26 Feb, 2019
Rare Disease Day Kit
30 Nov, 2018
Minutes package HAEi – Youngsters Advocacy Workshop
Dear HAEi Member Organization,
As you recall, the “Youngster Track” at the 2018 HAE Global Conference in Vienna was our first dedicated two-day youngster track and was a great success. At the end of the conference we proudly introduced the official HAEi Youngsters Community. […]
23 Nov, 2018
Remembering Joseph Armando Peter Andolfatto
8 Nov, 2018
Newfoundland and Labrador Prescription Drug Program, BULLETIN – NO. 164
Benefit List Update: New Addition
Effective October 1, 2018, FIRAZYR 10MG/ML SOLUTION will be added to the benefit list with the following special authorization criteria: […]
23 Aug, 2018
HAE Canada Global Walk Success 2018
While participants of the HAEi 2018 Global Conference were in Vienna, they had the opportunity to take part in the HAEi Jakobsweg Walk to help raise HAE awareness. […]
23 Aug, 2018
Plant Tours of Sponsors
While attending the Global Conference in Vienna 6 Board of Directors of HAE Canada participated in plant tours for our sponsors […]
21 Jun, 2018
FDA Approval for Label Expansion of Cinryze
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. […]
4 Jun, 2018
Record-breaking HAE Global Walk 2018
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 […]
30 May, 2018
Additional Analyses of APeX-1 Clinical Trial of BCX7353
BioCryst Pharmaceuticals, Inc. presented additional analyses of the APeX-1 trial of BCX7353 for the prevention of attacks in patients with HAE at the annual congress of the European Academy of Allergy and Clinical Immunology (EAACI) […]
26 May, 2018
Official video from the 2018 HAE Global Conference
17-20 May 2018 more than 730 delegates from 57 countries met in Vienna, Austria for the 4th HAE Global Conference. You will find the official video with impressions from the record-breaking conference here.
25 May, 2018
European Regulatory Designations for HAE Treatment
The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion on BioCryst Pharmaceuticals, Inc.’s application for orphan designation of BCX7353 for the treatment of HAE. In addition, the United […]
15 May, 2018
Pharming supports haeday 🙂
On May 16th HAE patient organizations from around the world, led by HAEi (The international HAE patient organisation), come together to support the global awareness day for Hereditary Angioedema; a rare life-threatening condition. “haeday:)” aims to […]
8 May, 2018
Agreement for production of recombinant C1 Esterase Inhibitor
CEVEC Pharmaceuticals GmbH and CSL Limited have signed an exclusive licensing agreement for the development, manufacture, and commercialization of recombinant C1 Esterase Inhibitor (C1-INH) proteins for HAE and other potential indications using CEVEC’s proprietary CAP®Go technology. […]
8 May, 2018
HAEi Jakobsweg Walk 2018 – information for all pilgrims
Buen Camino, HAE pilgrims! Thank you for signing up for the HAEi Jakobsweg Walk 2018 — we look very much forward to walking with you and the many other HAE pilgrims. On this page, you […]
18 Apr, 2018
Swissmedic Validates Marketing Authorization Application for Lanadelumab
The Swiss Agency for Therapeutic Products (Swissmedic) has validated the marketing authorization application (MAA) for Lanadelumab (SHP643). The validation of the MAA confirms that the Lanadelumab MAA submission is complete and that the formal review […]
15 Apr, 2018
Join the community for HAEi youngsters
As you may recall the HAE Global Conference 2016 in Madrid, Spain contained a full track for HAE youngsters – and in August 2017 HAEi arranged the first ever HAEi Youngsters’ Summer Camp, which took […]
14 Apr, 2018
The HAEi Global Access Program helps changing lives of HAE patients
Since announcing the appointment of Inceptua Medicines Access as the new HAEi Global Access Program (GAP) distribution partner last year, HAEi has been responding to a number of inquiries from those in countries where RUCONEST® […]
13 Apr, 2018
HAEi Connect – online membership database free for all member organizations
HAEi is proud to present a new initiative for the member organizations: HAEi Connect – a cloud-based member database for national organizations to manage their members. “In our close collaboration with the national member organizations, […]
12 Apr, 2018
Get your website hosted at haei.org
A growing number of national HAE organizations have their own websites with their own individual hosting solution. However, some of them would like to change hosting or altogether change the look and content of their […]
11 Apr, 2018
HAEi Jakobsweg Walk 2018
