Author Archives: Marta Figueiredo PhD

Orladeyo Approved in United Arab Emirates to Prevent HAE Attacks

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 provides an important new treatment option for patients and physicians,” Henrik Balle Boysen, executive vice president and chief operating…

Orladeyo Approval for HAE Under Review in Canada, Switzerland

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 12 and older. The applications — to Heath Canada and Swissmedic — followed Orladeyo’s recent approvals in the…

Early Phase 1 Data Support PHA121 as Oral HAE Prophylactic

Multiple doses of PHA121 (PHA-022121), an investigational oral therapy for hereditary angioedema (HAE), were well-tolerated and rapidly reached therapeutic levels in healthy volunteers, according to data from a Phase 1 clinical trial. The therapy’s pharmacological profile “suggests that the therapeutic effect of PHA121 can be achieved as early…

Oral PHA121 May Lead to Faster, More Durable Effects Than Firazyr

PHA121 (PHA-022121), Pharvaris’ experimental oral therapy for hereditary angioedema (HAE), results in faster, more potent, and more durable suppression of bradykinin-induced changes in blood pressure than injectable Firazyr (icatibant), according to studies in monkeys and healthy volunteers. These findings, along with evidence of PHA121’s favorable pharmacological…

Intellia Seeks to Advance Its Gene-editing Therapy to Human Trials

Intellia Therapeutics is advancing clinical manufacturing activities to support its plans to file regulatory applications for first-in-human clinical trials of NTLA-2002, its experimental gene-editing therapy for hereditary angioedema (HAE). The submissions are anticipated in the second half of the year, and the first trial is expected to evaluate…