News

News 2018-01-23T21:36:09+00:00
20Sep, 2018

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 [...]

14Sep, 2018

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 [...]

6Sep, 2018

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 [...]

6Sep, 2018

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, [...]

26Aug, 2018

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 [...]

24Aug, 2018

US FDA approves TAKHZYRO

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 [...]

7Aug, 2018

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 [...]

3Aug, 2018

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 [...]

27Jul, 2018

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 [...]

26Jul, 2018

APeX-1 Clinical Trial Results for BCX7353

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 [...]

11Jul, 2018

The HAEi Magazine ‘Global Perspectives’ out now

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 [...]

21Jun, 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. [...]

4Jun, 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 [...]

30May, 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) [...]

26May, 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.  

25May, 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 [...]

15May, 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 [...]

8May, 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.  [...]

8May, 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 [...]

18Apr, 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 [...]

15Apr, 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 [...]

14Apr, 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® [...]

13Apr, 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, [...]

12Apr, 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 [...]

11Apr, 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 [...]

10Apr, 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 [...]

9Apr, 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 [...]

8Apr, 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 [...]

31Mar, 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 [...]

30Mar, 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 [...]

29Mar, 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 [...]

16Mar, 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 [...]

16Mar, 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 [...]

16Mar, 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 [...]

15Mar, 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 [...]

7Mar, 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 [...]

7Mar, 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 [...]

28Feb, 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 [...]

28Feb, 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. [...]

28Feb, 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 [...]

27Feb, 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, [...]

23Feb, 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 [...]

23Feb, 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 [...]

20Feb, 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 [...]

16Feb, 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 [...]

24Jan, 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 [...]

22Jan, 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 [...]

19Jan, 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 [...]

11Jan, 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. [...]

6Jan, 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. [...]

6Jan, 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 [...]

5Jan, 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 [...]

4Jan, 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 [...]

3Jan, 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 [...]

2Jan, 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 [...]

1Jan, 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. [...]

22Dec, 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 [...]

15Dec, 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 [...]

7Dec, 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 [...]

28Nov, 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 [...]

10Nov, 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 [...]

31Oct, 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 [...]

27Oct, 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 [...]

27Oct, 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 [...]

26Oct, 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. [...]

2Oct, 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 [...]

28Sep, 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 [...]

27Sep, 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 [...]

26Sep, 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 [...]

18Sep, 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 [...]

17Sep, 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 [...]

16Sep, 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 [...]

11Sep, 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 [...]

11Sep, 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 [...]

7Sep, 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 [...]

11Aug, 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 [...]

10Aug, 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 [...]

9Aug, 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 [...]

3Aug, 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 [...]

28Jul, 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 [...]

27Jul, 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 [...]

26Jul, 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 [...]

22Jul, 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. [...]

30Jun, 2017

June magazine out now

Download  the HAEi magazine "Global Perspectives" June 2017 here: Global Perspectives - June 2017

27Jun, 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 [...]

23Jun, 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 [...]

2Jun, 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 [...]

28May, 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 [...]

23May, 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 [...]

19May, 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 [...]

16May, 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 [...]

16May, 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 [...]

11May, 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 [...]

5May, 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 [...]

2May, 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 [...]

1May, 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 [...]

13Apr, 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 [...]

30Mar, 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.  

25Mar, 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 [...]

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HAEi South Eastern Europe Workshop - Skopje
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Sep 28, 2018 - Sep 29, 2018

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Oct 6, 2018

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Oct 6, 2018

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Oct 6, 2018

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