Biotechnology
The FDA's approval of Denali's Avlayah marks a breakthrough in blood-brain barrier delivery and signals the agency's continued commitment to rare disease innovation amid recent regulatory turbulence.
The Trump administration is waging one of its most aggressive campaigns against healthcare fraud in years, and wound care is squarely in the crosshairs. In February 2026, the Centers for Medicare & Medicaid Services (CMS) imposed a nationwide six-month moratorium on new Medicare enrollment for certain medical supply companies,
Pharmaceuticals
Ionis Pharmaceuticals cuts Tryngolza's price by 93%, from $595,000 to $40,000 annually, signaling a strategic shift in rare disease drug pricing ahead of a broader indication approval.
Biotechnology
The FDA's approval of nivolumab plus chemotherapy for previously untreated Stage III and IV classical Hodgkin lymphoma on March 20, 2026 marks the formal arrival of immunotherapy at the front line of one of oncology's most treatable cancers.
Markets
There is a particular kind of corporate transaction that tells you more about where an industry is heading than any analyst forecast. Abbott's completion of its $21 billion acquisition of Exact Sciences on March 23, 2026 is one of those transactions. It is not simply a large deal
Pharmaceuticals
Eli Lilly's Phase 3 retatrutide data reveals a triple-acting metabolic drug that may push weight loss efficacy beyond tirzepatide, with patients losing up to 15% of body weight over 40 weeks—and the trajectory still heading downward.
Pharmaceuticals
The FDA approved IMCIVREE (setmelanotide) as the first therapy for acquired hypothalamic obesity on March 19, 2026. Here is what the TRANSCEND trial data mean for patients, the MC4R pathway platform, and the rare disease market.
Pharmaceuticals
For the roughly 125 million people worldwide living with psoriasis, the treatment conversation has long followed a familiar and frustrating arc. Start with topical creams. Cycle through them. Graduate, eventually, to an injectable biologic that requires a needle, a refrigerator, and a willingness to self-administer a shot every few
Pharmaceuticals
Pfizer's Phase 2 FOURLIGHT-1 data on atirmociclib show a 40% reduction in disease progression risk in CDK4/6 inhibitor-resistant breast cancer, positioning the selective CDK4 inhibitor as a potential successor to Ibrance ahead of its 2027 patent expiry.
Biotechnology
Structure Therapeutics' aleniglipron showed 15% weight loss in Phase 2 trials, delivering injectable-like efficacy in pill form and reshaping the competitive landscape for oral GLP-1 obesity drugs.
Biotechnology
Sana Biotechnology's 14-month follow-up data shows transplanted insulin-producing cells surviving and functioning without immunosuppression—a potential breakthrough in type 1 diabetes treatment.
Pharmaceuticals
Evaluate's 2026 Orphan Drug Report projects rare disease drug sales will exceed $400 billion by 2032. Behind the headline number lies a more complicated story about FDA volatility, IRA policy shifts, and the structural forces reshaping who wins in rare disease medicine.