News

FDA Rejects Changes to Natpara Cartridge, Delaying Its Relaunch

The U.S. Food and Drug Administration (FDA) has rejected Takeda Pharmaceutical’s proposed changes to address the risk of rubber fragments detaching into Natpara’s cartridge that led to the therapy’s U.S. recall in 2019. This treatment for hypoparathyroidism involves puncturing the medication’s cartridge rubber septum — a disk…

Increased Education Crucial to Improving Rare Disease Care, Survey Finds

Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact on treating these conditions over the next five years, according to results from a 2021 survey. Definitive Healthcare, a healthcare commercial intelligence company, conducted…

#RAREis Representation Program Promotes Equity, Diversity

Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…

Investigational Therapy AZP-3601 Granted Orphan Status in Europe

The European Commission has granted orphan drug designation to AZP-3601, Amolyt Pharma’s investigational therapy to treat hypoparathyroidism. Orphan designation is given to promising treatment candidates for life-threatening and debilitating rare diseases, those affecting not more than five in every 10,000 people in the European Union. The designation will…

Rare Disease Day Panel Opens Window to Patient Experience

BioNews, the publisher of this website, hosted a virtual panel discussion on Rare Disease Day 2022, taking a deeper dive into what it’s like to live with a rare disease, including conversations about advocacy, mental health, survivor’s guilt, treatment of minority patients, and more. The Monday event, “A…