HAEi News

EMA approval of new Ruconest facility

2020-01-21T16:18:30+01:00January 21, 2020|HAEi News|

Pharming Group N.V. has received European Medicines Agency (EMA) approval of a Type II Variation for a new production facility for the Ruconest. With the addition of this new facility, Pharming will significantly increase the production capacity of Ruconest as it becomes fully operational over the coming year. Pharming is now also able to release [...]

KalVista updates on oral treatment

2020-01-13T13:48:30+01:00January 13, 2020|HAEi News|

“Based upon the progress of the ongoing KVD900 Phase 2 clinical trial, we expect to announce data from that trial in the second quarter of this year,” says Andrew Crockett, CEO of KalVista Pharmaceuticals, Inc. “We are also pleased to announce the selection of KVD824 for development as an oral prophylactic treatment for HAE. Based [...]

Regenxbio expects selection of lead product candidate in 2020

2020-01-13T08:59:48+01:00January 13, 2020|HAEi News|

As first announced in July 2019, Regenxbio Inc. is developing a one-time gene therapy candidate to deliver a gene encoding a therapeutic antibody against plasma kallikrein, a key protein of the plasma contact pathway which is left unregulated in patients with HAE. Preclinical animal studies conducted using NAV AAV8 indicate the potential for a sustained [...]

Getting ready for the 2020 HAE Global Conference

2020-01-03T13:47:47+01:00December 30, 2019|HAEi News|

The 2020 HAE Global Conference in Frankfurt, Germany is only about five months away, and at HAE International everyone is doing their part to get the largest HAE gathering ever ready. “At this point, all travel grants have been allocated to patients and caregivers from around the globe, and every day we are receiving new registrations for the conference. Momentarily we have [...]

Pharming reacquires Ruconest-licensed territories

2020-01-03T13:58:17+01:00December 30, 2019|HAEi News|

Pharming Group N.V. has entered into a definitive agreement with Swedish Orphan Biovitrum AB (publ) (“Sobi”) to reacquire the commercial rights to Pharming’s product, Ruconest, in all remaining territories, which includes all remaining EU markets. Under the agreement, the license is being terminated with effect from 1 January 2020 in all 36 countries with a [...]

Global Perspectives #4/2019 out now

2019-12-22T13:17:21+01:00December 22, 2019|HAEi News, Newsletters|

With our very best holiday regards we send you the fourth 2019 issue of the HAE International magazine Global Perspectives – your ultimate source of HAE information globally. This time you will, among many other items, find articles on these topics: A new global network of specialized treatment centers becomes a reality News from [...]

BioCryst submits new drug application to FDA

2020-01-03T13:59:14+01:00December 11, 2019|HAEi News|

BioCryst Pharmaceuticals, Inc. has submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for approval of oral, once daily berotralstat (BCX7353) for the prevention of HAE attacks. “HAE patients are waiting for a safe, effective oral therapy to manage their disease and this NDA submission brings berotralstat an important step [...]

A New Global Network of Specialized Treatment Centers Becomes a Reality

2019-12-06T18:21:25+01:00December 6, 2019|HAEi News|

HAE International (HAEi) is excited to announce a new collaboration with GA2LEN— a nonprofit European based organization of leading clinical and research facilities in the field of allergy and asthma—to establish GA2LEN/HAEi ACARE (Angioedema Centers of Reference and Excellence). The joint venture with GA2LEN fulfills HAEi’s longstanding goal of establishing a world-wide network of accredited [...]

KalVista expect Phase 2 clinical trial data in 2020

2019-12-04T08:45:25+01:00December 4, 2019|HAEi News|

“We recently received Fast Track designation for KVD900, illustrating the high level of unmet need in the HAE community for efficacious and safe, orally-delivered therapies,” said Andrew Crockett, CEO of KalVista Pharmaceuticals, Inc. says in an operational update and financial results for the fiscal second quarter ended 31 October 2019. “Our Phase 2 clinical trial for KVD900 [...]