News

Fewer HAE Attacks Evident With Takhzyro Use in Canadian Study

Use of Takhzyro (lanadelumab) for at least six months led to fewer hereditary angioedema (HAE) attacks and treated attacks, a case review study from Canada reported. Three of its 12 patients went into complete remission, and all enjoyed a more comfortable social life, the investigators noted. The study, by…

‘Rare’ Documentary in Kickstarter Campaign to Raise $45K by Oct. 28

A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.

New Data-sharing Program Aims to Speed Innovation in Rare Diseases

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…

Cycle Launches Sajazir as Treatment Option for Acute HAE Attacks

Cycle Pharmaceuticals has launched Sajazir, an injectable form of icatibant, as a new treatment option for acute attacks of hereditary angioedema (HAE) in adults. Patients who use Sajazir can take advantage of Cycle Vita, the company’s recently created rare disease product support hub. Icatibant, also sold as Firazyr,…

Catabasis, Now Renamed Astria, Working Toward HAE Trial Launch

Astria Therapeutics is the new name of the company that’s developing STAR-0215, an investigational kallikrein-inhibitor previously known as QLS-215, for the treatment of hereditary angioedema (HAE). Formerly called Catabasis Pharmaceuticals, the company said it changed its name to reflect its pledge of having patients serve as its guiding…

Oral Orladeyo Now Available in UK to Treat HAE Swelling Attacks

Orladeyo (berotralstat), a daily oral therapy to prevent swelling attacks in people with hereditary angioedema (HAE), is now available in England, Wales, and Northern Ireland for eligible patients ages 12 and older. That authorization came as the U.K.’s National Institute for Health and Care Excellence (NICE) recommended Orladeyo —…

New Institute Aims to Leave No Rare Disease Patient Behind

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…