FDA Brief: Week of Mar 7, 2016


 

FDA approved


IDELVION (coagulation Factor IX albumin fusion protein)

CSL Behring, King of Prussia, Pennsylvania, USA

Indication:  Children and adults with hemophilia B (congenital factor IX deficiency) for:

  • On-demand control and prevention of bleeding episodes
  • Perioperative management of bleeding
  • Routine prophylaxis to prevent or reduce the frequency of bleeding episodes

Unmet need:

  • Rare inherited bleeding disorder; prevents normal clotting
  • Repeated episodes of potentially serious bleeding, mainly into the joints, which can be damaged by the bleeding
  • Need for another important therapeutic option for children and adults to prevent or control bleeding and reduce the bleeding episode frequency

Reg. Pathway: BLA, first coagulation factor-albumin fusion protein product to be approved

Mechanism of Action: Replace Factor IX, a naturally occurring clotting factor that is missing (functionally deficient) or defective in people with Hemophilia B

Efficacy:

  • Two multicenter studies (n=90), adult and pediatric patients with Hemophilia B between 12 and 61 years of age
  • On-demand Control and Prevention of Bleeding Episodes: >90% Excellent/Good based on investigator assessed 4-point scale
  • Perioperative Management of Bleeding:  >80% assessed as excellent/good by investigator/surgeon
  • Routine Prophylaxis: 89% reduction in the annualized bleeding rate

Safety:  No safety concerns identified; most common side effect was headache.

Prescribing Information


triggerfish.JPG

 

  TRIGGERFISH (soft silicone contact lens) 

Sensimed AG, Lausanne, Switzerland

Intended Use: One-time use contact lens that may help practitioners identify the best time of day to measure a patient’s intraocular pressure (IOP).

Unmet Need:

  • Glaucoma, due to elevated IOP, leading cause of vision loss; 3 million Americans
  • IOP varies throughout the day; may not be abnormally high during eye exam
  • Elevated IOP is often associated with the optic nerve damage in glaucoma
  • Need for device to detect fluctuations in IOP

Reg. Pathway: De Novo premarket review

Device Description:

  • Sensor: Embedded in a soft silicone contact lens that detects tiny changes or fluctuations in an eye’s volume
  • Adhesive antenna: Worn around eye; receives data
  • Portable data recorder: Worn by patient;  transfers  data via Bluetooth to clinician’s computer
  • Shows the range of time during the day the pressure of the eye may be increasing.

Effectiveness: Several studies; association between device output and IOP fluctuation.

Safety: Temporary side effects were pressure marks from the contact lens, ocular hyperemia (red eyes) and punctate keratitis (irritation of the cornea)


xalkori Xalkori (crizotinib) capsules

Pfizer, New York, USA

Indication: Treatment of metastatic non-small cell lung cancer (NSCLC) whose tumors are ROS1-positive.  (Crizotinib was first approved in 2011 for the treatment of patients whose tumors are anaplastic lymphoma kinase (ALK)-positive).

 

Reg. Pathway: Breakthrough Therapy Designation,  Priority Review, approved prior to PDUFA goal date

Efficacy: 

  • Single multicenter, single-arm trial (n=50), 25-77 yrs,  in patients with metastatic ROS1 rearrangement-positive NSCLC
  • Objective response rate (ORR), RECIST v1.0 , by independent radiology review: 66%
  • Duration of response: 18 months
 Safety: 
  • Consistent with the safety profile of crizotinib
  • Most common adverse reactions: vision disorders, nausea, diarrhea, vomiting, edema, constipation, elevated transaminases, fatigue, decreased appetite, upper respiratory infection, dizziness, and neuropathy.

 

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