FDA BRIEF: Week of May 8, 2017

FDA approved


Image result for radicava image

RADICAVA (edaravone) intrvenous infusion

Mitsubishi Tanabe Pharma America

INDICATION:  Treatment of amyotrophic lateral sclerosis (ALS)

ADDRESSING UNMET NEED:

  •  ALS is rare disease; attacks and kills nerve cells controlling voluntary muscles leading to paralysis
  • 12,000-15,000 Americans have ALS; most  die from respiratory failure within 3-5 years

REG. PATHWAY: NDA

  • Leveraged data from Japanese trials
  • Orphan drug designation

MECHANISM OF ACTION: Unknown

EFFICACY:

  • 6-month, randomized, double-blind study, n =137 Japanese patients with ALS, RADICAVA vs. placebo
  • Primary efficacy endpoint: Change in the ALS Functional Rating Scale – Revised (ALSFRS-R) total scores from baseline at week 24
  • Decline in ALSFRS-R scores from baseline significantly less in the RADICAVA-treated patients (p=0.0013)

SAFETY:

  • Most common adverse reactions: Bruising (contusion), gait disturbance
  • Serious risk: Hives, swelling, or shortness of breath, and allergic reactions to sodium bisulfite, an ingredient in the drug

LABEL


Image result for BAVENCIO IMAGE

BAVENCIO (avelumab)

EMD Serono

SUPPLEMENTAL INDICATION: Treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) who:

  • Have disease progression during or following platinum-containing chemotherapy
  • Have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy

REG PATHWAY: sBLA

  • Accelerated approval, Priority review
  • Approved based on tumor response and duration of response
  • Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials

EFFICACY:

  • Open-label, single-arm, multi-center study, n=242 patients with locally advanced or metastatic UC with disease progression
  • Efficacy outcome measure: Confirmed overall response rate (ORR) using RECIST criteria, duration of response (DOR); followed for minimum of both 13 weeks and 6 months
  • ORR: For at least 13 weeks : 13.3% (n=30), 6 weeks 16.1% (n=26)
  • Median time to response: 2.0 months

SAFETY:

  • Most frequent serious adverse reactions: Urinary tract infection/urosepsis, abdominal pain, musculoskeletal pain, creatinine increased/renal failure, dehydration, hematuria/urinary tract hemorrhage, intestinal obstruction/small intestinal obstruction, and pyrexia
  • Most common adverse reactions: fatigue, infusion-related reaction, musculoskeletal pain, nausea, decreased appetite, and urinary tract infection

LABEL


Image result for keytruda IMAGE

KEYTRUDA (pembrolizumab) injection

Merck

SAFETY:

  • Most common AEs : Fatigue, nausea, constipation
  • Most common grade 3-4 adverse reactions: Fatigue, dyspnea, nausea, vomiting, diarrhea, and rash
  • Most common adverse reaction: Acute kidney injury
  • Immune-mediated adverse reactions: Pneumonitis, colitis, hepatitis, endocrinopathies, and nephritis

LABEL


Picture of the device, highlighting upper esophageal pouch, oral catheter, magnet, stomach, gastric catheter, and gastronomy site.

FLOURISH Pediatric Esophageal Atresia Device

Wilson-Cook Medical 

INDICATION FOR USE:  For use in pediatric patients, up to one year of age, with esophageal atresia without a tracheoesophageal fistula (TEF) or for whom a TEF has been closed as a result of a prior procedure. This device should be used only when the gap between the upper and lower portions of the esophagus is less than 4 centimeters apart.

ADDRESSING UNMET NEED:

  • 1 in every 2,500 babies born with esophageal atresia; birth defect causing gap in esophagus
  • Condition cannot feed normally, also have a tracheoesophageal fistula, breathing inteference
  • First non-surgical option to treat this birth defect

REG PATHWAY: Humanitarian Device Exemption

DEVICE DESCRIPTION:

  • 2 catheters  with a magnet at its tip
  • One inserted through mouth and other through stomach
  • Magnetic ends attract one another and pull ends of esophagus together; surrounding tissue grows together

EFFECTIVENESS:

  • Implanted in 16 patients
  • All had successful joining of esophagus within 3-10
  • However, 13 of 16 patients developed anastomotic stricture requiring balloon dilation procedure, a stent or both to repair

SAFETY:

  • Contraindicated in infants with existing tracheoesophageal fistula or who have esophageal segments that are more than 4 centimeters apart
  • Potential complications: Ulceration, tissue irritation, gum irritation
  • Potential long-term complication: Gastroesophageal reflux

FDA OVERVIEW


Image credits: FDA, Google

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