After the successful HAEi/AEDAF Camino Walk on the Camino de Santiago in northwestern Spain in May 2016 and May 2017, HAEi is organizing yet another walk. In order to raise awareness, HAEi brings together HAE […]
10 Apr, 2018
2017 revision and update of the international WAO/EAACI guideline for the management of HAE
An international expert panel has reviewed the existing evidence and developed 20 recommendations that were discussed, finalized and consented during the guideline consensus conference in 2016 in Vienna, Austria. The final version of this update […]
9 Apr, 2018
Two decades with AEDAF in Spain
Asociación Española de Angioedema Familiar por Deficiencia del Inhibidor de C1 – or in short AEDAF – held its 20th Annual Meeting and General Assembly in Madrid Saturday 10 March 2018. Dr. Teresa Caballero Molina […]
8 Apr, 2018
Potentially 28,000 HAE patients in China
By Fiona Wardman, HAEi Executive Committee member and President of HAE Australasia China has a population of approximately 1.4 billion, which means there are potentially 28,000 patients with HAE in this huge country. Based on […]
31 Mar, 2018
Thousands will help raise HAE awareness step by step
On and around hae day 🙂 2018 a group of HAE patients, relatives, caregivers, doctors, nurses, people from HAE organizations, and industry will walk four stages of the Jakobsweg through Vienna, Austria. Jakobsweg is German for […]
30 Mar, 2018
Regulatory Milestones for Investigational HAE Treatment Lanadelumab
The European Medicines Agency (EMA) has validated its marketing authorization application (MAA) for Lanadelumab (SHP643) from Shire plc – and Health Canada has completed screening and accepted the New Drug Submission (NDS) under Priority Review […]
29 Mar, 2018
HAEi magazine #1/2018 out now
April is approaching fast, and so is the 2018 HAE Global Conference in Vienna, Austria. Therefore we publish the first issue of the HAEi magazine ”Global Perspectives” where you, among many other topics, will find articles […]
16 Mar, 2018
KalVista Reports Fiscal Third Quarter Results
KalVista Pharmaceuticals, Inc. reports operational and financial results for the fiscal third quarter ended 31 January 2018. “We are pleased to have the second candidate from our oral HAE portfolio in a Phase 1 trial as we […]
16 Mar, 2018
CSL Behring Receives Patient Impact Award
Imagine not knowing when you might experience unpredictable, severe and painful swelling without warning in different parts of your body, including the abdomen, face, and larynx. This is what it’s like for patients with HAE […]
16 Mar, 2018
BioCryst Announces Initiation of Phase 3 APeX-2 Trial
BioCryst Pharmaceuticals, Inc. announces the dosing of the first patient into APeX-2, a Phase 3 clinical trial evaluating two dosage strengths of BCX7353 administered orally once-daily (QD) as a preventive treatment to reduce the frequency […]
15 Mar, 2018
CSL’s CEO on Industry to Improve Patients’ Access to Medicines
In a keynote address on patients’ access to medicines at the eyeforpharma Barcelona summit, CSL Behring CEO and Managing Director Paul Perreault told fellow biotech industry leaders that access to life-saving medicines remains a global […]
7 Mar, 2018
Pharming’s Preliminary Financial Results for 2017
At the presentation of the Pharming Group N.V. preliminary (unaudited) financial report for the full year ended 31 December 2017 CEO Sijmen de Vries said: “The remarkable growth reported in 2017 was a direct result of […]
7 Mar, 2018
Adverum Reports Fourth Quarter 2017 Financial Results
From the Adverum Biotechnologies, Inc. financial results for the fourth quarter ended 31 December 2017 and corporate update: “We enter 2018 with significant momentum following a year of critical execution to transform Adverum into a […]
28 Feb, 2018
Sobi launches enhanced corporate sustainability initiative
Swedish Orphan Biovitrum AB (Sobi) announces the launch of an enhanced corporate sustainability programme with a strong connection to the company’s strategy of providing sustainable access to treatments for rare diseases. As part of the […]
28 Feb, 2018
BioCryst reports full year financial results
At the announcement of the BioCryst Pharmaceuticals, Inc. financial results for 2017 President & CEO Jon P. Stonehouse said: “Our team made significant progress in 2017 and we are off to a strong start in 2018. […]
28 Feb, 2018
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 […]
27 Feb, 2018
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, […]
23 Feb, 2018
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 […]
23 Feb, 2018
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 […]
20 Feb, 2018
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 […]
16 Feb, 2018
