FDA Approved: IDYLLA Ebola Test, OCALIVA, ZINBRYTA, COBAS v2, NETSPOT – Drug and Device Digest

FDA BRIEF: Week of May 3o, 3016


FDA approved


Biocartis NV, Mechelen BELGIUM


INDICATION: Presumptive detection of Ebola Zaire virus1 (detected in the West Africa outbreak in 2014) in EDTA venous whole blood specimens from individuals with signs and symptoms of Ebola virus infection in conjunction with epidemiological risk factors

REG PATHWAY:  Emergency Use Authorization (EUA). Test only by laboratories  certified under the Clinical Laboratory Improvement Amendments (CLIA)


  • Automated real-time reverse transcription polymerase chain reaction (rRT-PCR) assay
  • Consisting of instrument,  console, and single-use test-specific cartridge
  • Samples inserted into Cartridge are processed using encrypted software,  Test Type Packages (TTP)
  • System covers entire sample-to-result process: preparation (homogenization, cell lysis and RNA extraction), rRTPCR amplification, target sequence detection, PCR data analysis, result reporting


  • Ebola Zaire viruscan cause Ebola virus disease, a serious or life-threatening disease or condition to humans
  • Based Totality of Scientific Evidence, Virus Triage Test may be effective in diagnosing Ebola Zaire virus and that the known and potential benefits of detection of infection outweigh the known and potential risks
  • No adequate, approved, and available alternative to the emergency use of the Triage Test



OCALIVA (obeticholic acid) tablets

Intercept Pharmaceuticals, New York, New York, USA

INDICATION:  treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in adults unable to tolerate UDCA


  • PBC is  chronic, or long lasting, disease that causes inflammation and damage of  small bile ducts in liver
  • Damages liver over time and loses its ability to function.
  • Left untreated, or unresponsive to UDCA, are at risk for liver failure and death

REG PATHWAY: NDA, Fast Track Designation, Orphan Drug Designation, Accelerated Approval

MECHANISM OF ACTION:  Binds to the farnesoid X receptor (FXR), a receptor found in cell nucleus in liver and intestine; increases bile flow from liverm suppresses bile acid production, thus reducing liver  exposure to toxic bile acid levels


  • Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
  • Trial 1: Randomized, double-blind, placebo-controlled, 12-month trial (n=216), vs. placebo
  • Primary endpoint: Composite Responder analysis, Month 12,:  ALP less than 1.67-times the ULN, total bilirubin less than or equal to ULN, and an ALP decrease of at least 15%. : 48%, 46% on OCALIVA vs 10% for placebo


  • Most common side effects: Severe itching of the skin (pruritus), fatigue, abdominal pain and discomfort, joint pain (arthralgia), pain in the middle part of the throat (oropharyngeal), dizziness and constipation


ZINBRYTA (daclizumab) injection

Biogen Inc., Cambridge, Massachusetts, USA

INDICATION:  Treatment of adult patients with relapsing forms of multiple sclerosis (MS). Because of its safety profile, the use of ZINBRYTA should generally be reserved for patients who have had an inadequate response to two or more drugs indicated for the treatment of MS


  • MS is a chronic, inflammatory, autoimmune CNS disease
  • Most common cause of neurological disability in young adults; more frequently in women
  • Most people experience their first symptoms of MS between the ages of 20 and 40
  • Need for additional choice to patients who may require a new option


MECHANISM OF ACTION: Precise mechanism unknown; presumed to involve modulation of IL-2 mediated activation of lymphocytes through binding to CD25


  • 2 randomized, double-blind, controlled studies (n=1841, 412 respectively) subcutaneous ZINBRYTA taken once every four weeks in patients with relapsing multiple sclerosis (RMS).
  • Study 1: ZINBRYTA vs Avonex. ZINBRYTA had statistically significant effect on annualized relapse rate (ARR) and on the number of new or newly enlarging T2 hyperintense lesions.
  • Study 2:  ZINBRYTA vs. placebo in 412 patients. ZINBRYTA had a statistically significant effect on ARR (week 52), the proportion of patients relapse free, the number of new T1 Gd-enhancing lesions, and the number of new or newly enlarging T2 hyperintense lesions


  • Boxed Warning on severe liver injury, including life-threatening and fatal events; perform routine blood tests
  • Boxed Warning on immune conditions eg non-infectious colitis, skin reactions, lymphadenopathy
  • Warnings on  hypersensitivity reactions (anaphylaxis or angioedema), increased risk of infections, and symptoms of depression and/or suicidal ideation
  • Common adverse reactions:  Cold symptoms, upper respiratory tract infection, rash, influenza, dermatitis, throat pain, eczema, and enlargement of lymph nodes.



