News

Pharming Group has committed to building a new manufacturing facility in the Netherlands to expand production of Ruconest (conestat alfa), its treatment for acute attacks of hereditary angioedema (HAE). Construction will begin in mid-2021. Once finished, it will create at least 40 new jobs, the company said. “With an…

Just a couple of weeks after its U.S. approval, Orladeyo (berotralstat) is now available for direct shipment to hereditary angioedema (HAE) patients in the United States who hold a prescription for the oral medication. According to BioCryst Pharmaceuticals, the maker of Orladeyo, the medicine will be provided exclusively…

Takeda’s Takhzyro (lanadelumab) has been approved in China for the routine prevention of hereditary angioedema (HAE) attacks in patients ages 12 and older, the company announced. It now becomes the “first approved modern therapy” available in the country for this indication, Takeda said in a press release.

PHA121 (PHA-022121), Pharvaris‘ oral therapy for hereditary angioedema (HAE), outperformed Firazyr (icatibant acetate) — an approved therapy with a similar mechanism of action — in a small study of healthy volunteers, the company announced in a press release. That…

The U.S. Food and Drug Administration has approved Orladeyo (berotralstat) as an oral treatment to prevent swelling attacks in people with hereditary angioedema (HAE), ages 12 and older. This approval makes Orladeyo, developed by BioCryst Pharmaceuticals, the first and only oral treatment available for these patients, for whom…

Icatibant injection, a generic form of Firazyr from Fresenius Kabi, is now available to adults in the United States for the treatment of acute attacks of hereditary angioedema (HAE), the company announced. “For people with HAE, an acute attack can occur anywhere, at any time and…

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…

Routine injectable treatments to prevent hereditary angioedema (HAE) attacks place a large burden on patients and their caregivers, a survey study has found. Surveyed patients and doctors also agreed that less burdensome and easier-to-administer preventive treatments are needed to improve the quality of life and independence of those with…

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…