News

News 2017-06-01T17:42:42+00:00
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 [...]

23Mar, 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 [...]

22Mar, 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 [...]

16Mar, 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 [...]

10Mar, 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 [...]

10Mar, 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 [...]

8Mar, 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 [...]

28Feb, 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 [...]

23Feb, 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 [...]

17Jan, 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 [...]

24Dec, 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.  

23Nov, 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 [...]

17Nov, 2016

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 evaluated a volume-reduced, subcutaneous C1-inhibitor preparation, CSL830, for the prevention of HAE attacks. After obtaining [...]

15Nov, 2016

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 presented by Marco Cicardi, Professor of Internal Medicine University of Milan, Hospital L. Sacco Milan [...]

15Nov, 2016

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, Inc. signed on 8 August 2016 for Pharming to acquire the commercialization rights to its [...]

11Nov, 2016

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 a positive opinion recommending an extension to the terms of the marketing authorisation for Ruconest [...]

28Oct, 2016

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: After a relatively modest start to sales of Ruconest® (recombinant C1 esterase inhibitor, 50 IU/kg) [...]

12Sep, 2016

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 readers either a "Magazine View" og a "Single Page View". Feel free to download it [...]

31Aug, 2016

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 subcutaneous (SC) C1-Esterase Inhibitor (C1-INH) Human replacement therapy, CSL830, as prophylaxis to prevent HAE attacks. [...]

11Aug, 2016

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 pharmacodynamics of orally delivered KVD818 in healthy volunteers. A KalVista discovery, KVD818 is a novel, [...]

11Aug, 2016

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 HAE. "We are very pleased that the adaptively-designed APeX-1 trial is now under way, and [...]

9Aug, 2016

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 Ruconest (recombinant human C1 esterase inhibitor), including all rights in the US, Mexico and Canada, from [...]

5Aug, 2016

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 make progress, and have initiated subject screening to start the APeX-1 trial evaluating our once [...]

19Jul, 2016

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 HAE attacks, for use in pediatric patients. This expands the use of Berinert into all age groups, [...]

18Jul, 2016

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) for prophylaxis in patients with HAE. In the study, Ruconest showed a clinically relevant and [...]

15Jul, 2016

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 to Austria, Germany and Netherlands, Pharming will market Ruconest directly into an additional 21 countries, [...]

22Jun, 2016

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 readers either a "Magazine View" og a "Single Page View". Feel free to download it [...]

16Jun, 2016

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 of KalVista will become the majority owners of Carbylan. David Renzi, President and CEO of [...]

2Jun, 2016

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 part in the Camino Walk but were for one reason or the other unable to [...]

26May, 2016

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 of the legendary Camino de Santiago in northern Spain together. Over three days they took [...]

19May, 2016

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: "KalVista is delighted to support the HAE Global Conference, which aligns with our mission to [...]

19May, 2016

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 sales of Ruconest in the countries which Pharming now commercializes directly, in Germany, Austria and the [...]

17May, 2016

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 by HAEi, the international patient organization for C1-inhibitor deficiency, along with the support of its national [...]

17May, 2016

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 to raise awareness around HAE. hae day :-) is coordinated by the HAEi, the International Patient [...]

6May, 2016

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 in HAE patients and are targeting the end of the year to report results," said Jon [...]

1May, 2016

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 readers either a "Magazine View" og a "Single Page View". Feel free to download it [...]

27Apr, 2016

Join the global HAE Walk

  Quite a number of people have told HAEi that they would really have liked to be with us in northern Spain but are unable for one reason or the other. Therefore we have arranged [...]

22Apr, 2016

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 Spain in order to walk part of the legendary Camino de Santiago together. The patients [...]

8Apr, 2016

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 to remove the requirements for rabbit IgE testing that formed part of the EU label [...]

30Mar, 2016

Shire 2015 Annual Report

HAE related information from the Shire plc 2015 Annual Report: The $6 billion acquisition of Dyax expands and extends our industry-leading portfolio in HAE. With Dyax we bring into our portfolio DX-2930 (now SHP643). If [...]

30Mar, 2016

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 needs, has reported business progress and financial results for the fourth quarter and year ended December [...]

24Mar, 2016

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 in Canada with an approved indication for routine prevention against angioedema attacks in adolescent and adult patients [...]

