STAT+: Pharmalittle: We’re reading about a Regeneron rare disease drug, an Ultragenyx gene therapy, and more
A Regeneron drug for an ultra-rare disease that causes bone to grow where it shouldn't won U.S. regulatory approval, capping a three-decade effort
On a pleasant morning, the Pharmalot campus welcomed clear skies and gentle breezes as reporters prepared for the day's latest developments. Among the morning's highlights was the announcement of U.S. regulatory approval for a rare disease drug developed by Regeneron Pharmaceuticals. The drug, known as Pasatru, targets an ultra-rare condition called fibrodysplasia ossificans progressiva (FOP), which causes bones to form in inappropriate locations.
FOP severely impacts mobility, with most patients requiring wheelchairs by age 25 and living only into their 50s. Clinical trials revealed that Pasatru reduced new bone lesions by 94% and by 90% with high-dose treatment. The U.S. Food and Drug Administration (FDA) granted approval for this groundbreaking gene therapy after three decades of research and development.
Another notable addition to the day's news was the FDA's approval of a gene therapy from Ultragenyx Pharmaeutical, aimed at treating a metabolic disorder known as glycogen storage disease type Ia (GSDIa), also referred to as Von Gierke disease. This condition affects 1,500 to 2,500 patients in the United States. GSDIa impairs the liver's ability to regulate glucose levels, potentially leading to life-threatening low blood sugar levels.
The new drug, Genglycos, will be priced at $2.7 million per patient and will be accessible through specialized treatment centers within 30 to 60 days.
Written by urgent.news from STAT News's reporting — not their text. Machine-written — may contain errors; check the original before relying on it.
