From its Phase 1 multiple-ascending-dose study demonstrating PHA121’s pharmacokinetics and tolerability Pharvaris announces that PHA121 was well tolerated at all doses studied, with approximately dose-proportional exposure. “We are encouraged by these results to continue development of PHA121 as an oral treatment for HAE,” says Berndt Modig, CEO and co-founder of Pharvaris. “In 2021, we will [...]
The Ministry of Health, Labor and Welfare (MHLW) in Japan has granted marketing and manufacturing approval for BioCryst Pharmaceuticals, Inc.’s oral, once-daily Orladeyo (berotralstat) 150 mg for prophylactic treatment of HAE in adults and pediatric patients 12 years and older. Orladeyo is the first and only prophylactic HAE medication approved in Japan. One capsule of [...]
A recent Harris Poll, commissioned by CSL Behring, asked patients who have HAE to assess how the unpredictable condition affects them. In the survey, patients also gave their thoughts on treating the condition, which causes dangerous swelling episodes. Here are some highlights from the survey, which also included responses from physicians: More than 40% of [...]
Intellia Therapeutics, Inc. outlines its expected 2021 milestones and strategic priorities regarding HAE therapy: “Since our founding, we set out to develop modular platform components that could serve as the engine powering an expansive portfolio of curative therapeutics. We have paved a rapid and reproducible development path for both in vivo and engineered cell therapies [...]
BioCryst Pharmaceuticals, Inc. announces that oral, once-daily Orladeyo (berotralstat) is now available for shipment to patients with a prescription in the United States. Orladeyo was approved by the U.S. Food and Drug Administration (FDA) on 3 December 2020 for prophylaxis to prevent attacks of HAE in adults and pediatric patients 12 years and older. Optime Care, Inc., the exclusive [...]
In the operational update and financial results for the second fiscal quarter ended 31 October 2020 CEO Andrew Crockett of KalVista Pharmaceuticals, Inc. says: “We have completed the patient treatment phase of our KVD900 Phase 2 trial and are in the process of wrapping up that study. Data from this trial evaluating KVD900 as an [...]
The first patient has been enrolled in a randomised, open label, parallel group, controlled, pilot clinical trial in up to 120 patients hospitalized with confirmed COVID-19 treated with Ruconest (recombinant human C1 inhibitor) for the prevention of severe SARS-CoV-2 infections at the Valley Hospital in Ridgewood, New Jersey in the United States. Initially based at [...]
China’s National Medical Products Administration (NMPA) has approved Takhzyro (lanadelumab) subcutaneous injection for prophylaxis to prevent attacks of HAE in patients 12 years and older. Takhzyro from Takeda Pharmaceutical Company Limited is a fully human monoclonal antibody (mAb) that inhibits the activity of plasma kallikrein, an enzyme which is uncontrolled in people with HAE, to help [...]
The U.S. Food and Drug Administration (FDA) has approved oral, once-daily Orladeyo (berotralstat) for prophylaxis to prevent attacks of HAE in adults and pediatric patients 12 years and older. “Orladeyo offers people with HAE and their physicians the first orally administered non-steroidal option for preventing HAE attacks and represents an important and welcome step in [...]
The journal Allergy has published data from the APeX-J trial, a randomized, placebo-controlled trial conducted in Japan evaluating oral, once-daily berotralstat for the prophylactic treatment of HAE. The APeX-J trial met its primary endpoint of a reduction in the rate of HAE attacks for berotralstat 150 mg compared to placebo during the 24-week period (p=0.003). [...]
