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.
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.
Revenue from AstraZeneca’s rare diseases portfolio, boosted by the $39 billion acquisition of Alexion in 2021, has swelled in recent years, to nearly $7.8 billion in 2023.
The deal, which is expected to close by April 15, 2024, includes the acquisition of insulin brands Basalog and Insugen which Eris said will help its diabetes treatment franchise reach 10 billion rupees in revenue, without specifying a timeline.
The collaboration will include the development of computational pathology algorithms powered by AI and machine learning (ML) that connect baseline pathology data, such as molecular tumor profiles, with clinical data, such as patient outcomes, to enable better patient identification, stratification, and selection for clinical trials.
In a briefing document for Thursday’s advisory committee meeting, the FDA pointed to efficacy and safety issues with Geron’s New Drug Application for imetelstat in myelodysplastic syndromes.
Staff reviewers said that the pattern of early deaths observed in patients treated with both the therapies in their respective trials raised uncertainty about the treatments’ overall survival benefit in patients.
Eli Lilly has partnered with Amazon Pharmacy to help fill online orders of its obesity drug Zepbound—as well as migraine and diabetes medicines—placed through the pharma’s online portal LillyDirect.
Ionis Pharmaceuticals said on Wednesday its experimental drug to treat a type of fatty liver disease met the main goal of reducing inflammation of the critical organ in a mid-stage trial.
The company, whose products includes Cetaphil, a product for damaged and sensitive skin, as well as fillers and skin creams, early on Wednesday announced a price range of 49 to 53 Swiss francs for its flotation – likely to be one of the biggest in Europe this year.
