The U.S. Food and Drug Administration (FDA) has approved an expanded indication for HAEGARDA for routine prophylaxis to prevent HAE attacks in patients 6 years of age and older. HAEGARDA, the HAE therapy that reduces attacks by a median of 95%, is now the first and only subcutaneous treatment option for prevention of HAE attacks [...]
Bridge Medicines and The Rockefeller University has signed an exclusive license agreement to further develop a series of small molecule inhibitors of activated Factor XII (FXIIa) discovered in the laboratory of Jan L. Breslow, M.D. Breslow and his colleague Manish Ponda, M.D., illuminated a novel role for FXIIa as a key target for HAE and [...]
At the presentation of the operational update and released financial results for the first fiscal quarter ended 31 July 2020 KalVista Pharmaceuticals, Inc. CEO Andrew Crockett says: “We are pleased that our KVD900 Phase 2 trial has met its enrollment target and data is expected before the end of this year. We believe KVD900 can bring the [...]
Ionis Pharmaceuticals, Inc. announces the publication of the results from a compassionate-use study evaluating IONIS-PKKRx and IONIS-PKK-LRx in patients living with severe bradykinin-mediated angioedema in The New England Journal of Medicine(NEJM). IONIS-PKKRx and IONIS-PKK-LRx are investigational antisense medicines designed to reduce the production of prekallikrein (PKK), which plays a key role in the activation of [...]
Pharming Group N.V. announces the publication of data in the peer-reviewed journal, Frontiers in Immunology, from a compassionate use programme of five patients with confirmed COVID-19 (SARS-CoV-2) infections hospitalized with related severe pneumonia that were treated with Ruconest (recombinant human C1 inhibitor, conestat alfa) at the University Hospital Basel, Switzerland. As reported on 21 April [...]
The first patient has been enrolled in a randomized, controlled, investigator-initiated clinical trial in up to 150 patients for the treatment with Ruconest (recombinant human C1 inhibitor) of patients with confirmed COVID-19 (SARS-CoV-2) infections hospitalised with related severe pneumonia at the University Hospital Basel in Basel, Switzerland. In April 2020, Pharming Group N.V. reported encouraging results [...]
From Intellia Therapeutics, Inc.’s presentation of the financial results for the second quarter of 2020: NTLA-2002 is a wholly owned, in vivo development candidate for the treatment of HAE. Building on Intellia’s modular lipid nanoparticle (LNP) delivery system, NTLA-2002 is designed to knock out the prekallikrein B1 (KLKB1) gene in the liver after a single [...]
“We are currently in an exciting transformation from a company primarily focused on R&D to one that is about to launch its first oral drug to patients with HAE (ORLADEYO) this year. We expect to end the year with ORLADEYO approved in the U.S. and Japan”, says President and CEO Jon Stonehouse, BioCryst Pharmaceuticals, Inc., [...]
Together with the COVID R&D Alliance partners AbbVie, Inc. and Amgen Inc., Takeda Pharmaceutical Co. Ltd. has enrolled the first patients in the I-SPY COVID Trial (Investigation of Serial Studies to Predict Your COVID Therapeutic Response with Biomarker Integration and Adaptive Learning) clinical trial. The trial will evaluate the efficacy of a chemokine dual-receptor antagonist, a PDE4 [...]
Cipla Limited has received final approval for its Abbreviated New Drug Application (ANDA) for Icatibant Injectable 30mg/3mL from the United States Food and Drug Administration (US FDA). Cipla’s Icatibant Injectable Pre-Filled Syringe 30mg/3mL is AP-rated generic version of Shire’s Firazyr. Icatibant injection is indicated for the treatment of acute attacks of HAE in adults 18 years [...]
