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FDA's Gene Therapy Demand Sparks Ethical Firestorm
2 Mar
Summary
- FDA requests controversial sham control study for Huntington's therapy.
- UniQure calls the proposed study potentially unethical and burdensome.
- Company shares fell significantly following the FDA's recommendation.
UniQure's gene therapy for Huntington's disease faces a significant setback as the FDA has recommended a controversial sham control study. This proposed trial would require a control group to undergo the surgical procedure without receiving the gene therapy, a design UniQure's Chief Medical Officer has called potentially unethical and a substantial burden to patients.
The company's shares experienced a sharp decline following the FDA's recommendation for this additional clinical trial. UniQure previously reported positive results in a smaller trial, showing a significant slowing of disease progression. However, the FDA later indicated that this data was insufficient for approval.
UniQure intends to seek further discussion with the FDA regarding alternative study designs in the second quarter. Patient advocates have also voiced their concerns, submitting a petition with approximately 50,000 signatures urging the FDA not to delay the review process.
This situation occurs amidst broader scrutiny of the FDA's handling of rare disease treatments. The agency has faced pressure from advocates and investors regarding its review timelines and decisions on several other rare disease therapies.
