Pharmaceutical Technology

SEPTEMBER 5, 2022

The treatment is indicated for CF patients who are homozygous for the F508del mutation (F/F genotype) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The latest approval for use in children aged 12 to below 24 months is based on an open-label, multicentre Phase III clinical trial for 24 weeks.

FDA Approval

Worldwide Clinical Trials

AUGUST 14, 2024

FLT3 mutations seen in acute myeloid leukemia (AML) and BCR-ABL1 fusion gene seen in chronic myeloid leukemia (CML) are two examples. Understand Resistance Mechanisms : Biomarkers can help identify mechanisms of resistance to treatments, as mutations in certain genes can alter the effectiveness of targeted therapies.

Genetics

XTalks

SEPTEMBER 28, 2020

The test is based on the detection of actionable genes – genes with known driver mutations that can be targeted by an approved therapy (or investigational therapies in clinical trials). The panel of genes in the test only include known NSCLC-associated genes, making the test specific to the disease.

DNA

Pharmaceutical Technology

DECEMBER 15, 2022

Since the drug treated CFD population is very small, drug developers are not heavily invested in the space and clinical trials frequently struggle with recruitment, especially in areas with low diagnosed prevalence. CFD is an extremely rare disease in which many patients are often asymptomatic and do not require treatment.

Marketing

XTalks

DECEMBER 12, 2023

The world of neuroscience trials is rapidly evolving, presenting new challenges and opportunities for researchers and clinicians. The worldwide market for neurology clinical trials stood at a valuation of $5.24 There is currently a heightened emphasis on imaging and liquid biomarkers in neuroscience clinical trials.

Trials

Bioengineer

JANUARY 16, 2024

In patients with T790M mutant NSCLC receiving osimertinib, 63% experienced loss of T790M at progression, commonly linked to histological transdifferentiation, KRAS mutations, or gene fusions. In the same study, those with T790M loss had a shorter treatment duration (6.1 months vs. 15.2 months, p = 0.01).

Genomics

XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

Gene Therapy

XTalks

APRIL 15, 2021

Late-stage clinical trial failures are largely to blame for the lack of effective treatment options for patients with HFpEF, and poor five-year survival rates make this an area of great unmet need. A lack of efficacy is the greatest reason for clinical trial failures, especially in Phase II and Phase III,” says Dr. Tyl.

Development

The Pharma Data

NOVEMBER 2, 2020

ONWARD is investigating Adial’s lead drug candidate, AD04, as a therapeutic agent for the treatment of Alcohol Use Disorder (AUD) in persons with certain target genotypes related to the serotonin transporter and receptor genes. Key ONWARD highlights include: All 25 planned investigative sites are active.

Trials

XTalks

MARCH 3, 2021

MoCD Type A is an autosomal recessive, inborn error of metabolism caused by mutations in the molybdenum cofactor synthesis 1 gene. The efficacy of Nulibry in the treatment of MoCD Type A was demonstrated in data from three different clinical trials that was compared to data from a natural history study.

FDA Approval

The Pharma Data

NOVEMBER 24, 2020

The safety and efficacy of Oxlumo are also being evaluated in the ongoing ILLUMINATE-C Phase 3 clinical trial in patients of all ages with advanced PH1, including patients on dialysis. Until today, there were no approved pharmaceutical therapies for PH1.

FDA Approval