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 […]
24 Jan, 2018
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 […]
22 Jan, 2018
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 […]
19 Jan, 2018
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 […]
11 Jan, 2018
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. […]
6 Jan, 2018
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. […]
6 Jan, 2018
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 […]
5 Jan, 2018
HAEi Connect is ready to help the global member organizations
Over the last months, HAEi has been working hard on completing HAEi Connect – a member database, communication management tool, and much more that HAEi is offering free of charge to its member organizations. “HAEi […]
Jan, 2018
HAE Scandinavia Conference 2017
The second weekend in November 2017 the beautiful conference venue Bergendal – right at the rim of Edsviken bay in Sollentuna just 20 minutes north of Stockholm, Sweden served as the inspirational setting for the […]
3 Jan, 2018
First HAE doctors and patients meeting in South Africa
The first doctor and patient HAE meeting was held in Cape Town at the Groote Schuur Hospital on the 25 October 2017 gathering patients from various regions of South Africa. There were 27 patients and […]
2 Jan, 2018
HAEi Central and Eastern Europe Workshop 2017
7-8 October 2017 the combined 4th HAE National Conference and the 2nd HAEi Central and Eastern Europe Workshop took place at the Westin Warsaw Hotel in Warsaw, Poland. This is a report from the HAEi […]
1 Jan, 2018
HAEi South Eastern Europe Workshop 2017
HAE Macedonia and the Regional Patient Advocate for South Eastern Europe Natasa Angjeleska hosted the second regional meeting for HAE patients, caregivers, and physicians in Skopje 29-30 September 2017. This is a report from Mrs. […]
22 Dec, 2017
December magazine out now
More and more HAE related activities are going on around the world. In the December 2017 issue of the HAEi magazine ‘Global Perspectives’ you can – for example – read about: The 2018 HAE Global […]
15 Dec, 2017
KalVista Reports Second Quarter Results
KalVista Pharmaceuticals, Inc. today reported operational and financial results for the fiscal second quarter ended October 31, 2017. The initial focus is on inhibitors of plasma kallikrein, which is an important component of the body’s […]
7 Dec, 2017
The dawn of a new era of HAE management
Symposium 19th June 2017 – as part of the European Academy of Allergy and Clinical Immunology (EAACI) Annual Meeting in Helsinki, Finland. This symposium provided an overview of past, current, and future therapies and routes […]
28 Nov, 2017
Supplemental Biologics License Application to FDA
Pharming Group N.V. has submitted a supplemental Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for Ruconest [Recombinant Human C1 Esterase Inhibitor/ conestat alfa] for routine prophylaxis to prevent attacks in […]
10 Nov, 2017
FDA application for new drug in 2018
Amber Salzman, Ph.D., President and CEO of Adverum Biotechnologies Inc., in the company’s financial results for the third quarter of 2017: “We continue to make progress advancing our gene therapy programs to reach our goal […]
31 Oct, 2017
98 percent median reduction in HAE attacks
CSL Behring presents data indicating that, at the approved dose of 60 IU/kg, HAEGARDA®reduced the median number of HAE attacks per month by 98 percent in subjects who had frequent attacks, from a 16-week placebo […]
27 Oct, 2017
Cinryze production has resumed
Shire plc announces unaudited results for the three months ended September 30, 2017. Flemming Ornskov, M.D., M.P.H., Shire CEO, commented: “We delivered strong growth this quarter despite a Cinryze supply shortage. We experienced a product shortage of […]
27 Oct, 2017
On the right track
Pharming Group N.V. presents its (unaudited) financial report for the first nine months and the third quarter ended 30 September 2017. Operational highlights during the third quarter On September 11, following the conclusion of the […]
26 Oct, 2017
European Approval for Label Extension of Firazyr
The European Commission (EC) has approved a label extension granting a new indication for Firazyr (icatibant injection), broadening its use to adolescents and children aged 2 years and older, with HAE caused by C1-esterase-inhibitor (C1-INH) deficiency. […]
2 Oct, 2017
Positive data from paediatric clinical trial with Ruconest
Pharming Group N.V. announces positive data from a clinical trial with the use of Ruconest [Recombinant Human C1 Esterase Inhibitor/ conestat alfa] for the treatment of HAE attacks in children. The open-label, single arm, Phase II […]
