Entries by BioSpace

FDA action alert: Orchard, ITF, Merck and more

The FDA’s busy March continues with six target action dates remaining on the calendar. Over the next two weeks, the regulator will decide on investigational therapies for Duchenne muscular dystrophy, chronic kidney disease anemia, a rare metabolic disorder and more.

Mirum secures Livmarli’s expansion into familial intrahepatic cholestasis

The California-based biotech also announced that it has filed a supplemental New Drug Application (sNDA) seeking approval for a higher concentration formulation of Livmarli for younger patients with progressive familial intrahepatic cholestasis (PFIC), a rare genetic disorder that causes progressive liver disease and can lead to significant morbidity and mortality. Mirum hopes to introduce this higher-concentration regimen later this year, according to its announcement.

,

PhRMA loses another legal battle as court sides with Arkansas over drug discount program

The U.S. Court of Appeals for the Eighth Circuit on Tuesday ruled against industry group Pharmaceutical Research and Manufacturers of America, upholding an Arkansas law that enables healthcare providers to dispense drugs—obtained at a discounted price via a federal program—through certain pharmacies. The ruling would also allow Arkansas to prohibit drugmakers from limiting the availability of their products on contract pharmacies and could potentially embolden other states to enact similar laws.