FDA clearance of Skytrofa (lonapegsomatropin-tcgd) from Ascendis Pharma A/S gave pediatric patients a weekly option for growth hormone deficiency (GHD) treatment. The long-acting prodrug of somatropin releases the same medicine as daily therapies. Regulators cleared the compound for patients one year and older who weigh at least 11.5 kg (25.4 lbs.) and have growth failure due to inadequate secretion of endogenous growth hormone.

AZ/Alexion’s Wilson disease drug hits the mark in phase III, calming post-merger nerves

The first 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 from ALXN-1840. Astrazeneca’s shares (LSE:AZN) ticked up following the announcement in early trading. 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.

Amneal looks to file NDA in Parkinson’s following phase III readout

Following positive new top-line results from its pivotal phase III study of IPX-203, Amneal Pharmaceuticals Inc. said an NDA submission is now planned for the middle of 2022. The study met its primary endpoint in treating patients with Parkinson’s disease who experience motor fluctuations, demonstrating statistically significant improvement in efficacy for IPX-203 compared to immediate-release carbidopa/levodopa (CD/LD), even when IPX-203 was dosed less frequently.

Study casts dementia subtype as lysosomal storage disorder

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. They also showed that delivery of their experimental therapeutic, PTV:PGRN, reduced cell damage and symptoms of FTD in cell and animal models.

Tired of waiting for vaccines, PAHO proposes making its own

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. “Limited production and unequal distribution of vaccines” have compromised the region’s pandemic response and “put public health at very high risk,” PAHO Director Carissa Etienne said at a weekly pandemic media briefing. Noting delays in getting vaccines ordered months ago from the handful of companies making all the COVID-19 vaccines for the world, Etienne said the PAHO region needs “to be in the driver’s seat of our own pandemic responses.”

Samsung Bioepis’ Byooviz wins EMA approval

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.

Second Circuit: Jury got it right on Insys execs

In a passionately worded decision, the U.S. Court of Appeals for the Second Circuit removed any hint of leniency a district court showed to five former Insys Therapeutics Inc. executives convicted of a scheme to bribe doctors to prescribe Subsys (fentanyl), which was approved only to treat breakthrough cancer pain. Reinstating the full jury verdict, the appellate court said the executives, including Insys founder John Kapoor, “chose to shunt medical necessity to one side and shamelessly proceeded to exploit the sickest and most vulnerable among us – all in an effort to fatten the bottom line and pad their own pockets.”

Fed Circuit erases Juno’s $1.2B CAR T patent win

That $1.2 billion in damages a jury awarded Juno Therapeutics Inc. and the Sloan Kettering Institute for Cancer Research in a patent infringement case against Kite Pharma Inc.’s CAR T-cell drug Yescarta (axicabtagene ciloleucel)? Kite is no longer on the hook for it. The U.S. Circuit Court of Appeals for the Federal Circuit reversed the jury Aug. 26, saying its verdict regarding the written description in the patent claims the jury held as valid was not supported by substantial evidence. Thus, there is no infringement because the claims are invalid, the appeals court said.

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