STAT+: Pharmalittle: We’re reading about a Regeneron rare disease drug, an Ultragenyx gene therapy, and more

Rise and shine, everyone, another busy day is on the way. And it is getting off to a pleasant start here on the Pharmalot campus, where clear blue skies and comfy breezes are greeting us. As for the official mascots, they are happily snoozing in their respective corners. Meanwhile, we are firing up the trusty kettle for another cuppa stimulation. Our choice today is Earl Grey. And here is a helpful tip — a teaspoon of honey enhances the flavors splendidly. Of course, you are invited to join us. For the full experience, we are now hawking replicas — take a look. Meanwhile, here are a few items of interest. As always, do keep in touch. We appreciate feedback, criticism, and tips. …

A medicine from Regeneron Pharmaceuticals for an ultra-rare disease that causes bone to grow where it shouldn’t, won U.S. regulatory approval, the capstone of a three-decade effort, STAT writes. The hope is that the new medicine, called Pasatru, can help patients with fibrodysplasia ossificans progressiva, or FOP, maintain their mobility and perhaps even live longer. Typically, people with FOP rely on wheelchairs by age 25 as the bone accumulation locks up their limbs, and only some live into their 50s. A Phase 3 trial demonstrated the drug led to a 94% reduction in new lesions that go on to form bone, while a high dose led to a 90% reduction.

The U.S. Food and Drug Administration approved a gene therapy from Ultragenyx Pharmaeutical that will be the first for treating a rare metabolic disorder, Reuters tells us. The drug, branded as Genglycos, will be used to combat glycogen storage disease type Ia (GSDIa), also known ​as Von Gierke disease, in adults and children. GSDIa affects 1,500 to 2,500 patients in the U.S. The deficiency ​reduces the liver’s ability to control glucose levels and is associated with potentially life-threatening low blood sugar levels. Genglycos will have a U.S. per-patient list price of $2.7 million and be available through qualified treatment centers within 30 to 60 days.

Continue to STAT+ to read the full story…