Dermatologic and Ophthalmic Drugs
27 August 2004
Questions for the Advisory Committee
- Has sufficient data been submitted to
evaluate the efficacy and safety profile of pegaptanib sodium? If not, what additional data are needed?
- Based on the Inclusion/Exclusion
Criteria, are there patients excluded from the studies that you believe
need to be studied?
- Are additional analyses of the current
data needed to understand the efficacy or safety of pegaptanib sodium for
the treatment of age-related macular degeneration?
- Visual acuity measurements were
conducted using the ETDRS scale placed at 2 meters from the patient. The validity of the ETDRS scale was
established based on readings at 4 meters.
Are the visual acuity findings sufficiently robust to overcome the
potential bias introduced by visual acuity measurements at 2 meters?
- Has the concomitant use of PDT therapy
with pegaptanib been explored sufficiently? Are there concerns with using this
product concomitantly with PDT therapy?
- Do the route and/or frequency of
administration of the drug raise any concerns that are not addressed by
- Are there adverse experiences that are
of particular concern for this product?
- Endophthalmitis (approximately 2%) was
observed in these studies. What is
the optimal follow-up needed to minimize the impact of potential
- Vascular Endothelial Growth Factor
(VEGF) has been shown to be an important component in the development of
collateral vessels in ischemic heart disease. Inhibition of VEGF in the systemic
circulation could present a theoretical increased risk of symptomatic
cardiovascular disease in the target population of elderly patients with
AMD. Has the adverse event profile
of the two randomized phase 3 trials raised any concern over the possible
systemic effects of this therapy?
Is there additional monitoring that should be in place for patients
on pegaptanib sodium therapy?
- Do the benefits of using pegaptanib
sodium outweigh the risks in the treatment of age-related macular