News

New clinical trial data show that BioCryst Pharmaceuticals‘ investigational therapy for hereditary angioedema (HAE), berotralstat, reduces the frequency of HAE attacks and improves patients’ quality of life through 48 weeks of treatment. The data were presented at the European Academy of Allergy and Clinical Immunology Digital Congress. HAE…

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a disease that few people — sometimes even physicians — have heard of is challenging on its own merit.

The U.S. Patent and Trademark Office (USPTO) issued a new allowance for a patent submitted by BioCryst Pharmaceuticals for berotralstat (BCX7353), an investigational therapy to treat hereditary angioedema (HAE). The USPTO issued a notice of allowance to BioCryst, indicating future approval. Once approved, the new “composition…

The Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency (EMA), is recommending the approval of a prefilled syringe to administer Takhzyro (lanadelumab), a preventative treatment for hereditary angioedema (HAE) in patients 12 or older. The European Commission (EC), which typically accepts…

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…

After showing a sustained reduction in kallikrein levels in animal models, Intellia Therapeutics will be moving forward with the development of its gene-editing candidate NTLA-2002 for the treatment of hereditary angioedema (HAE). The company is now planning to ask regulatory authorities to advance the candidate into clinical studies…

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…

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 Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…