FDA News, Week of July 13th – Drug and Device Digest

century curesUS House passes 21st Century Cures Act to accelerate drug approvals

  • Removing barriers to increased research collaboration
  • Incorporating the patient perspective into the drug development and regulatory review
  • Measuring success and identifying diseases earlier through personalized medicine.
  • Modernizing clinical trials.
  • Removing regulatory uncertainty for the development of new medical apps
  • Providing new incentives for the development of drugs for rare diseases
  • Helping the entire biomedical ecosystem coordinate more efficiently to find faster cures

Storyline :  Step in the right direction for expedited development of drugs and devices (including Apps). However, use of non-traditional approaches for drug approval, e.g. biomarkers, Patient Reported Outcomes, will still need to meet evidentiary standards for the protection of public health. http://energycommerce.house.gov/cures

FDA approved

FDA Approval: In vitro Diagnostic test :VENTANA ALK (D5F3) CDx Assay (Ventana Medical Systems, Inc. Tucson, AZ)

Reg pathway : PMA

Indication : Qualitative detection of the anaplastic lymphoma kinase (ALK) protein in formalin-fixed, paraffin-embedded (FFPE) non-small cell lung carcinoma (NSCLC) tissue stained with a BenchMark XT automated staining instrument. It is indicated as an aid in identifying patients eligible for treatment with XALKORI® (crizotinib).

Clinical Program : Clinical outcome results and retrospective samples from Phase 3 Pfizer Study of efficacy and safety study of crizotinib vs. first-line chemotherapy (pemetrexed/cisplatin or pemetrexed/carboplatin) in previously untreated patients with ALK positive locally advanced or metastatic nonsquamous NSCLC.

Effectiveness : Based on agreement rates between the VENTANA ALK (D5F3) CDx Assay and the FDA-approved Vysis ALK Break Apart FISH Probe Kit. A statistically significant efficacy benefit for crizotinib vs. chemotherapy was observed in the subset of NSCLC patients whose tumors had ALK protein expression, as detected by the VENTANA ALK (D5F3) CDx Assay.

Safety : No additional safety hazard as tissues are routinely removed as part of NSCLC diagnosis.

Benefit/Risk : Adds another option of screening and diagnosis of ALK positivity as a key part of diagnostic evaluation for NSCLC patients supporting crizotinib treatment options.


FDA Approval : Atypical antipsychotic Rexulti (Brexpiprazole, Otsuka Pharmaceuticals, Tokyo Japan, Princeton, NJ)

Indication : Adjunctive therapy to antidepressants for the treatment of major depressive disorder (MDD), and Treatment of schizophrenia

Efficacy :

MDD :   Rexulti superior to placebo in change from baseline to Week 6 in the Montgomery-Asberg Depression Rating Scale (MADRS) in 2 studies.

Schizophrenia :  Rexulti superior to placebo in change from baseline to Week 6 in the Positive and Negative Syndrome Scale (PANSS) total score in 2 studies

Safety: Increased risk of death in patients with dementia-related psychosis, risk of suicidal thoughts and behaviors

Storyline : Brexpiprazole being positioned as a better alternative to aripiprazole that has lost patent exclusivity.


FDA Approval : Tyrosine kinase inhibitor, Iressa (Gefitinib, AstraZeneca, Wilmington, DE)

Reg Pathway :  Orphan Drug Designation, Concurrent labeling expansion of the therascreen EGFR RGQ PCR Kit, a companion diagnostic test for patient selection.

Indication : For the treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 (L858R) substitution mutations as detected by an FDA-approved test.

Efficacy : Single trial in metastatic NSCLC containing EGFR exon 19 deletions or L858R substitution mutations. Tumor samples tested retrospectively using the therascreen® EGFR RGQ PCR Kit . A 50% objective response rate with a median duration of response of 6 months. Response rates were similar in patients whose tumors had EGFR exon 19 deletions and exon 21 L858R substitution mutations.

Supportive exploratory anlaysis in patients with metastatic adenocarcinoma histology NSCLC receiving first-line treatment. Median Progression Free Survival of 10.9 months vs 7.4 months for the carboplatin/paclitaxel. Duration of Response 9.6 months vs 5.5 months.

Safety : Most common skin reactions, aspartate aminotransferase (AST) increased, alanine aminotransferase (ALT) increased, proteinuria, and diarrhea

Storyline : Iressa has received Accelerated Approval in 2003 for the broad indication of metastatic NSCLC, third line setting without demonstration of clinical benefit. Phase IV commitments under Subpart H, failed to demonstrate increase in survival, and the drug was withdrawn. This new approval is based on demonstration of clinical benefit in specific NSCLC patient subsets – for optimized, individualized therapy.http://www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm454692.htm

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