Clinical Trial News

The DEA approved Takeda Pharmaceuticals to increase Vyvanse production by 24% to address ongoing ADHD drug shortages, with a 13,478-pound increase including 3434 pounds for domestic and 10,313 pounds for foreign demand.

AI's potential in drug discovery is highlighted by Recursion Pharmaceuticals' Phase 2 SYCAMORE trial for REC-994, targeting cerebral cavernous malformation, which met safety endpoints but showed mixed efficacy. Despite AI's ability to analyze vast datasets, challenges include data scarcity for rare conditions and predicting drug efficacy accurately. Experts suggest developing more complex AI models and combining AI with traditional techniques to advance drug discovery.

Obeticholic acid (Ocaliva) faces EU ban for lack of benefit in early-stage PBC, but Advanz Pharma secured a temporary suspension of the ban. Patients protest, highlighting the drug's critical role when initial treatments fail.

Strides Pharma Science's subsidiary, Strides Pharma Global Singapore, received USFDA approval for generic Theophylline Extended-Release Tablets, 300 mg and 450 mg, bioequivalent to Schering Corp's THEO-DUR. Indicated for chronic asthma and lung diseases, the tablets will be manufactured in Bengaluru, with a market size of ~$11.5 million.

Drug-drug interactions with statins and calcium channel blockers did not significantly alter colchicine's safety and efficacy in COVID-19 patients, suggesting pharmacokinetic changes do not translate into clinically significant effects.

New research from Cincinnati Children’s suggests that eliminating animal milk alone may be as effective as a four-food elimination diet for treating pediatric eosinophilic esophagitis (EoE). The study, published in The Journal of Allergy and Clinical Immunology, found similar remission rates and symptom improvements between the two diets, supporting a simpler initial approach for children with EoE.

A phase 3 study shows CT-132, a prescription digital therapeutic, significantly reduces monthly migraine days by 3.04 after 12 weeks, improving quality of life and disability scores, with no adverse events reported.

Analysis of COBALT trial shows OCA significantly reduces risk of death, liver transplant, and hepatic decompensation in PBC patients compared to non-OCA treated controls, highlighting the importance of external control data in rare disease trials.

A multicenter study of 11,000+ patients found that omitting the bolus component of 5-fluorouracil (5-FU) in multidrug chemotherapy for advanced gastrointestinal cancers reduces side effects without affecting survival.

CRISPR technology, including CRISPR-Cas9, CRISPR-Cas12, and CRISPR-Cas13, is revolutionizing genome editing. FDA approved the first CRISPR-Cas9 drug, Casgevy®, for sickle cell disease and beta thalassemia. Other applications include treating urinary tract infections, hereditary transthyretin amyloidosis, hereditary angioedema, cardiovascular diseases, type 1 diabetes, systemic lupus erythematosus, HIV, and blood cancers. Challenges include cost, regulatory standards, and ethical considerations.