Angioedema News

September 16, 2021September 16, 2021

New Institute Aims to Leave No Rare Disease Patient Behind

Angioedema News

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest ... Read more

September 16, 2021September 16, 2021

FDA Lifts Hold, Allows Phase 2 Trial of KVD824 for HAE to Open

Angioedema News

The U.S. Food and Drug Administration (FDA) has lifted the hold it had placed on KalVista Pharmaceuticals’ Phase 2 KOMPLETE trial of KVD824 as a treatment to prevent swelling attacks in people ... Read more

September 13, 2021September 13, 2021

Orladeyo Approved in United Arab Emirates to Prevent HAE Attacks

Angioedema News

The United Arab Emirates (UAE) has approved BioCryst Pharmaceuticals’ Orladeyo (berotralstat) for preventing hereditary angioedema (HAE) attacks in patients, 12 and older. “As the first targeted oral, once-daily treatment, Orladeyo ... Read more

September 9, 2021September 9, 2021

Group Focuses on Rare Disease Clinical Trial Participation

Angioedema News

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running ... Read more

September 3, 2021September 3, 2021

‘Running for Rare’: NORD a Charity Partner of 2021 NYC Marathon

Angioedema News

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both ... Read more

September 2, 2021September 2, 2021

HAE Preventive Therapy Not Cost-effective, Prices Need to Drop 50%

Angioedema News

The prices of three approved preventive treatments — Cinryze, Haegarda, and Takhzyro (lanadelumab) — for hereditary angioedema (HAE) must be reduced by more than half for these medications to be cost-effective. ... Read more

August 30, 2021August 30, 2021

Orladeyo Approval for HAE Under Review in Canada, Switzerland

Angioedema News

Regulatory health agencies in Canada and Switzerland are reviewing BioCryst Pharmaceuticals’ applications for the approval of Orladeyo (berotralstat) in their countries for preventing hereditary angioedema (HAE) attacks in patients ages ... Read more

August 26, 2021August 26, 2021

Phase 2 Trial of Oral KVD824 in Preventing HAE Attacks to Open

Angioedema News

KalVista Pharmaceuticals announced plans to soon begin enrolling patients in KOMPLETE, a Phase 2 clinical trial testing its investigational oral therapy KVD824 in preventing swelling attacks in people with hereditary angioedema (HAE) at ... Read more

August 23, 2021August 23, 2021

‘Not Alone in the Dark’: New Guide for, by Caregivers of Children With Rare Diseases

Angioedema News

The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The ... Read more

August 19, 2021August 19, 2021

Potential Blood Biomarkers for Types of Angioedema Noted in Study

Angioedema News

Higher blood levels of molecules involved in blood vessel formation and permeability — Tie-2, Ang-2, and VE-Cadherin — along with sE-selectin, which is involved in vascular inflammation, may help to ... Read more

August 16, 2021August 16, 2021

Register Now for Global Genes’ RARE Patient Advocacy Summit

Angioedema News

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending ... Read more

August 12, 2021August 12, 2021

Takhzyro With Long-term Use Safely Prevent HAE Attacks, Study Finds

Angioedema News

Takhzyro (lanadelumab) safely and markedly reduced the frequency of swelling attacks in people with hereditary angioedema (HAE) over 2.5 years, according to results of the HELP open-label extension (OLE) study. ... Read more

August 9, 2021August 9, 2021

NORD Rare Disease Summit, Online Oct. 18-19, Open for Registration

Angioedema News, COVID-19 News

Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for ... Read more

August 6, 2021August 6, 2021

Advocates Lobby US Congress During Virtual Rare Disease Week

Angioedema News, COVID-19 News

More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the ... Read more

August 5, 2021August 5, 2021

FDA Approves Combo Kit for Easier At-home Use of Berinert

Angioedema News

The U.S. Food and Drug Administration (FDA) has approved a new convenience administration kit for people using Berinert, an at-home infusion treatment of acute hereditary angioedema (HAE) attacks in children ... Read more