News

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…

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…

Takeda Canada announced reaching an agreement for public funding in Canada of Takhzyro (lanadelumab), its treatment for eligible hereditary angioedema (HAE) patients. The agreement was reached between the company and the pan-Canadian Pharmaceutical Alliance, the agency responsible for setting the prices of brand and generic medications available throughout…

Berotralstat — an oral therapy by BioCryst Pharmaceuticals for preventing swelling attacks in people with hereditary angioedema (HAE) — will be available to patients, 12 and older, in the U.K. through an Early Access to Medicines Scheme (EAMS) before its potential regulatory approval. An EAMS gives patient access to…

Preventive treatment with oral berotralstat (BCX7353) significantly lowers the frequency of swelling attacks in people with hereditary angioedema (HAE), newly published results from the APeX-2 clinical trial demonstrate. The study, “Oral once-daily berotralstat for the prevention of hereditary angioedema attacks: a randomized, double-blind, placebo-controlled phase 3…