Three years after Alnylam Pharmaceuticals Inc. gained U.S. FDA approval of the first treatment for rare disease primary hyperoxaluria type 1 (PH1), Novo Nordisk A/S solidified its own marketing clearance for RNAi therapy Rivfloza (nedosiran).
After a nearly year-long delay pegged to COVID-19 travel restrictions, the U.S. FDA has approved Amicus Therapeutics Inc.’s Pompe disease drug, introducing competition for Sanofi SA’s standard-of-care treatment and anticipating blockbuster sales. The combination of Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) 65-mg capsules was approved for adults with late-onset Pompe disease, who weigh at least 40 kg and who are not improving on their current enzyme replacement therapy.
Positive top-line phase III study results for olezarsen in treating familial chylomicronemia syndrome has Ionis Pharmaceuticals Inc. looking down the road to U.S. FDA approval. It’s the company’s second attempt at getting an approval for treating the rare disorder.
Soleno Therapeutics Inc. (NASDAQ: SLNO) pulled off in a major way its randomized-withdrawal phase III study with DCCR (diazoxide choline) in Prader-Willi syndrome, boosting the shares by $22.37, or 505%, to close Sept. 26 at $26.80. “Our work is not done, but this was a big step,” said CEO Anish Bhatnagar.
A hot biopharma research approach and an equally hot therapeutic area came together in the potential $2.7 billion deal that pairs artificial intelligence (AI) company Valo Health Inc. with Novo Nordisk A/S, which will strive to advance new treatments for cardiometabolic diseases.
Neurocrine Biosciences Inc.’s positive top-line data from the phase III study called Cahtalyst in classic congenital adrenal hyperplasia (CAH) further whetted Wall Street’s appetite for soon-to-come results in the same indication from Spruce Therapeutics Inc.
The majority of patients with acromegaly who switched from therapy with injected somatostatin receptor ligands to Crinetics Pharmaceuticals Inc.’s once-daily, oral drug paltusotine maintained stable levels of insulin-like growth factor 1 in a phase III trial, moving the company’s first-in-class lead closer to becoming the potential new standard of care for the rare hormonal disorder.
Genexine Co. Ltd.’s recombinant human growth hormone eftansomatropin alfa (GX-H9/TJ-101) met the primary endpoint in a phase III pediatric study conducted in China in children with growth hormone deficiency, and the company plans to file a BLA in 2024 in China on the data.
China’s National Medical Products Administration has approved Innovent Biologics Inc.’s proprotein convertase subtilisin/kexin-type 9 (PCSK9) inhibitor, Sintbilo (tafolecimab), making it the first first locally developed PCSK9 monoclonal antibody to be approved in China. The approval is for treatment of adult patients with primary hypercholesterolemia (including heterozygous familial and non-familial hypercholesterolemia) and mixed dyslipidemia (abnormal lipid levels). Tafolecimab marks Innovent's first cardiovascular drug as well as the company’s 10th approval.
Camurus AB’s subcutaneous octreotide hydrochloride injection pen, CAM-2029, yielded statistically significant improvements in “multiple endpoints” in a 52-week phase III extension trial in patients with acromegaly, moving it closer to providing a more convenient treatment option for patients with the rare, chronic growth disorder than currently available therapies.
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