Patricia Inácio, PhD, science writer —

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

BioCryst Pharmaceuticals is seeking the approval of Orladeyo (berotralstat) in the United Kingdom as a preventive treatment for sudden and recurrent swelling attacks in people with hereditary angioedema (HAE), ages 12 and older. If granted, Orladeyo would become the first oral therapy — found in trials to be…

Subcutaneous (under-the-skin) administration of human plasma-derived C1 inhibitor is an effective and safe option for managing hereditary angioedema during pregnancy and lactation, a case report suggests. The study, “Subcutaneous C1‐Inhibitor Concentrate for prophylaxis during pregnancy and lactation in a patient with C1‐INH‐HAE,” was published in the…

PHA121, an investigational oral therapy for hereditary angioedema attacks, was well tolerated across multiple doses in a group of healthy volunteers in a Phase 1 trial. Study data also indicates that PHA121 reached therapeutic levels in the first day of a 10-day treatment period, and stable…

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.

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…

The Phase 2 clinical trial testing KalVista Pharmaceuticals‘ oral investigational therapy KVD900 as an on-demand treatment for hereditary angioedema (HAE) attacks has completed enrollment, the company announced. Results from the trial are expected by year’s end. Meanwhile, the company has submitted a pediatric investigational plan for KVD900 to the European Medicines…

Costs are unsustainable for preventive treatment of hereditary angioedema (HAE) attacks with approved therapies Takhzyro or Haegarda, according to an analysis of six-month real-world data performed by the pharmacy benefit manager Prime Therapeutics. In addition, on-demand therapy plus Takhzyro cost $26,000 more than the same regimen plus Haegarda.

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…