Keep yourself updated with HAEi news and events
HAEi News Archive
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. [...]
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 [...]
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 [...]
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 [...]
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 [...]
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 [...]
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 [...]
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: [...]
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 [...]
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 [...]
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 [...]
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 [...]
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 [...]
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. [...]
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, [...]
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, [...]
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 [...]
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 [...]
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 [...]
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) [...]
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 [...]
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 [...]
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 [...]
A Randomized Trial of Subcutaneous C1-INH for HAE Attack Prevention
Current options for prophylaxis of acute HAE attacks are very limited. The international, prospective, double-blind, crossover, phase III COMPACT trial [...]
Results of phase II study for prophylaxis of HAE attacks
The results of a “Randomized, Double-Blind, Placebo-Controlled Trial of Recombinant Human C1 Inhibitor for Prophylaxis of Hereditary Angioedema Attacks” has been [...]
Pharming announces progress on financing
Pharming Group N.V. has made significant progress with financing towards completion of the transaction with subsidiaries of Valeant Pharmaceuticals International, [...]
CHMP adopts positive opinion to include self-administration for Ruconest
The Committee for Medicinal Products for Human Use (CHMP), an advisory committee of the European Medicine Agency (EMA), has adopted [...]
Financial report from Pharming
Pharming Group N.V. presents its financial report for the nine months ended 30 September 2016. From CEO Sijmen de Vries' comments: [...]
HAEi Newsletter #4/2016 out now
We are excited to inform you that the fourth edition of the 2016 newsletter is out now. Again, the newsletter offers our [...]
FDA Accepts Application for Subcutaneous Prophylactic Therapy
The U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for CSL Behring's low-volume [...]
KalVista Initiates Phase 1 Clinical Trial for HAE Treatment
KalVista Pharmaceuticals announces the dosing of the first subject in a first-in-human clinical trial to evaluate the safety, pharmacokinetics and [...]
BioCryst Initiates APeX-1 Clinical Trial of BCX7353 for HAE
BioCryst Pharmaceuticals, Inc. has dosed the first subject in the APeX-1 clinical trial of BCX7353 for the oral treatment of [...]
Pharming acquire all North American commercialization rights to Ruconest
Pharming Group N.V. has entered into a definitive agreement to acquire all North American commercialization rights to its own product [...]
BioCryst continues to make progress
Announcing the BioCryst Pharmaceuticals financial results for the second quarter of 2016 Jon P. Stonehouse, President & CEO, said: "We continue to [...]
U.S. FDA Approves Berinert as the First and Only Pediatric Treatment
The U.S. Food and Drug Administration (FDA) has approved the use of Berinert [C1 Esterase Inhibitor (Human)], the CSL Behring therapy for treating [...]
Pharming Announces Positive Results from Trial with Ruconest
Pharming Group N.V. has announced positive results from a Phase 2 clinical study of Ruconest (recombinant C1 esterase inhibitor, 50 IU/kg) [...]
Pharming amends Ruconest Distribution Agreement with Sobi
Pharming Group NV and Swedish Orphan Biovitrum AB (Sobi) has signed an amendment of their Ruconest distribution agreement from 2009. In addition [...]
HAEi Newsletter #3/2016 out now
We are excited to inform you that the third edition of the 2016 newsletter is out now. Again, the newsletter offers our [...]
Carbylan and KalVista Enter into Share Purchase Agreement
Carbylan Therapeutics, Inc. and KalVista Pharmaceuticals Ltd. have entered into a definitive share purchase agreement pursuant to which the shareholders [...]
12,000,000+ steps for HAE
Before hae day :-) 2016 quite a number of people told HAEi that they would really have liked to take [...]
80 people walked the Camino for HAE
From 15 to 17 May 2016 almost 80 people – the majority of them suffering from HAE – walked part [...]
KalVista to Support Global HAE Conference
KalVista Pharmaceuticals supports the third HAE Global Conference in Madrid, Spain, from 19-22 May 2016. Andrew Crockett, KalVista’s CEO, said: [...]
Change to Pharming strategy now bearing fruit
From the Pharming Group N.V. financial report for the first quarter ended 31 March 2016: During the quarter we initiated [...]
CSL Behring Commemorates 2016 hae day :-)
CSL Behring joins the global HAE community today in recognition of the fifth annual hae day :-), an initiative led [...]
Pharming Group Supports HAE DAY :-) 2016
Pharming Group N.V. supports the Fifth Annual International Hereditary Angioedema Day, hae day :-) taking place on 16 May 2016 and aiming [...]
Working through the start-up activities
"We are currently working through the start-up activities for the APeX-1 trial of BCX7353 for prevention of angioedema attacks [...]
HAEi Newsletter #2/2016 out now
We are excited to inform you that the second edition of the 2016 newsletter is out now. Again, the newsletter offers our [...]
Join the global HAE Walk
Quite a number of people have told HAEi that they would really have liked to be with us in [...]
Raising awareness step by step on the Camino
80 people – most of them suffering from a rare and potentially fatal disease – will meet mid-May in northern [...]
