Author Archives: Marta Figueiredo PhD

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…

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…

Orladeyo (berotralstat), an oral therapy to prevent hereditary angioedema (HAE) attacks, is now available to patients, 12 and older, in Germany. “We have an experienced team in place in Germany that is excited and honored to bring the first oral, once daily therapy to HAE patients in Europe,”…

The European Commission has approved BioCryst Pharmaceuticals’ Orladeyo (berotralstat) as the first oral, daily therapy to prevent swelling attacks in adults and adolescents, ages 12 and older, with hereditary angioedema (HAE). An oral preventive therapy should be easier to take than standard under-the-skin and into-the-vein alternatives, and as…

Haegarda, CSL Behring’s preventive treatment for hereditary angioedema (HAE) attacks, will be available to eligible HAE patients across Canada starting on April 6. The availability announcement was issued by Canadian Blood Services (CBS), the country’s blood authority, which is responsible for managing the blood supply in all…

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…

BioCryst Pharmaceuticals’ Orladeyo (berotralstat) has been recommended for approval in the European Union as the first oral therapy for preventing swelling attacks in people with hereditary angioedema (HAE), ages 12 and older. The recommendation was made by the Committee for Medicinal Products for Human Use (CHMP), an arm…

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…

Takeda’s Takhzyro (lanadelumab) continues to safely prevent and reduce the frequency of swelling attacks in people with hereditary angioedema (HAE), according to 2.5 years of data from the HELP open-label extension (OLE) trial. The study’s final results were presented in two company-sponsored posters at the 2020…

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…