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With CBER director Vinay Prasad set to depart the agency at the end of the month, a coalition of patient groups and biotech executives penned a letter imploring the Trump administration to “restore regulatory clarity” for rare disease therapies. Experts on a BioSpace panel last week also acknowledged the challenges faced by a more stringent FDA.
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Following Insmed’s decision to hold off on launching a newly approved lung disease drug in Europe, experts anticipate more companies will do the same as they seek to avoid price erosion in the U.S. Will Chinese biotechs fill the void?
The recent uptick in IPOs is an encouraging signal after a drought for much of 2025. Experts point to AI as a driving force behind this resurgence.
Deal-hungry Big Pharmas, a long-sought biotech prize, an infrequent buyer and one serial biotech rabblerouser highlight a busy quarter in biopharma M&A.
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While requests by government officials for anonymity when speaking to the media are nothing new, the practice attracts more scrutiny when the Department for Health and Human Services has pledged a commitment to “radical transparency.”
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TrumpRX and DTC sales may expand prescription drug access, but they will not solve the affordability crisis by themselves.
Zealand Pharma’s shares fell 32% in early morning trading Friday after its Roche-partnered asset petrelintide failed to meet investor expectations in a mid-stage clinical trial.
The FDA granted Johnson & Johnson a Commissioner’s National Priority Voucher in December 2025 after the combination of Darzalex and Tecvayli improved progression-free survival by 83%. The new approval is for second-line treatment of the blood cancer.
UniQure does not have to drill placebo burr holes in the skulls of patients with Huntington’s disease, an unnamed FDA senior official said on Thursday. Instead, the company would anesthetize them and put “one to three nicks” in their scalp.
Psychedelics are a “game changer” in depression care, according to William Blair, but the complicated treatment regimens mean they will likely be supplanted by more-traditional options once they become available.
The alliance, which pairs Tenaya’s modality agnostic target identification and validation capabilities with Alnylam’s deep experience in RNA interference therapeutics, comes during a period of resurgence for the cardiovascular space.
UniQure’s Path for Huntington’s Gene Therapy Clouded by Ethical Questions as Potential Phase 3 Looms
While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be considered for approval, experts believe there is an alternate path forward for the therapy, perhaps even based on precedent from the recent drama surrounding Moderna’s mRNA flu vaccine.
Adam Urato, who is currently a vaccine advisor to the CDC, is closely associated with acting CDER director Tracy Beth Høeg and is a fellow skeptic of the use of selective serotonin reuptake inhibitors during pregnancy.
The timing of the partial hold is “odd,” according to analysts at Stifel, who noted that the preclinical data the FDA took issue with were filed in mid-2024.
South Korea has attracted increasing investment from the pharmaceutical industry, which is drawn to the Asian country due to its experience in antibody-drug conjugates and cell and gene therapies, according to McKinsey.