Change in Marketing Authorization Terms For Ruconest
The European Commission has adopted the CHMP recommendation to include the treatment of HAE attacks in adolescents with HAE and [...]
Shire 2015 Annual Report
HAE related information from the Shire plc 2015 Annual Report: The $6 billion acquisition of Dyax expands and extends our [...]
Global Blood Therapeutics Reports Recent Business Progress
Global Blood Therapeutics, Inc., a biopharmaceutical company developing novel therapeutics for the treatment of grievous blood-based disorders with significant unmet [...]
Cinryze now available in Canada
Cinryze is now available in Canada to patients with HAE who may benefit from routine prevention. It is the first and only treatment [...]
80 people to walk the Camino for HAE
For the 2016 version of the awareness day hae day :-) the global organization HAEi and the Spanish HAE organization AEDAF [...]
Pharming’s Report on Preliminary Financial Results 2015
In the financial report for the full year that ended 31 December 2015 Sijmen de Vries, CEO and Chairman of the [...]
Arrowhead Presents Preclinical Data on Possible New HAE Medication
Arrowhead Research Corporation, a biopharmaceutical company developing targeted RNAi therapeutics, has presented additional preclinical data suggesting that ARC-F12, an RNAi therapeutic [...]
Alnylam announcing addition of therapeutic for HAE treatment
Alnylam Pharmaceuticals, Inc. is announcing the addition of a new program to the company’s genetic medicines pipeline, ALN-F12, an investigational [...]
Queen Letizia Accepts Presidency of Honor for HAE Global Conference
It is with the greatest of pleasure that HAEi and AEDAF have received confirmation from the Royal Household of His [...]
Avalanche Biotechnologies Reports Fourth Quarter and Fiscal 2015 Financial Results
Avalanche Biotechnologies, Inc., a gene therapy company committed to discovering and developing novel medicines for patients suffering from chronic or [...]
HAE organization established in Serbia
Over the last few days 21 out of 33 known Serbian HAE families have attended the founding assembly of HAE [...]
More than 400 signed up for HAE Global Conference
We are more than two months away from the opening of the HAE Global Conference 2016 but already we have [...]
Pharming Supports Rare Disease Day
Pharming Group N.V. reiterates it commitment to the treatment of rare diseases on Rare Disease Day 2016, an annual global [...]
Committee for Medicinal Products for Human Use issues positive opinion to European Commission
Following evaluation of a dossier submitted by Pharming Group N.V. last year, the Committee for Medicinal Products for Human Use (CHMP) [...]
BioCryst Reports Full Year 2015 Financial Results
BioCryst Pharmaceuticals, Inc. has announced financial results for the fourth quarter and full year ended December 31, 2015. "In 2015, we gained clarity [...]
Shire announces full year 2015 results
As Shire announces unaudited results for the year to 31 December 2015, Flemming Ornskov, M.D. CEO, comments: "In 2015, we significantly [...]
Zika Virus and Plasma Protein Therapies
Recent scientific and public press reports have heightened awareness of the Zika virus and its emergence in the Americas. The [...]
Shire Marks Anniversary with Global Childrens’ Program
To mark the company’s founding 30 years ago this year, Shire plc is launching a new initiative designed to positively affect [...]
Pharming And Cytobioteck Announce Extension Of Distribution Agreement For Ruconest
Pharming Group N.V. has extended the exclusive distribution agreement with Cytobioteck S.A.S., a privately owned Bogota, Colombia based specialty healthcare [...]
BioCryst Announces Results From OPuS-2
BioCryst Pharmaceuticals, Inc. has announced results from OPuS-2 (Oral ProphylaxiS-2), a clinical trial of avoralstat administered three times daily as a liquid-filled soft gel formulation [...]
Avalanche and Annapurna Announce Proposed Merger
Avalanche Biotechnologies, Inc. and Annapurna Therapeutics SAS have entered into a definitive agreement providing for the acquisition of all outstanding [...]
Come join us in Spain
As you might have noticed in previous information from HAEi, we will be walking part of the pilgrimage route El [...]
Shire Completes Acquisition of Dyax
Shire plc has completed its acquisition of Dyax Corp. in an all-cash transaction. Shire’s CEO, Flemming Ornskov, MD, MPH, commented: “We [...]
Shire to combine with Baxalta, creating the global leader in rare diseases
The boards of directors of Shire plc and Baxalta Incorporated have reached an agreement under which Shire will combine with [...]
Completion Of Patient Enrolment In Clinical Study Of Ruconest
Pharming Group N.V. has completed the patient enrolment in the Phase 2 clinical study of Ruconest, (recombinant C1 esterase Inhibitor, 50 IU/kg), [...]
South Korean Marketing Authorization For Ruconest
Pharming Group N.V. and it's Seoul based South Korean specialty pharma partner Hyupjin have received the marketing authorization for Ruconest in South [...]
Shire goes to Phase 3 in 2016
In 2016, the Shire plc pipeline will include more than 10 programs in Phase 3 trials and compounds already under regulatory review. Shire’s [...]
