BioSpaceOpinion

Opinion: Direct-to-Consumer Drugs Are Not a Panacea for High Costs

TrumpRX and DTC sales may expand prescription drug access, but they will not solve the affordability crisis by themselves.
BioSpace

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 ...
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Psychedelics Are Placeholders for More Traditional Depression Therapies: Analysts

Psychedelics are a “game changer” in depression care, according to William Blair, but the complicated treatment regimens mean ...
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FDA’s Hoeg Reportedly Trying To Hire Friend, Fellow Antidepressant Skeptic

Adam Urato, who is currently a vaccine advisor to the CDC, is closely associated with acting CDER director Tracy Beth Høeg ...
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FDA’s Prasad To Depart FDA Following Tumultuous Term as CBER Chief

Center for Biologics Evaluation and Research Director Vinay Prasad will exit the FDA for the second time after a controversial run that saw the oncologist butt heads with colleagues and ruffle ...
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PepGen’s Mid-Stage Myotonic Dystrophy Study Hit With ‘Surprise’ Pause

The timing of the partial hold is “odd,” according to analysts at Stifel, who noted that the preclinical data the FDA took ...
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Intellia’s CRISPR Gene Therapy Free To Move Forward After FDA Fully Lifts Hold

The FDA last October paused Intellia Therapeutics’ late-stage CRISPR studies after detecting life-threatening enzyme ...
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FDA Official Fires Back at UniQure, Doubles Down on Sham-Controlled Trial

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 ...
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UniQure Eyes Phase 3 for Huntington’s Gene Therapy Despite Makary’s Apparent Criticism

Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed ...
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Bespoke Startup EveryONE Folds 1 Week After Plausible Mechanism Guidance

While an anonymous source tied the closure to shortcomings in the FDA’s new pathway, a spokesperson for the Department of ...
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Merck, Pfizer’s Drug Combo ‘Rewrites the Standard of Care’ in Bladder Cancer

A combination of Merck’s Keytruda and Pfizer’s Padcev could offer a chemotherapy-free treatment alternative for patients with ...
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REGENXBIO’s Hunter Rejection Hinged on Inadequate Controls, Surrogate Endpoint

In a complete response letter published by the FDA on Monday, the agency said a resubmission for REGENXBIO’s Hunter syndrome gene therapy should provide evidence of normalized or improved biomarker ...