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A handful of billion-dollar deals in the rare disease space highlights the uptick in Big Pharma’s investment, but it’s still extremely low compared to the money flowing to more common indications.
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With AbbVie’s $1.2 billion acquisition of Gilgamesh Pharmaceuticals’ lead depression drug, the psychedelic therapeutics space has soundly rebounded from Lykos’ rejection last year. There are now seven programs in Phase III trials across the sector, with multiple companies vying for that first approval.
A decade-long journey has come to an end for Stealth BioTherapeutics and the Barth syndrome community with the first-ever treatment for this uncommon mitochondrial disease. CEO Reenie McCarthy called it a “pivotal victory” that “offers hope for expedited regulatory attention to other ultra-rare diseases.”
After a tension-packed two days that saw recommended changes to the MMRV vaccine schedule and COVID-19 vaccine access, as well as a delayed hepatitis B vaccine vote, policy experts expressed concern with the reconstituted committee’s dearth of previous experience and understanding of their role.
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Merck announced today that the U.S. Food and Drug Administration (FDA) has approved the addition of the intramuscular (IM) route of administration to the United States Product Insert (USPI) for Merck’s MMRV family of vaccines: M-M-R®II, VARIVAX®, and ProQuad®.
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The FDA has vowed to fix a pharma ad loophole—but they’re targeting the wrong one.
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Interius BioTherapeutics has received approval from Australia’s Human Research Ethics Committee to begin the first-in-human trial of an investigational in vivo CAR-T therapy designed to treat B-cell malignancies.
The regulator Tuesday added atopic dermatitis to the label of Arcutis Biotherapeutics’ topical PDE4 inhibitor Zoryve for the treatment of patients six years of age and older.
Investment in the development of new antimicrobials is falling rapidly, even as the global public health threat of antimicrobial resistance is growing increasingly severe worldwide.
After pulling its ALS drug Relyvrio from the market, Amylyx Pharmaceuticals is looking to target the GLP-1 space with the purchase of Eiger BioPharmaceuticals’ avexitide, which has been studied for the treatment of hyperinsulinemic hypoglycemia.
Preliminary data from a late-stage trial of Alnylam’s RNAi therapy for ATTR amyloidosis with cardiomyopathy appear strong but details expected later this summer are critical.
The pharma giant announced Tuesday that Mikael Dolsten is leaving his role as chief science officer after more than 15 years. Pfizer is conducting an external search for a replacement with Dolsten staying on to assist in finding his replacement over next several months.
As Novo Nordisk’s weight loss drug enters the Chinese market, its patent is expiring in two years and biosimilar competition is rising.
The Federal Trade Commission on Tuesday issued an interim report on the top pharmacy benefit managers, showing that they are generating massive profit at the expense of patients by inflating prescription drug costs.
House Speaker Mike Johnson (R-La.) on Monday said he intends to have a “significant package of China-related legislation” signed into law by the end of this year, including the BIOSECURE Act which intends to stop federal contracts with Chinese “companies of concern.”
Eli Lilly’s Mounjaro outperformed Novo Nordisk’s Ozempic at inducing weight loss in obese or overweight adults, according to an observational study published Monday in JAMA Internal Medicine.