CSL Behring Expands Operations to Russia
CSL Behring is opening operations in Russia in order to provide more patients with greater access to treatment. This is particularly significant [...]
EMA Reaffirms Important Health Benefits And Safety Of Ruconest
The European Medicines Agency (EMA) has recently renewed the marketing authorization for Ruconest for an unlimited period. Ruconest, the first and only [...]
Newsletter #7/2015 out now
We are excited to inform you that the seventh edition of the 2015 newsletter is out today. Again, the newsletter offers our [...]
Dyax Corp. presents clinical data from DX-2930 Phase 1b study
Dyax Corp. presented two oral presentations describing clinical data from its DX-2930 Phase 1b study at the American College of [...]
BioCryst Reports Third Quarter 2015 Financial Results
At the presentation of the BioCryst Pharmaceuticals, Inc. financial results for the third quarter of 2015 Jon P. Stonehouse, [...]
Shire to acquire Dyax, extending industry-leading HAE portfolio
Shire will acquire Dyax Corp., the publicly traded biotechnology company primarily focused on the development of plasma kallikrein (pKal) inhibitors for [...]
More patients are using Ruconest®
Pharming Group N.V. has presented its unaudited financial report for the first nine months ended 30 September 2015. CEO Sijmen De [...]
Newsletter #6/2015 out now
We are excited to inform you that the sixth edition of the 2015 newsletter is out today. Again, the newsletter offers our [...]
Cinryze Receives FDA Fast Track Designation
The United States Food and Drug Administration (FDA) has granted Fast Track designation for the investigation of Cinryze® (C1 esterase inhibitor [human]) [...]
Biocryst Announces Successful Phase 1 Clinical Trial Of BCX7353
BioCryst Pharmaceuticals, Inc. has announced that the randomized, placebo-controlled, Phase 1 clinical trial of orally-administered BCX7353 in healthy volunteers successfully [...]
BioCryst Announces Completion of Patient Enrollment
BioCryst Pharmaceuticals, Inc. has completed enrollment in OPuS-2 (Oral ProphylaxiS-2), a blinded, randomized, placebo-controlled clinical trial of orally-administered avoralstat in patients with HAE. OPuS-2 is [...]
FDA Grants Ruconest Twelve-Year Reference Product Exclusivity
The U.S. Food and Drug Administration (FDA) has granted 12 years of exclusivity to Ruconest® (C1 esterase inhibitor [recombinant]) 50 [...]
Dyax Corp. Receives Positive Opinion in the European Union
The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending DX-2930 for designation as an [...]
Sanj K. Patel Appointed to BioCryst’s Board of Directors
BioCryst Pharmaceuticals, Inc. has elected Sanj K. Patel to the Company's Board of Directors. Mr. Patel has a proven track record of successfully developing [...]
Rare disorders process yields first funding decision
People with HAE are the first to benefit from the increased competition for rare disorders medicines promoted by Pharmac, the [...]
Newsletter #5/2015 out now
We are excited to inform you that the fifth edition of the 2015 newsletter is out today. The newsletter comes in a [...]
Shire to attain enhanced Cinryze manufacturing flexibility and capacity
Shire plc has entered into an agreement with Sanquin Blood Supply, the manufacturer of Cinryze® (C1 esterase inhibitor [human]), providing Shire [...]
Prometic reports its second quarter 2015 highlights and financial results
From the Prometic report on the company's second quarter 2015 highlights and financial results: "With our cash position combined with proceeds [...]
Dyax provides update on DX-2930 clinical and commercial supply initiatives
Dyax Corp. has provided an update regarding its ongoing manufacturing initiatives for DX-2930. Discovered by Dyax, DX-2930 is an investigational fully [...]
BioCryst reports second quarter 2015 financial results
BioCryst Pharmaceuticals, Inc. has announced financial results for the second quarter ended 30 June 2015. “During the second quarter, we [...]
Dyax Corp. Announces Second Quarter 2015 Financial Results
Dyax Corp. has announced financial results for the second quarter ended June 30, 2015. Recent highlights include: Receipt of Breakthrough Therapy [...]
Pharming reports on financial results first half year 2015
Biotech company Pharming Group N.V. has published its (unaudited) financial report for the six months ended 30 June 2015. From [...]
Unique patient organization-driven program
The global collaboration between Clinigen Group plc and Pharming Group N.V. to provide access to Pharming’s Ruconest (conestat alfa) is [...]
Upcoming Global HAE Events
May 30, 2017 at 5:00 pm - 8:00 pm
Jun 6, 2017 - Jun 9, 2017 at 5:00 pm - 7:00 pm
Jun 17, 2017 at 12:00 am
Aug 3, 2017 - Aug 6, 2017 at All Day
Sep 29, 2017 - Oct 1, 2017 at All Day
Sep 30, 2017 at All Day
Oct 7, 2017 at All Day
Oct 22, 2017 at All Day
Nov 10, 2017 - Nov 12, 2017 at All Day
Nov 18, 2017 at All Day