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ORILISSA (elogolix) Tablets

AbbVie

INDICATION: Management of moderate to severe pain associated with endometriosis

ADDRESSING UNMET NEED:  First FDA-approved oral treatment for management of moderate to severe pain associated with endometriosis in over a decade

MECHANISM OF ACTION:  Nonpeptide small molecule gonadotropin-releasing hormone (GnRH) receptor antagonist; causes  suppression of luteinizing hormone (LH) and follicle-stimulating hormone (FSH), leading to decreased blood concentrations of ovarian sex hormones, estradiol and progesterone.

EFFICACY:

  • Two multinational double-blind, placebo-controlled trials, n=1686 premenopausal women with moderate to severe pain associated with endometriosis
  • Co-primary efficacy endpoints: (1) dysmenorrhea response at Month 3 and (2) non-menstrual pelvic pain response at Month 3
  • Daily self-assessment of their endometriosis pain using numeric rating scale (NRS)
  •  Statistically significant greater responses (mean decreases from baseline) vs. placebo
  • Statistically (p <0.001) significant reduction from baseline in NRS scores vs. placebo

SAFETY:

  • Most common side effects: hot flashes or night sweats, headache, nausea, difficulty sleeping, absence of periods, anxiety, joint pain, depression and mood changes
  • Warnings and Precautions: Bone loss, reduced ability to recognize pregnancy, suicidal ideation and mood disorder, reduced efficacy if contraceptives

REGULATORY PATHWAY: NDA

  • Priority Review
  • Pediatric Assessments: Waived -product does not represent meaningful therapeutic benefit over existing therapies for pediatric patients and is not likely to be used in a substantial number of pediatric patient
  • Postmarketing Requirements:  Prospective pregnancy registry to evaluate effects  on pregnancy  and maternal and fetal/neonatal outcomes

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Capture.JPGMULPLETA (lusutrombopag) Tablets

Shionogi Inc.

INDICATION: Treatment of thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo a procedure

ADDRESSING UNMET NEED: Offers physicians and patients another choice beyond platelet transfusions as adult patients with CLD often undergo procedures that could put them at increased risk for bleeding

MECHANISM OF ACTION:  Thrombopoietin (TPO) receptor agonist; induces proliferation and differentiation of megakaryocytic progenitor cells from hematopoietic stem cells and megakaryocyte maturation

EFFICACY:

  • 2 randomized, double‐blind, placebo‐controlled trials; n=312 patients with chronic liver disease who were undergoing an invasive procedure
  • Major efficacy outcome was the proportion of patients who require no platelet transfusion prior to the primary invasive procedure
  • Statistically significant (p<0.0001) treatment difference vs, placebo

SAFETY:

  • Most common adverse reaction: Headache
  • Warning and Precautions: Thrombotic/Thromboembolic Complications

REGULATORY PATHWAY: NDA

  • Priority Review
  • Pediatric assessments: Waived as necessary studies are impossible or highly impracticable

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AZEDRA (iobenguane I 131) injection

Progenics Pharmaceuticals

INDICATION: Treatment of adult and pediatric patients 12 years and older with iobenguane scan positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma who require systemic anticancer therapy

ADDRESSING UNMET NEED:  

  • First FDA-approved drug for this use in ultra-rare cancer
  • Has been shown to decrease the need for blood pressure medication and reduce tumor size in some patients

MECHANISM OF ACTION: I 131 labeled iobenguane; taken up and accumulates within pheochromocytoma and paraganglioma cells, and radiation resulting from radioactive decay of I 131 causes cell death and tumor necrosis

EFFICACY:

  • Single-arm, open-label, clinical trial, n=68 patients
  • Primary Endpoint: ≥ 50% reduction of all antihypertensive medications lasting for at least six months
  • Secondary Endpoint: Overall tumor response measured by traditional imaging criteria
  • 25%  experienced ≥ 50% reduction of all antihypertensive medication
  • 22% with overall tumor response

SAFETY:

  • Most common severe side effects:  Lymphopenia, neutropenia, thrombocytopenia, fatigue, anemia, increased INR, nausea, dizziness, hypertension and vomiting
  • Warning about radiation exposure to patients and family members
  • Other warnings and precautions:  Myelosuppression, underactive thyroid, elevations in blood pressure, renal failure or kidney injury and inflammation of lung tissue (pneumonitis)

REGULATORY PATHWAY: NDA

  • Priority review, orphan product, fast track status, and breakthrough therapy designation

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FREESTYLE LIBRE Libre 14 Day Flash Glucose Monitoring System

Abbott

INDICATION FOR USE:  Continuous glucose monitoring (CGM) device indicated for the management of diabetes in persons age 18 and older. It is designed to replace blood glucose testing for diabetes treatment decisions

The System detects trends and tracks patterns aiding in the detection of episodes of hyperglycemia and hypoglycemia, facilitating both acute and long-term therapy adjustments

Interpretation of the System readings should be based on the glucose trends and several sequential readings over time. The System is intended for single patient use and requires a prescription

Expanding indication to:

  • extend the sensor wear period to 14 days
  • reduce the sensor warm up time to 1 hour

ADDRESSING UNMET NEED: Longest-lasting self-applied personal blood sugar sensor on the market

SYSTEM DESCRIPTION:

  • Externally-worn glucose sensor that continuously measures glucose levels and displays values to the user in response to a user-initiated action (scan)
  • Provide on-demand glucose information to user for up to fourteen days (the life of each sensor)
  • Does not passively monitor glucose levels or provide messages, alarms or alerts in the absence of user-intiated action

EFFECTIVENESS AND SAFETY:

  • Non-randomized, single arm, multi-center, prospective, pivotal, nonsignificant risk study, without controls
  • Primary endpoint: Accuracy performance evaluation vs. laboratory glucose analyzer during in-clinic sessions that spanned the wear period of the device (days 1, 4, 7 and 10)
  • Comparable to performance of current generation CGM systems; established accuracy across the claimed measuring range (40 to 500 mg/dL glucose), precision, 14 day wear period (following the 1 hour warm-up period) for the sensor, notifications (Glucose Messages), and number of readings displayed during the wear period
  • Possible adverse effects of inserting sensor and wearing adhesive patch: local erythema, local infection, inflammation, pain or discomfort, bleeding at the glucose sensor insertion site, bruising, itching, scarring or skin discoloration, hematoma, and adhesive irritation
  • Potential adverse effects associated with making diabetes treatment decisions when glucose values and rates of change provided by the device are inaccurate

REGULATORY PATHWAY: PMA Supplement

  • Previous version marketed in the US since October 2017 under P160030

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Image credits: AbbVie, Shionogi, Progenics, Abbott

 

 

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