Curated news and analysis on clinical trials, drug approvals, and medical research.
INmune Bio will present new Phase III clinical data for its CORDStrom cell therapy for recessive dystrophic epidermolysis bullosa. The webinar on February 26, 2026, will feature investigators discussing outcomes including skin score improvements and quality-of-life measures. CORDStrom represents a systemic, disease-modifying alternative to topical treatments for this rare skin disorder.
SK pharmteco commits $100 million to expand its viral vector business across U.S. and European sites, while Minaris joins CIRM's Industry Resource Partner Program to provide end-to-end cell and gene therapy manufacturing capabilities to funded innovators. Both moves strengthen manufacturing infrastructure for advanced therapies.
The TrumpRx prescription discount website launched in February 2026 offers coupons for 43 brand-name drugs but has limited usefulness for most patients. Experts found 18 drugs are cheaper through other discount services, and the website doesn't indicate when generic alternatives exist. The program links to broader tariff and pricing policies affecting pharmaceutical companies.
The FDA has granted Priority Review to relutrigine for SCN2A and SCN8A developmental and epileptic encephalopathies, with a PDUFA target date of September 2026. Phase 2/3 trial data showed a 53% placebo-adjusted reduction in motor seizures and 66.2% increase in seizure-free days. If approved, it would be the first targeted therapy for these rare, fatal childhood conditions.
The global advanced drug delivery systems market is projected to grow from $255 billion in 2024 to $439.7 billion by 2034 at a 5.6% CAGR. Cardiovascular disease applications dominate with 37.8% market share, while North America leads regionally with 41.3% of the market. Emerging technologies include 3D-printed drug implants, smart hydrogels, and magnetic nanofiber platforms.
Delaware State Police arrested a North Carolina man and seized 2.3 kilograms of cocaine during a traffic stop on I-95. The suspect faces multiple felony drug charges and is being held on $120,801 cash bond. Separate drug investigations in West Virginia and Ohio also resulted in arrests and seizures of fentanyl, methamphetamine, firearms, and child pornography.
The European Medicines Agency has approved Shield Therapeutics' FeRACCRU® for adolescents aged 12 and older, following FDA approval of ACCRUFeR® for children 10+. The expansion was supported by Phase 3 pediatric trial data showing efficacy and safety in children as young as 1 month.
The European Commission has approved KYGEVVI (doxecitine and doxribtimine) as the first treatment for thymidine kinase 2 deficiency (TK2d), an ultra-rare mitochondrial disease. Clinical studies showed the drug reduces death risk by 95% and helps 84% of patients regain motor functions. The approval covers pediatric and adult patients with symptom onset before age 12.
Sobi reported 15% revenue growth to SEK 28 billion in 2025 while treating over 53,000 patients. The company published its Annual and Sustainability Report, highlighting pipeline advancements and transition to new sustainability reporting standards.
South Korea has approved fast-track drug listing for rare disease treatments, reducing the insurance coverage process from 240 days to 100 days. The reform includes post-listing effectiveness evaluations and flexible pricing agreements, aiming to improve patient access to innovative treatments amid ongoing budget concerns.
The global GMP consumables market is projected for significant growth through 2035, driven by expansion in biologics, cell and gene therapies, and mRNA vaccine manufacturing. The market will shift from basic contamination control to enabling advanced therapeutic production with specialized single-use systems. Growth will be supported by regulatory standards, supply chain resilience, and increasing biosimilar production in Asia-Pacific and emerging markets.
Pharmaceutical companies are delaying European drug launches due to uncertainty over U.S. pricing policies under President Trump. New drug launches in EU markets fell 35% following Trump's executive order on international reference pricing. Companies fear lower European prices could undermine their ability to maintain higher prices in the $700 billion U.S. market.
A multinational survey shows bridging therapy is used for over 85% of multiple myeloma patients awaiting CAR T-cell therapy, with proteasome inhibitors being the most common approach. Most centers lack standardized protocols and typically bridge patients for 1-2 months, with regimen selection driven primarily by prior therapy history and disease burden.
Researchers have developed a machine learning method called STRUCTURES25 that enables precise quantum chemistry calculations for large drug-like molecules using an orbital-free approach. The breakthrough solves a decades-old stability problem in computational chemistry while reducing computational requirements. This advancement could accelerate drug discovery and materials science research.
The FDA has accepted Praxis Precision Medicines' NDA for relutrigine with priority review and set a PDUFA target date of September 27, 2026. The therapy targets SCN2A and SCN8A developmental and epileptic encephalopathies and could become the first approved treatment for these rare conditions. The company has bolstered its cash position to support commercial launch preparations.
The FDA has granted Priority Review for lirafugratinib, an FGFR2 inhibitor for second-line cholangiocarcinoma treatment, with a PDUFA date of September 27, 2026. Clinical trial data showed a 46.5% objective response rate and 11.8-month median duration of response. The therapy demonstrated a manageable safety profile with lower rates of common FGFR inhibitor side effects.
Clinical trials take 10-15 years and cost $1-2 billion from discovery to approval, progressing through four phases from safety testing to real-world monitoring. These studies face challenges including high costs, regulatory oversight, and participant recruitment while new technologies are transforming trial design. Illinois faces a shortage of Phase I clinical trial facilities despite local companies conducting over 50 such trials annually.
Biodexa Pharmaceuticals has licensed MTX240, a Phase 1-ready molecular glue therapy for gastrointestinal stromal tumors from Otsuka. The drug demonstrated preclinical efficacy in resistant tumors and is eligible for orphan drug exclusivity, with clinical trials planned by year-end.
Multiple Indian pharmaceutical companies including Zydus, Glenmark, Dr. Reddy's, and Alkem have launched semaglutide versions following patent expiry, dramatically reducing prices and expanding access. Companies are competing on both price and innovative delivery systems, with monthly therapy costs now ranging from Rs 1,300 to Rs 4,200. This development could significantly impact diabetes and obesity treatment in India, where over 100 million people live with diabetes.
Ataraxis AI has launched Ataraxis Breast CTX, an AI test that predicts individualized chemotherapy benefit for breast cancer patients. The tool uses digital pathology and clinical data to estimate patient-level outcomes with and without chemotherapy, moving beyond population averages. The test has been validated across studies involving over 10,000 patients and is now available through CLIA-certified laboratories.
