“Finally!” Piper Sandler analysts cheered in a research note on Ardelyx Inc., which after spending three years battling the U.S. FDA, secured approval of Xphozah (tenapanor) for treating hyperphosphatemia in adults with chronic kidney disease (CKD). The company will waste no time getting to market, aiming to have the NHE3 inhibitor available to patients next month, marking the first new treatment class for hyperphosphatemia in 30 years.
Following an unexpected FDA complete response letter (CRL), Alnylam Pharmaceuticals Inc. said it will no longer pursue an expanded indication for Onpattro (patisiran) in the U.S. The RNAi therapeutic was approved in 2018 to treat polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis and seemed well on its way to snagging a second U.S. indication after the FDA’s Cardiovascular and Renal Drugs Advisory Committee voted 9-3 in September that the drug’s benefits outweighed its risks as a treatment for cardiomyopathy of ATTR amyloidosis. The FDA disagreed with the committee, saying in the CRL that patisiran’s clinical meaningfulness had not been established in the proposed indication.
In the ultra-rare disease congenital hyperinsulinism, an overproduction of insulin leads to persistent hypoglycemia and can cause neurological complications due to high glucose needs of the brain. About half of children go on to develop seizures or intellectual problems, but current therapeutic options are limited and there are no approved drugs specifically for the condition.
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.
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