12Mar, 2016

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 will bring together HAE patients, relatives, caregivers, doctors, nurses, and industry for a mutual experience: [...]

10Mar, 2016

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 Board of Pharming Group N.V. writes (in extracts): 2015 was the year that Pharming started to [...]

9Mar, 2016

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 that inhibits the production of Factor XII (F12), has the potential to treat HAE and to [...]

9Mar, 2016

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 RNAi therapeutic targeting F12 for the treatment of HAE. Pre-clinical data for ALN-F12 were presented [...]

4Mar, 2016

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 Majesty the King of Spain that Her Majesty the Queen has agreed to accept the [...]

4Mar, 2016

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 debilitating disease, has reported financial results for the fourth quarter and year ended December 31, 2015. “We [...]

2Mar, 2016

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 Serbia. Ivana Golubović was appointed president, Jovana Cvetković vice-president, and Dušan Dašić board member - and now the organization [...]

1Mar, 2016

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 well over 400 delegates signed up for the conference taking place in Madrid, Spain 19-22 [...]

1Mar, 2016

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 initiative committed to improving public understanding of rare diseases and highlighting the exceptional challenges facing [...]

27Feb, 2016

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) has issued a positive opinion to the European Commission on the company's request to include the [...]

24Feb, 2016

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 on both of our HAE drug candidates. Our efforts are now focused on completing the [...]

12Feb, 2016

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 advanced our strategy to become a leading global biotechnology company focused on rare diseases and [...]

11Feb, 2016

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 temporal link between the emergence of the virus in Brazil and the birth of babies [...]

10Feb, 2016

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 the lives of children born with rare diseases, as well as the future of rare [...]

9Feb, 2016

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 company, for the distribution of Ruconest® (recombinant, non-blood derived human C1 inhibitor) for the treatment [...]

8Feb, 2016

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 for the prophylactic treatment of HAE attacks. In the OPuS-2 study, HAE patients with a [...]

2Feb, 2016

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 shares of Annapurna by Avalanche. Upon completion of the proposed acquisition, the combined company’s pipeline [...]

26Jan, 2016

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 Camino in the days leading up to the HAE Global Conference in Madrid in May [...]

24Jan, 2016

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 are excited to complete the acquisition of Dyax and look forward to working alongside their [...]

12Jan, 2016

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 Baxalta. Shire CEO Flemming Ornskov, M.D., M.P.H., commented: “This proposed combination allows us to realize our vision [...]

6Jan, 2016

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), for prophylaxis in patients with HAE. Thirty HAE patients deficient in C1 esterase Inhibitor and [...]

21Dec, 2015

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 Korea. Ruconest is approved for the treatment of acute angioedema attacks in adult patients with [...]

15Dec, 2015

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 planned Phase 3 programs for 2016 include SHP-616 (Cinryze) for subcutaneous delivery for HAE prophylaxis, SHP-616 [...]

10Dec, 2015

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 in Russia where the healthcare system has some unmet needs for state-of-the-art biotherapies and blood [...]

7Dec, 2015

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 recombinant (non-blood-derived) C1-esterase inhibitor replacement therapy, was first approved by the EMA in June 2010 [...]

26Nov, 2015

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 readers either a "Magazine View" og a "Single Page View". Feel free to download it [...]

10Nov, 2015

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 Allergy, Asthma, and Immunology (ACAAI) Annual Meeting. DX-2930 is an investigational fully human monoclonal antibody [...]

8Nov, 2015

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, President & CEO, said: “The completion of enrollment of our OPuS-2 trial of avoralstat and [...]

3Nov, 2015

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 the treatment of HAE. Dyax has already successfully developed and commercialized Kalbitor, which is approved [...]

28Oct, 2015

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 Vries comments (in extract): "During the third quarter, the number of prescriptions for our lead [...]

28Oct, 2015

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 readers either a "Magazine View" og a "Single Page View". Feel free to download it [...]

16Oct, 2015

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]) for intravenous administration in subjects with Antibody Mediated Rejection (AMR) in renal transplant recipients. Cinryze [...]

9Oct, 2015

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 met all of its objectives. The safety, tolerability, drug exposure and on-target plasma kallikrein inhibition [...]

9Oct, 2015

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 a 12-week, three-arm, parallel cohort trial designed to evaluate the efficacy and safety of two [...]

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