FDA BRIEF: Week of December 5 and 12, 2016
MACI (autologous cultured chondrocytes on porcine collagen membrane)
Vericel Corporation, Cambridge, MA, USA
INDICATION: Repair of single or multiple symptomatic, full thickness cartilage defects of the knee with or without bone involvement in adults.
- Knee problems common in all ages
- Knee cartilage defects due injury, straining, overuse, muscle weakness, general wear and tear
- Tailored therapy required; need to additional treatment option
REG. PATHWAY: BLA
- First approval for process of tissue engineering to grow cells on scaffolds using healthy cartilage tissue from the patient’s own knee.
- Autologous cultured chondrocytes on porcine Type I/III collagen
- Autologous chondrocytes propagated in cell culture and seeded on collagen
- MACI implant contains at least 500,000 cells per cm 2 and does not contain any preservative
- 2-year prospective, multicenter, randomized, open-label, parallel-group study, MACI vs. Microfracture Treatment., patients with symptomatic articular cartilage defects in the knee, n=144, 104 weeks
- Co-primary efficacy endpoint: Change in Knee injury and Osteoarthritis Outcome Score (KOOS) in two subscales: Pain and Function (Sports and Recreational Activities [SRA]), week 104
- Statististically significant improvement in KOOS pain and function (SRA) p = 0.001
SAFETY: Most common side effects: joint pain, common cold-like symptoms, headache and back pain.
EUCRISA (crisaborole) ointment
Anacor Pharmaceuticals (Pfizer), Palo Alto, CA, USA
INDICATION: Topical treatment of mild to moderate atopic dermatitis in patients 2 years of age and older.
- Atopic dermatitis (Eczema) , a chronic inflammatory skin disease
- Caused by combination of genetic, immune and environmental factors
- Skin develops red, scaly and crusted bumps, itchy and “weeping” clear fluid
- Need to another treatment option
REG. PATHWAY: NDA
MECHANISM OF ACTION: Phosphodiesterase 4 (PDE-4) inhibitor; results in increased intracellular cyclic adenosine monophosphate (cAMP) levels; specific therapeutic mechanism(s) not well defined
- 2 multicenter, randomized, double-blind, parallel-group, vehicle-controlled trials , n=1522, EUCRISA vs. vehicle, twice daily for 28 days
- Primary efficacy endpoint: Proportion of subjects with ISGA grade of Clear (score of 0) or Almost Clear (score of 1) with a 2-grade or greater improvement from baseline
- Greater ‘success’ with EUCRISA
- Serious side effect: Hypersensitivity reactions
- Most common side effect: Site pain, including burning or stinging