Clinical Research Focus. 45th Edition

Trump Administration Weighs “Severe Restrictions” on U.S. Drug Licensing from China

According to a New York Times report, the Trump administration is considering a draft executive order that would impose stricter oversight on U.S. pharmaceutical deals with Chinese biotech firms, including national security reviews of licensing agreements and tougher scrutiny of Chinese clinical trial data

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Cromos Pharma Experts Lead Successful “Mastering CTIS” Webinar

On September 23, 2025, Cromos Pharma’s regulatory experts hosted the live webinar “Mastering CTIS: Strategies for Multi-Country Submissions, Compliance & Operational Success.” They shared insider guidance on avoiding RFIs, aligning with country-specific requirements, and ensuring smooth multi-country submissions under the EU CTR framework.

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FDA Grants First-Ever Treatment Approval for Barth Syndrome

The U.S. FDA has granted accelerated approval to Forzinity (elamipretide), marking the first approved therapy for Barth syndrome, a rare and life-threatening mitochondrial disease. Designed to improve muscle strength and mitochondrial function, Forzinity offers new hope to patients while requiring confirmatory trials to validate long-term clinical benefits.

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ISO 9001:2015 and ICH GCP E6(R3): Building One Language of Quality in Clinical Research

Cromos Pharma explores how integrating ISO 9001:2015, the world’s most widely adopted quality standard, with the latest revision of ICH GCP E6(R3) creates a unified system of governance, transparency, and patient protection. For sponsors, this integration means stronger compliance, streamlined operations, and results that stand up to both regulatory scrutiny and patient trust.

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EMA Recommends First Treatment for Rare IgG4-Related Disease

The European Medicines Agency’s CHMP has recommended expanding the use of Uplizna (inebilizumab) to treat adults with immunoglobulin G4-related disease (IgG4-RD), a rare autoimmune disorder with no approved therapies in the EU. Phase 3 trial results showed Uplizna significantly reduced disease flares and enabled more patients to achieve corticosteroid-free remission, marking a major step toward addressing a critical unmet medical need.

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Single-Dose Antibiotic Found as Effective as Standard Three-Dose Regimen for Early Syphilis

An NIH-funded clinical trial has shown that a single injection of benzathine penicillin G is just as effective as the traditional three-dose regimen in treating early syphilis. The findings, published in The New England Journal of Medicine, could simplify treatment, reduce patient burden, and help address rising syphilis rates amid ongoing antibiotic shortages.

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Rapid Clinical Immunity to P. vivax Shown After a Single Controlled Human Infection

A Nature Communications study (Sept 25, 2025) reports that malaria-naïve adults developed clinical immunity to Plasmodium vivax after just one controlled human malaria infection—showing fewer fevers and laboratory abnormalities on rechallenge without reducing parasite growth. The effect was species-specific (no protection against P. falciparum), highlighting new directions for vaccine and trial design focused on disease tolerance and inflammation control.

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uniQure’s Gene Therapy Slows Huntington’s Progression by 75%, Eyes 2026 FDA Filing

uniQure’s experimental gene therapy AMT-130 has delivered a landmark win in phase 1/2, showing a 75% slowing of disease progression at 36 months in Huntington’s patients on a high dose. With strong functional and biomarker data, the biotech now plans to submit a BLA in early 2026, aiming to launch the first disease-modifying therapy for Huntington’s next year.

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HeartFlow’s Next-Gen AI for Coronary Plaque Wins FDA Clearance & Expands Insurance Coverage

HeartFlow has received FDA clearance for an upgraded AI platform that quantifies coronary plaque in 3D—improving detection by 21% over prior versions and aligning with intravascular imaging 95% of the time. The move is backed by payer momentum: Cigna will begin covering the software in both commercial and Medicare Advantage plans, making it the second major insurer to do so.

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