FDA Brief: Week of Mar 7, 2016
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
- 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
- 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.
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).
- 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
- 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 (crizotinib) capsules
Pfizer, New York, USA
Reg. Pathway: Breakthrough Therapy Designation, Priority Review, approved prior to PDUFA goal date
- 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
- 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.