News

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

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…

EMA Grants Priority Medicines Designation to Sotatercept for PAH

The European Medicines Agency (EMA) has granted priority medicines (PRIME) designation to investigational therapy sotatercept (ACE-011) for the treatment of patients with pulmonary arterial hypertension (PAH). This designation is given to medications in development that have the potential to treat conditions with an unmet medical need…

PAH Trials Pausing Enrollment for COVID-19, United Therapeutic Says

United Therapeutics has temporarily stopped enrolling patients in ongoing clinical trials of treprostinil-based therapies for pulmonary arterial hypertension (PAH) amid the COVID-19 pandemic, the company announced. Treprostinil is a man-made version of the hormone prostacyclin, a natural compound that helps blood vessels relax and widen, prevents arteries from blocking, and…