Ascendis Pharma A/S kept mum on the cost of just-approved Skytrofa (lonapegsomatropin) for pediatric growth hormone deficiency (GHD) – saying only that “premium responsible pricing” would be put in place – but Wall Street speculated freely about revenues likely due from the first-ever weekly injection treatment. Read More
A potential new treatment for the rare condition Wilson disease moved a step closer to reality after Alexion Pharmaceuticals Inc., now part of Astrazeneca plc, announced positive top-line results from a phase III trial of ALXN-1840 (tiomolibdate choline). Astrazeneca’s shares (NASDAQ:AZN) ticked up following the announcement, closing Aug. 26 at $58.76, up 18 cents. From the point of view of the big U.K. pharma, it’s a good piece of news as it hopes to build a rare disease franchise from its $39 billion acquisition of Alexion, which was completed last month. Read More
Following new positive top-line results from its pivotal phase III study of IPX-203, Amneal Pharmaceuticals Inc. said an NDA submission for the Parkinson’s disease therapy (PD) is now planned for the middle of 2022. Read More
Researchers from Denali Therapeutics Inc. have identified new functional links between progranulin, lysosomal function, and a subtype of frontotemporal dementia caused by progranulin deficiency (FTD-GRN) that suggest progranulin-mediated FTD could be conceptualized as a lysosomal storage disorder (LSD). They also showed that delivery of their experimental therapeutic PTV:PGRN, also known as DNL-593, reduced cell damage and symptoms of FTD in cell and animal models. Read More
With the Americas having the dubious distinction of the most COVID-19 cases in the world, the Pan American Health Organization (PAHO) is answering that “wake-up call” with plans to develop a collaborative platform to develop mRNA vaccine production in Latin America and the Caribbean. Read More
Samsung Bioepis Co. Ltd. has emerged as the first company to obtain marketing authorization from the EMA for a biosimilar of Lucentis (ranibizumab), a significant development for the Korean biosimilar specialist. The approval comes less than two months after the company received a positive opinion from the EMA’s CHMP for Byooviz (ranibizumab), formerly called SB-11. Read More
In a passionately worded 141-page decision, the U.S. Court of Appeals for the First Circuit said the lower court was wrong in overturning part of a jury verdict convicting former Insys Therapeutics Inc. executives of a scheme to bribe doctors to prescribe Subsys (fentanyl), which was approved only to treat breakthrough cancer pain. Read More
Kite Pharma Inc. is no longer on the hook for $1.2 billion in damages and royalties a jury awarded to Juno Therapeutics Inc. and the Sloan Kettering Institute for Cancer Research in a patent infringement suit involving Kite’s CAR T therapy Yescarta (axicabtagene ciloleucel). Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Aerami, Argenx, Blade, CTI, Moderna, RDIF, Taysha. Read More
