Soleno Therapeutics’ Vykat XR is now FDA approved for treating hyperphagia, or excessive hunger, in patients with Prader-Willi syndrome. The once-daily pill is the first approved therapy for this rare disease, a leading cause of childhood obesity.
Novartis’s Fabhalta is the first FDA-approved therapy for C3 glomerulopathy. But Apellis Pharmaceuticals’ Empaveli has superior clinical data, and that drug has been submitted for FDA review in the same rare kidney disease as well as another one that does not yet have an approved therapy.
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Beam Therapeutics’ first clinical data in the rare disease alpha-1 antitrypsin deficiency “set a bar for efficacy in the space,” according to one analyst. But the ongoing market slide poses challenges for biotech companies aiming to raise money by selling stock.
Zevra Therapeutics received a priority review voucher for Miplyffa, the first FDA-approved treatment for the rare metabolic disorder Niemann Pick disease type C. Though Zevra has other drug candidates that could benefit from the voucher, the biotech is selling it to an undisclosed buyer.
PepGen is one of several companies vying to bring to market the first treatment for the rare muscle disorder myotonic dystrophy type 1, or DM1. Preliminary Phase 1 data indicate its drug candidate is competitive with others further ahead in development.
GLP-1 drugs dominate the obesity market, but Aardvark Therapeutics aims to curb appetite by going after a different target: TAS2 receptors in the gut. The biotech’s lead drug candidate is in Phase 3 testing in Prader-Willi syndrome, a genetic disorder that causes persistent hunger leading to overeating.
The ongoing nursing shortage facilitates high turnover rates since nurses know they won’t have difficulties finding new jobs. In order to retain and attract staff, it’s in a facility’s best interest to understand what nurses want.
SpringWorks Therapeutics’ Gomekli is now FDA approved for treating tumors caused by the rare genetic disorder neurofibromatosis type 1. The approval comes as SpringWorks has emerged as a potential acquisition target.
Currently available therapies for Hunter syndrome don’t cross the blood-brain barrier to address cognitive symptoms of the inherited enzyme deficiency. Denali Therapeutics' technology gets its drug across the protective membrane, and the biotech plans to seek accelerated FDA approval in this rare disease.
Neurocrine Biosciences’ Crenessity is now FDA approved for treating classic congenital adrenal hyperplasia (CAH), a rare and potentially fatal endocrine disorder. The approval comes with a priority review voucher, which Neurocrine could sell for around $150 million.
Novo Nordisk’s new Denmark site will produce drugs for rare diseases, such as hemophilias. Separately, the pharma giant said all regulatory conditions have been met for Novo Holdings’ acquisition of Catalent, which will bring to Novo Nordisk three manufacturing sites for GLP-1 medications.
Balancing healthcare benefits and insurance costs requires research. From becoming a Difference Card member to offering wellness resources, there are many ways to improve healthcare at low cost.
A patient death in a clinical trial usually prompts the FDA to impose a clinical hold. But Neurogene is able to avoid a lengthy clinical trial pause largely because of its participation in an FDA pilot program intended to speed up the development of therapies for rare diseases.
The Silicon Valley startup formerly known as Ciitizen relaunched as Citizen Health. The company seeks to empower patients with access to their complete health history — and it’s starting off by focusing on patients with rare diseases.
The PTC Therapeutics gene therapy, Kebilidi, treats an enzyme deficiency that affects the body’s ability to produce dopamine, a neurotransmitter important for motor control. FDA approval for the product comes two years after it won its first regulatory approvals in Europe.
Neurogene’s Rett syndrome gene therapy has preliminary data supporting safety and efficacy of the one-time treatment. But a late-breaking report of a serious complication in a patient who received the high dose sent shares of the biotech downward.
Septerna’s lead program is in Phase 1 testing for hypoparathyroidism. The therapies available and in development for this rare disease are injectable, and Septerna aims to stand apart with an oral GPCR-targeting small molecule.