Roche Molecular Systems, Pleasanton, California, USA

INDICATION FOR USE: Companion diagnostic test for the detection of exon 19 deletions or exon 21 (L858R) substitution mutations in the epidermal growth factor recptor (EGFR) gene to identify patients with metastatic non-small cell lung cancer (NSCLC) eligible for treatment with Tarceva® (erlotinib).


  • 221,200 Americans diagnosed with lung cancer; 158,040 will die
  • NSCLC is the most common type of lung cancer.
  • EGFR mutations present in ~10-20 % NSCLC patients
  • Need for blood-based genetic test to detect EGFR gene mutations in NSCLC
  • Highly individualized health care for patients

REG PATHWAY: PMA, Priority Review. Classification: Somatic Gene Mutation Detection System, Pathology Advisory Committee.

Approved prior to MDUFA III goal date

  • Test already approved for NSCLC indication using formalin-fixed paraffin-embedded tissue specimens
  • New use is for detection of  specific mutations in circulating-free tumor DNA  isolated from plasma specimens – liquid biopsy specimens
  • First approved  “liquid biopsy test


  • Phase III TARCEVA vs gemcitabine + cisplatin study, first-line treatment for stage IIIB/IV NSCLC patients (ENSURE study)
  • Cobas EGFR Mutation Test v1 used to test tumor samples positive for EGFR exon 19 deletion or L858R mutations.
  • COBAS EGFR Mutation Test v2 used to test plasma samples for same mutation
  • Positive samples: Agreement 76.7% (70.5%, 81.9%)
  • Negative samples: Agreement 98.2% (95.4%, 99.3%)
  • Tarceva efficacy based on the cobas EGFR Mutation Test v2 in plasma bridged to   efficacy based on the cobas EGFR Mutation Test v1 in tissue


  • To be used to initially screen metastatic NSCLC patients with mEGFR mutations
  • If positive,  EGFR status should be confirmed using tissues specimens

NETSPOT (Somakit-TATE), Kit 

Advanced Accelerator Applications USA, Inc.

INDICATION FOR USE: a kit for the preparation of gallium Ga 68 dotatate injection for localization of somatostatin receptor positive neuroendocrine tumors (NETs) in adult and pediatric patients.


  • Critical to use advanced imaging techniques to detect rare neuroendocrine tumors at an early stage
  • Need for diagnostic tool to determine the location and extent of the tumor
  • Information is important for planning the appropriate course of therapy

REG. PATHWAY: Priority Review, Orphan Drug Designation, Classificaiton: Radioactive diagnostic agent


  • Sterile, single-dose kit for preparation of Ga 68 dotatate injection for intravenous use
  • Uptake of Ga 68 dotatate reflects the level of somatostatin receptor density in NETs
  • Uptake can  be seen in other tumor types, other pathologic conditions, normal variant
  • Uptake of Ga 68 dotatate may need to be confirmed by histopathology


  • Three studies
  • Study 1: Comparison of Ga 68 dotatate images of NETs to images obtained with approved drug, then confirmed with computed tomography (CT) and/ or magnetic resonance imaging (MRI)
  • Study 2: Evaluation of Ga 68 dotatate images using histopathology or clinical follow up as reference standards
  • Study 3: Evaluation of patients with NET recurrence using Ga 68 dotatate images
  • Results confirmed usefulness of Ga 68 dotatate images in finding neuroendocrine tumor location .


  • Contributes to overall long-term cumulative radiation exposure
  • Patients should drink and urinate as often as possible during the first hours following administration to help reduce this risk
  • No serious adverse reactions




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