28 Sep, 2017
Verseon developing oral treatment for HAE
Verseon Corporation has announced the launch of a drug program developing oral treatments for HAE. In their interim report, the Company presents data from a well-established HAE disease model which demonstrate the efficacy of their plasma […]
27 Sep, 2017
September magazine out now
More and more HAE related activities are going on around the world. In fact, we think it is safe to say that there has never been so many exciting events and HAE news to tell […]
26 Sep, 2017
Pharming and HAEi partners with Inceptua for Global Access Program
Pharming Group N.V in association with HAEi announce the appointment of Inceptua Medicines Access as their new distribution partner for the “HAEi Global Access Program” (HAEi GAP) enabling patients in all countries where Pharming’s product […]
18 Sep, 2017
Recommendations for Prophylaxis and Treatment of HAE Attacks
The Food and Drug Administration (FDA) has recently received reports from patients, physicians, and specialty pharmacies that they have been unable to obtain C1-Esterase Inhibitor (Human) Cinryze. Healthcare providers and patients may wish to consider […]
17 Sep, 2017
Diplomat acquires Focus Rx
Diplomat Pharmacy, Inc. has completed its acquisition of Focus Rx Pharmacy Services Inc., a customer-focused healthcare partner that provides home infusion and specialty prescription management services. This acquisition bolsters Diplomat’s offering of extensive solutions to […]
16 Sep, 2017
Portfolio of Oral Plasma Kallikrein Inhibitors Continues to Advance
From the KalVista Pharmaceuticals, Inc. operational and financial results for the fiscal first quarter ended July 31, 2017: “We continue to make progress with our portfolio of oral plasma kallikrein inhibitors in pursuit of […]
11 Sep, 2017
Significant reduction of attack rate
Shire plc announces positive topline Phase 3 results for the Sahara study, a global, multi-center, randomized, double-blind, placebo-controlled, partial crossover trial that evaluated the efficacy and safety of subcutaneously administered C1 esterase inhibitor [human] Liquid […]
11 Sep, 2017
Conclusion of FDA End-of-Phase 2 interactions on Ruconest
Pharming Group N.V. has concluded its End-of-Phase 2 interactions with the U.S. Food and Drug Administration (FDA). As part of these interactions, Pharming provided the FDA with the results of two completed Phase 2 trials […]
7 Sep, 2017
Positive Results from APeX-1 Phase 2 Trial
BioCryst Pharmaceuticals, Inc. has announced final results from its Phase 2 APeX-1 clinical trial in HAE. APeX-1 was a 3-part dose ranging trial designed to evaluate the efficacy, safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of […]
11 Aug, 2017
Berinert Approved by Health Canada
Heath Canada – responsible for helping Canadians maintain and improve their health – has approved Berinert (C1 Esterase Inhibitor, Human) from CSL Behring for pediatric HAE. “HAE is an unpredictable and life threatening disease with […]
10 Aug, 2017
Adverum Reports Second Quarter Financial Results
Adverum Biotechnologies, Inc. has reported financial results for the second quarter ended 30 June 2017 and provided a corporate update. “We are making important progress with our development and regulatory initiatives to transform Adverum into […]
9 Aug, 2017
BioCryst Reports Second Quarter Financial Results
BioCryst Pharmaceuticals, Inc. has announced financial results for the second quarter ended 30 June 2017. Jon P. Stonehouse, President & CEO, comments: “We are excited by the positive results previously reported in Parts 1 and […]
3 Aug, 2017
BioCryst Announces Initiation of ZENITH-1
BioCryst Pharmaceuticals, Inc. has announced the dosing of the first subject into ZENITH-1, a clinical trial studying up to three dosage strengths of a liquid formulation of BCX7353 given as a single oral dose for […]
28 Jul, 2017
KalVista Pharmaceuticals Provides Operational Update
KalVista Pharmaceuticals, Inc. provides an operational update and released financial results for the fiscal fourth quarter and full year ended 30 April 2017. From CEO Andrew Crockett’s remarks: “KalVista has been making substantial advances with our […]
27 Jul, 2017
Pharming Reports on Financial Results for the First Half of 2017
Pharming Group N.V. presents its (unaudited) financial report for the six months ended 30 June 2017. CEO Sijmen de Vries comments: “Our strategic decision to reacquire the commercial rights to sell RUCONEST in North America […]
26 Jul, 2017
The Lancet publicizes RUCONEST® prophylactic data
