News
UniQure Eyes Phase 3 for Huntington’s Gene Therapy Despite Makary’s Apparent Criticism
1+ hour, 52+ min ago (578+ words) After Marty Makary's interview with CNBC last week, in which the FDA Commissioner appeared to criticize uniQure's investigational gene therapy for Huntington's disease, the biotech sustained another blow on Monday, revealing that it will likely need to conduct a Phase…...
Rare Disease Groups Lobby for Regulatory Expediency as Makary Defends ‘Rigorous’ Approach
3+ day, 3+ hour ago (865+ words) Rare disease leaders convened in Washington, D.C., this week to advocate for regulatory flexibility when it comes to therapies targeting rare conditions. The meetings follow a spate of rejections in the space, including for drugs developed by Biohaven and Saol Therapeutics....
Makary Defends FDA’s Decision-Making Amid Contentious Rare Disease Rejections
3+ day, 5+ hour ago (382+ words) Makary Defends FDA's Decision-Making Amid Contentious Rare Disease Rejections'BioSpace Without naming a specific product, Commissioner Marty Makary referred to an investigational therapy, delivered surgically into the brain, that the FDA was "pressured" to approve even after finding no clinical benefit…...
New Analysis of Organon’s VTAMA® (tapinarof) cream, 1%, Phase 3 Data Showed Early and Consistent Improvement in Patient-Reported Sleep Subdomains in Pediatric Patients Down to 2 Years of Age with Atopic Dermatitis
3+ day, 9+ hour ago (304+ words) Consistent with the prescribing information, the most frequently reported (5% in any group) treatment-emergent adverse events in the ADORING 1 and ADORING 2 clinical trials were folliculitis, headache, and nasopharyngitis. In December 2024, the US Food and Drug Administration (FDA) approved VTAMA cream for…...
Bayer’s XOFIGO® (radium-223 dichloride) Plus Enzalutamide Demonstrates Significant Overall Survival Benefit in PEACE-3 Trial in Patients With Metastatic Castration-Resistant Prostate Cancer With Bone Metastases
3+ day, 14+ hour ago (443+ words) XOFIGO is indicated for the treatment of patients with castration-resistant prostate cancer, symptomatic bone metastases and no known visceral metastatic disease.2 XOFIGO is not approved in this investigational indication in combination with enzalutamide. At the time of the final OS…...
Deep Dive: BioSpace Takes On JPM26 in San Francisco
3+ day, 17+ hour ago (454+ words) A few key themes quickly emerged at the conference: China innovation, neuroscience, the return of early-stage science and, most prominently, a lack of major M&A to kick off the conference. That big buyout never materialized, forcing conference goers to…...
Feinstein Institutes’ Scientists Discover Brain Circuit That Links Inflammation and Stress Response
3+ day, 17+ hour ago (675+ words) Feinstein Institutes" Scientists Discover Brain Circuit That Links Inflammation and Stress Response'BioSpace Feinstein Institutes" Scientists Discover Brain Circuit That Links Inflammation and Stress Response New study reveals how the brain can "remember" inflammation, replay immune and stress responses, pointing to…...
Transformative Biotech Names Mark Kelleher, PhD as Executive VP of R&D
3+ day, 21+ hour ago (276+ words) Transformative Biotech Names Mark Kelleher, PhD as Executive VP of R&D'BioSpace Transformative Biotech Names Mark Kelleher, PhD as Executive VP of R&D Industry veteran to drive assay and system development for extraction-free PCR BOULDER, Colo., Feb. 26, 2026 /PRNewswire/ -- Transformative…...
FDA’s One Trial Policy Not a Revolution but a Potentially Risky Evolution
4+ day, 2+ hour ago (1113+ words) Last week, the FDA officially moved from a requirement of two pivotal trials to one for new drug applications. Reaction to the move has been tempered, with one regulatory expert referring to the new policy as being not necessarily a…...
NeuraLight at AD/PD 2026: Revealing Next-Generation Brain-Function Biomarker Readouts Powering a First-of-its-Kind Neuroprotection Signal in Drug Trials
4+ day, 3+ hour ago (391+ words) NeuraLight at AD/PD 2026: Revealing Next-Generation Brain-Function Biomarker Readouts Powering a First-of-its-Kind Neuroprotection Signal in Drug Trials'BioSpace NeuraLight at AD/PD 2026: Revealing Next-Generation Brain-Function Biomarker Readouts Powering a First-of-its-Kind Neuroprotection Signal in Drug Trials Breakthrough neuroprogression biomarker, which outperforms current…...