Following the recent recommendation of its advisory committee, the U.S. FDA gave the nod to Zevra Therapeutics Inc.’s arimoclomol, a day ahead of its Sept. 21 PDUFA date, as the first treatment for Niemann-Pick disease type C (NPC), a rare genetic disease linked to progressive neurological symptoms that is almost always fatal. Branded Miplyffa, it is indicated for use in combination with enzyme inhibitor miglustat in adults and children, ages 2 and older. Read More
An innovation that has kickstarted a revolution in the study and practice of health care is getting even more attention. Three scientists who pioneered the discovery and development of glucagon-like peptide-1 (GLP-1)-based obesity treatments have been named 2024 Lasker Award winners. Read More
The specter of elevated liver enzymes, a known problem with the drug class, became a topic of talk with regard to Sanofi SA’s tolebrutinib, the central nervous system-penetrating Bruton's tyrosine kinase (BTK) inhibitor for multiple sclerosis (MS). Meanwhile, another player in the space, Immunic Inc., has caught the eye of Wall Street lately. Read More
Ascletis Pharma Inc. is entering the obesity space, announcing it has begun two phase I trials for ASC-30, a small-molecule glucagon-like peptide-1 (GLP-1) receptor agonist that can be dosed once monthly subcutaneously and once daily orally for treating obesity. Read More
By treating mice with antifibrotic drugs, researchers were able to stave off functional decline of the ovaries. Six weeks of systemic treatment with the antifibrotic pirfenidone, which is approved for the treatment of idiopathic pulmonary fibrosis, in early middle age improved both the morphology and the endocrine function of the animals’ ovaries at 1 year of age. Read More
Although details are scant, Shanghai Fosun Pharmaceutical Co. Ltd. plans to buy out partner Kite Pharma Inc. (now Gilead Sciences Inc.) in the Fosun Pharma Kite Biotechnology Co. Ltd. joint venture for $27 million. Read More
The U.S. SEC settled charges against Philip Markin, a fifth person charged in connection with an insider trading scheme involving the February 2021 $1.85 billion offer by Merck & Co. Inc. to acquire Pandion Therapeutics Inc. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Achilles Therapeutics, Basilea Pharmaceuticals. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Bavarian Nordic, Ensysce Biosciences, Longboard Pharmaceuticals, Vanda Pharmaceuticals, Xspray Pharma. Read More