The weekly peer-reviewed general medical journal The Lancet has published data from a Phase II, double-blind, placebo-controlled, randomized clinical trial (NCT02247739) evaluating the efficacy and safety of RUCONEST (C1 esterase inhibitor [recombinant]) for the prevention […]
22 Jul, 2017
FDA Orphan-Drug Exclusivity for HAEGARDA
The U.S. Food and Drug Administration (FDA) has granted CSL Behring seven years of orphan-drug exclusivity for HAEGARDA (C1 Esterase Inhibitor Subcutaneous [Human]), the first and only subcutaneous treatment option for prevention of HAE attacks. […]
30 Jun, 2017
June magazine out now
Download the HAEi magazine “Global Perspectives” June 2017 here: Global Perspectives – June 2017
27 Jun, 2017
Regional Patient Advocate for sub-Sahara Africa
Early 2016 HAEi appointed five Regional Patient Advocates, dividing a large portion of the world between them. As per 1 July 2017, they are joined by Patricia Karani who will be focusing on the sub-Sahara […]
23 Jun, 2017
FDA approves first subcutaneous C1 Esterase Inhibitor
The U.S. Food and Drug Administration has approved Haegarda, the first C1 Esterase Inhibitor (Human) for subcutaneous (under the skin) administration to prevent HAE attacks in adolescent and adult patients. The subcutaneous route of administration […]
2 Jun, 2017
Thousands helped raise HAE awareness step by step
On and around hae day 🙂 2017 a group of HAE patients, caregivers, doctors and people from HAE organizations walked four stages of the Camino in northern Spain. Many more would have liked to take […]
28 May, 2017
Positive Results From the Second Interim Analysis
BioCryst Pharmaceuticals, Inc. has announced results from a second interim analysis of its Phase 2 APeX-1 clinical trial in HAE. APeX-1 is a 3-part dose ranging trial designed to evaluate the efficacy, safety, tolerability, pharmacokinetics […]
23 May, 2017
Attune Announces Positive Data from Pre-Clinical Studies
Attune Pharmaceuticals announces positive results from preclinical safety studies evaluating ATN-249, a novel orally administered plasma kallikrein inhibitor for the treatment of HAE. The strong safety, high potency, and high selectivity results suggest a […]
19 May, 2017
Lanadelumab Reduces HAE Monthly Attack Rate by 87 %
Shire plc announces positive topline Phase 3 results for the HELP study, a global, multi-center, randomized, double-blind placebo-controlled parallel group trial that evaluated the efficacy and safety of subcutaneously administered lanadelumab versus placebo over 26 […]
16 May, 2017
2018 HAE Global Conference will take place in Vienna, Austria
Following the very successful HAE conferences in Copenhagen, Denmark in 2012, Washington D.C., USA in 2014, and Madrid, Spain in 2016, HAEi is delighted to announce that the fourth HAE Global Conference will be held […]
16 May, 2017
A global awareness day for a rare life-threatening condition
On May the 16th HAE patient organizations from around the world, led by HAEi come together to support the global awareness day for HAE. Dr Sijmen de Vries, Pharming’s CEO commented: “We are proud to […]
11 May, 2017
Adverum Reports First Quarter 2017 Financial Results
Adverum Biotechnologies, Inc. has reported the financial results for the first quarter ended March 31, 2017. “Adverum is well positioned in the gene therapy space with a robust pipeline and a platform of industry-leading AAV […]
5 May, 2017
BioCryst Reports First Quarter 2017 Financial Results
BioCryst Pharmaceuticals, Inc. has announced the financial results for the first quarter ended March 31, 2017. “We have completed enrollment in Part 1 and 2 of the APeX-1 Phase 2 clinical trial of BCX7353 for […]
2 May, 2017
Shire delivers strong Q1 2017 revenue growth
Shire plc has announced the unaudited results for the three months ended March 31, 2017. Flemming Ornskov, Shire CEO, commented: “In the first quarter we delivered strong top-line growth. (…) Our priorities for the rest of […]
1 May, 2017
Raising awareness step by step on the Camino
30 people – most of them suffering from a rare and potentially fatal disease – will meet mid-May in northern Spain in order to walk part of the legendary Camino de Santiago together. The patients […]
13 Apr, 2017
BioCryst Expands Development to Explore Treatment of Acute HAE Attacks
BioCryst Pharmaceuticals, Inc. plans to explore a new oral liquid formulation of BCX7353 for the treatment of acute attacks in patients with HAE. The company has received initial regulatory approvals in Europe to initiate the […]
30 Mar, 2017
Global Perspectives – the HAEi magazine #1/2017 is out now
We are excited to inform you that the first edition of “Global Perspectives” in 2017 – the HAEi magazine – is out now. Please download it here.
