Researchers from the University of Pennsylvania have developed a gene therapy method that was successful in treating an autosomal dominant form of blindness in preclinical experiments. Read More
Takhzyro's (lanadelumab-flyo) breakthrough designation was only good for a one-business-day advantage in gaining FDA approval ahead of its Prescription Drug User Fee Act action date on Sunday. Read More
The U.S. Senate passed a spending bill that would provide the National Institutes of Health with roughly $39 billion in the coming fiscal year, but the legislation also provides funding for development of drug pricing regulations. While the Senate must reconcile its bill with the House of Representatives, the Trump administration has frequently promoted the idea of pharmaceutical price transparency, suggesting this provision has only to get past the House of Representatives to become law. Read More
LONDON – The U.K. government may still expect agreement on an orderly withdrawal from the EU, but with fewer than 140 working days to go, the timelines mean the industry must now act on government instructions to prepare for the worst-case scenario. Read More
A novel means of modeling sporadic amyotrophic lateral sclerosis (SALS) using motor neurons derived from induced pluripotent stem (iPS) cells should provide new insights into the pathology of the disease, researchers reported in the Aug. 20, 2018, edition of Nature Medicine. Read More
HONG KONG – Taiwan Liposome Co. Ltd. said its lead product candidate TLC-599, to treat osteoarthritis pain, has demonstrated promising results in its phase II study, making it a potential best-in-class intra-articular steroid for its duration of action. Read More
HONG KONG – The latest Chinese biopharma company to join the listing spree in Hong Kong was Suzhou-based Ascentage Pharma Group International, which last week filed its application to list on the Hong Kong Stock Exchange (HKEX). Read More
Veloxis Pharmaceuticals A/S, of Copenhagen, said its capital increased by 200,000 shares with a nominal value of DKK0.10 (US2 cents) each, corresponding to nominally DKK20,000 (US$3,100), following exercise of warrants by former employees. Read More
Peptilogics Inc., of Pittsburgh, held a pre-investigational new drug meeting with the FDA where the parties agreed to a regulatory pathway for the development of PLG-0206, an engineered cationic antibiotic peptide, to treat prosthetic joint infections. Read More
DUBLIN – Shire plc's newly approved plasma kallikrein inhibitor Takhzyro (lanadelumab-flyo) has, in the eyes of some analysts, the potential to reshape the hereditary angioedema (HAE) treatment landscape because of its combination of efficacy, safety and tolerability, and convenience. Read More
