NEWS
Pharvaris to advance novel oral HAE medication
Pharvaris has closed its oversubscribed 80 million USD Series C financing bringing its total venture funding to over 160 million USD to date. “Our team is committed to developing and delivering differentiated products to patients [...]
Vast majority of HAE patients value efficacy and specific correction
An online survey conducted the Harris Poll and sponsored by CSL Behring show that a vast majority of HAE patients (94%) say it’s important their preventive therapy specifically corrects C1 esterase inhibitor (C1-INH) deficiency. Both people [...]
New facility for Ruconest production
Pharming Group N.V. will be building a new facility to expand the company’s downstream processing capacity for its lead product, Ruconest (recombinant C1 esterase inhibitor (rhC1INH). The downstream processing facility will include the purification, filtration [...]
Pharvaris expects to advance rapid on-demand HAE treatment
At the presentation of data on the pharmacokinetic (PK), pharmacodynamic (PD), and safety profiles of PHA121 in healthy volunteers Pharvaris expects to advance PHVS416 (PHA121 in soft capsules) to provide a rapid and convenient on-demand HAE [...]
Final results from the Phase 3 HELP Study
Takeda Pharmaceutical Company Limited announces the final results from the Phase 3 HELP Study Open-label Extension (OLE) showing that Takhzyro (lanadelumab-flyo) helped prevent and reduce the frequency of HAE attacks long term in patients 12 years [...]
New data highlight unmet need in HAE medication administration options
BioCryst Pharmaceuticals, Inc. presents data from a cross-sectional study among patients, caregivers and physicians in the US, highlighting the unmet need in prophylaxis medication administration options through the experiences of HAE patients and caregivers, and [...]
Data show attack rate reductions, improved patient satisfaction and quality of life
New clinical data that further evaluates the attack rate reductions, patient satisfaction and quality of life of HAE patients in the APeX-2 trial over 48 weeks is being presented by BioCryst Pharmaceuticals, Inc. “The data [...]
Intellia progressing NTLA-2002 for the treatment of HAE
“We are progressing NTLA-2002 for the treatment of HAE, to a regulatory submission next year”, says President and CEO John Leonard, M.D., at the presentation of Intellia Therapeutics, Inc. operational highlights and financial results for the [...]
One step closer to first-of-its-kind treatment option
Takeda Canada Inc. has completed the negotiations with the pan Canadian Pharmaceutical Alliance (pCPA) with a Letter of Intent (LOI) regarding TAKHZYRO (lanadelumab injection) for routine prevention of HAE attacks in adolescents and adults (12 years [...]
BioCryst ready to launch oral, once-daily medicine
“We are ready to launch ORLADEYO to bring HAE patients the oral, once-daily medicine they have been waiting for to prevent attacks, reduce their burden of therapy and live a normal life,” says Jon Stonehouse, [...]
Advance in the Ionis’ HAE program
In its financial results for the third quarter of 2020 Ionis Pharmaceuticals, Inc. reports the advance in the IONIS-PKK-LRx program: Proof-of-concept data from the PKK development program in patients with HAE were reported in the [...]
Early Access to BioCryst’s Berotralstat in UK
The United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted oral, once-daily berotralstat from BioCryst Pharmaceuticals, Inc. a positive scientific opinion through the Early Access to Medicines Scheme (EAMS). Under the EAMS, HAE [...]
Operational highlights from Pharming
At the presentation of the financial report for the nine months and third quarter ended 30 September 2020 Pharming Group N.V. CEO Sijmen de Vries says: “We are pleased to announce continued growth, demonstrating consistent progress, [...]
Broad Response to COVID-19 and Advancements Across Strategic Scientific Platforms
In its annual R&D briefing to investors CSL Limited demonstrates how the company is advancing a novel research portfolio across four strategic scientific platforms (Plasma Fractionation, Recombinant Technology, Cell and Gene Therapy, Adjuvanted Cell and [...]
Berotralstat significantly reduced HAE attack rates
The Journal of Allergy and Clinical Immunology (JACI) publishes data from the first 24 weeks of the APeX-2 trial of oral, once-daily berotralstat in patients with HAE. APeX-2 was a Phase 3, double-blind, placebo-controlled, parallel-group trial [...]
Antisense medicine being evaluated in clinical study of COVID-19 patients
IONIS-PKK-LRx from Ionis Pharmaceuticals, Inc. is being evaluated in an investigator-initiated Phase 2 clinical study to determine its effectiveness in reducing the severity of respiratory complications in patients with COVID-19. The trial coordinators are Fernando [...]
Update on KalVista’s Oral HAE Franchise
KalVista Pharmaceuticals, Inc. provides an update on its franchise of oral therapies for treatment of HAE. “We have completed enrollment of our Phase 2 trial for our oral on-demand HAE treatment, KVD900, and remain on track [...]
New App from HAEi will be Your Loyal Companion
HAEi has developed HAE Companion an exciting new App that provides an easy way to access and store the popular HAE International emergency card that is currently a paper document. This App will become [...]
José Egidio Fabiani in memoriam
Dr. José Egidio Fabiani, who distinguished himself within HAE in his native Argentina as well as in Latin America, passed away earlier this year. In a eulogy Alejandra Menendez, President of HAE Argentina, writes: [...]
HAEi Advocacy Academy is Your First Step to Successful Advocacy
HAE International remains dedicated to finding innovative ways to serve the HAE community and are happy to announce the new initiative HAEi Advocacy Academy. “HAEi Advocacy Academy – a free online advocacy training platform [...]
Global Network of Accredited Angioedema Care Centers Grows Rapidly
Ten months ago, HAE International and the non-profit organization of leading clinical and research facilities in the field of allergy and asthma GA2LEN established GA2LEN/HAEi Angioedema Centers of Reference and Excellence – or in [...]
News from Caribbean and Central America
From Regional Patient Advocate Javier Santana Like many countries around the world, the Central American and Caribbean region has also been affected by the COVID-19 pandemic. This has allowed many governments to enact lockdown [...]
News from Sub-Sahara Africa
From Regional Patient Advocate Patricia Karani HAE International was able to make collaborations with the Sudanese Society of Clinical Immunology and Allergy (SSCI&A) to conduct the first doctor training seminar on HAE amongst health [...]
News from Nordics & DACH
From Regional Patient Advocate Jørn Schultz-Boysen My name is Jørn Schultz-Boysen, and I am extremely happy to be appointed as the Regional Patient Advocate for the Nordics (covering Denmark, the Faroe Islands, Finland, Greenland, [...]
News from Mediterranean, North Africa & British Isles
From Regional Patient Advocate Maria Ferron The ACARE Center initiative has been presented to all the countries in the region, and it has been well received. We already have ACARE Centers set up in [...]
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