for Japan, APeX-J, is a randomized, placebo-controlled double-blind trial of 24 weeks duration with [...]

FDA Approval for Label Expansion of Cinryze

2018-06-21T08:39:03+00:00June 21, 2018|HAEi News|

The U.S. Food and Drug Administration (FDA) has approved a label expansion for Cinryze® (C1 esterase inhibitor [human]), making it available to help prevent angioedema attacks in children aged 6 years and older with HAE. Cinryze has been approved in the U.S. since October 2008 for routine prophylaxis against attacks in adolescents and adults living [...]

Record-breaking HAE Global Walk 2018

2018-06-04T09:43:51+00:00June 4, 2018|HAEi News|

In 2016 HAEi introduced the HAE Global Walk in order to raise awareness around the world. The third edition of the HAE Global Walk took place from 1 April to 31 May 2018 and broke each and every record as 54,609,008 steps were taken by 4,262 people from 37 countries. That is 42,007 km or [...]