Belgium’s UCB is acquiring California-based Zogenix, a company focused on rare diseases, in a deal valued up to €1.7 billion ($1.9 billion).

Shares of Applied Therapeutics plunged in premarket trading on January 3 after the company announced it is unable to seek regulatory approval of the Galactosemia therapeutic AT-007 at this time.

Rare Patient Voice LLC, a leading market research patient recruitment firm, started off 2022 by reaching a milestone and announcing several staff changes.

Austin, Texas-based Aeglea Biotherapeutics reported what initially appears to be positive outcomes for the company’s Phase III PEACE trial of pegzilarginase in Arginase 1 Deficiency (ARG1-D), a rare, progressive and debilitating disease marked by high levels of the amino acid arginine.

The U.S. Food and Drug Administration greenlit Aadi Bioscience’s Fyarro for treating a rare and aggressive form of cancer that disproportionately affects women.

With more than 1,000 clinical trials for cell and gene therapies in process and predictions that such products will generate $12 billion in revenue annually by 2025, leaders for pharmaceutical companies and health plans must consider how to make gene therapies financially accessible to those who need them. According to Cotiviti Executive Vice President of Operations Jordan Bazinsky, this is an opportunity to explore a new paradigm for reimbursement for complex therapies—one that is informed by collaborative partnership, with careful consideration of the best way to pay for value.

EVERSANA, a pioneer of next-generation commercial services to the global life sciences industry, announced its membership in the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), the region’s principal trade association for small to medium-sized companies in the pharmaceutical, biotech and medtech industries.

The U.S. Food and Drug Administration approved Enzyvant Sciences’s Rethymic (allogeneic processed thymus tissue-agdc) for children with congenital athymia. 

Merck & Co. is buying Acceleron Pharma Inc. for about $11.5 billion, broadening the company’s portfolio beyond the aging cancer drug Keytruda with potential treatments that could bring in fresh revenue.

Mirum Pharmaceuticals Inc. said on Sept. 29 the company’s drug became the first U.S. approved therapy for treating itching in patients with a rare liver disorder called Alagille syndrome (ALGS).