25 Mar, 2017
Japan Approves Berinert for Short-Term Prophylaxis
As of 24 March 2017, Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved an extended use of Berinert from CSL Behring, a C1-esterase inhibitor (C1-INH) concentrate, for pre-procedure prevention (short-term prophylaxis) of acute episodes […]
23 Mar, 2017
Pivotal Phase III Data for CSL Behring’s Subcutaneous C1-Esterase Inhibitor
New England Journal of Medicine (NEJM) has published results from the COMPACT study, a pivotal Phase III study evaluating the safety and efficacy of CSL830 (a novel, investigational, self-administered, subcutaneous C1-Esterase Inhibitor [C1-INH] Human replacement […]
22 Mar, 2017
Cooperation and networking among health care professionals worldwide
From 7 to 9 March 2017, CSL Behring in Bern, Switzerland hosted the First Intercontinental Immunoglobulin and Hereditary Angioedema Academy. The conference included presentations from 13 international immunodeficiency and HAE specialists, as well as a […]
16 Mar, 2017
Cinryze first HAE treatment approved for routine prevention in paediatrics
The European Commission has approved a label extension granting three new indications for Cinryze (C1 inhibitor [human]) from Shire plc, broadening its use to children with HAE. Cinryze is now indicated for routine prevention of […]
10 Mar, 2017
Financial report 2016 from Pharming Group
Pharming Group N.V. has presented its (unaudited) financial report for the full year ended 31 December 2016. Sijmen de Vries, CEO and Chairman of the Board of Management, comments: 2016 was a major year for […]
10 Mar, 2017
KalVista appoints Senior Vice President of Medical
KalVista Pharmaceuticals, Inc. has appointed Andreas Maetzel, M.D., M.Sc., Ph.D, to the role of Senior Vice President, Medical. “Andreas Maetzel brings a wealth of experience in HAE and over 25 years of leadership in the […]
8 Mar, 2017
Attune Pharmaceuticals announces pre-clinical data
Attune Pharmaceuticals, a biotechnology company focused on the discovery and development of novel oral small molecule therapeutics for the treatment of rare diseases, has announced the first preclinical data results for ATN-249, a novel orally administered […]
28 Feb, 2017
Positive interim results from APeX-1 trial
BioCryst Pharmaceuticals, Inc. has announced results from an interim analysis of its Phase 2 APeX-1 trial in HAE. APeX-1 is a dose ranging trial designed to evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of orally administered […]
23 Feb, 2017
Phase 1b results for investigational treatment published
The New England Journal of Medicine has just published the results from the Phase 1b study of lanadelumab (SHP643; formerly DX-2930) from Shire plc. Lanadelumab is a subcutaneously administered, human monoclonal antibody that specifically binds and […]
17 Jan, 2017
European Commission amends Marketing Authorisation for Ruconest
Following the positive opinion of the Committee for Medicinal Products for Human Use (CHMP), the European Commission has adopted the Commission Implementing Decision to amend the marketing authorisation for Ruconest to include self administration using the […]
24 Dec, 2016
Global Perspectives – the HAEi magazine #1/2016 is out now
We are excited to inform you that the first edition of “Global Perspectives” – the HAEi magazine – is out now. Please download it here.
23 Nov, 2016
KalVista Closes Merger with Carbylan
KalVista Pharmaceuticals, Inc. has announced the closing of the previously announced merger with Carbylan Therapeutics, Inc. As a result of the completion of this transaction, Carbylan changed its name to KalVista Pharmaceuticals, Inc. KalVista is now […]