Drugs in the Pipeline Analysis

Generic Drug
Name (Brand)
Drug Company
Route Indication(s) Regulatory
Priority review * Status
Therapeutic Considerations
Affected
Population
Estimated
Peak Sales
Estimated
Cost/day
rilonacept
(IL-1 Trap)
Regeneron
Injection
Priority review,
Orphan drug
Treatment of
CIAS1-Associated
Periodic
Syndrome (CAPS)
NDA submitted:
5/2007
Estimated
approval date:
11/2007
dose of 20 mg/kg/day (Part 1). Of the 89
patients who completed Part 1, 50
subjects demonstrated a blood Phe
reduction of at least 30%, and 45 were
randomized to Kuvan (20 mg/kg/day) or
placebo in a 3:1 ratio, and enrolled in the
10-week double-blind, placebocontrolled
portion of the study (Part 2).
For the first three weeks, patients
maintained their pre-existing restricted
diet with no supplementation of
phenylalanine. Thereafter, every other
week, specific amounts of phenylalanine
were added (or removed) to the
restricted diet of each patient according
to pre-defined blood phenylalanine
levels. The maximum amount of Phe that
could be added to a patient diet during
the study was 50 mg/kg/day.
IL-1 is a soluble protein secreted by
certain cells in the body, which acts as a
messenger to help regulate immune and
inflammatory responses by attaching to
cell-surface receptors on cells that
participate in the body's immune system.
In excess, it can be harmful and has
been linked to a variety of inflammatory
diseases. The IL-1 Trap is designed to
attach to and neutralize IL-1 in the blood
PAH is not present
in sufficient
quantities, Phe
accumulates to
abnormally high
levels in the blood
and brain resulting
in a variety of
complications,
including severe
mental retardation
and brain damage,
mental illness,
seizures and
tremors, and
cognitive
problems.
CAPS is a family
of rare
autoinflammatory
diseases,
including Familial
Cold Autoinflammatory
Syndrome (FCAS)
and Muckle Wells
Syndrome (MWS).
NA $100-$200
per day
Updated 8/2007
Copyright © 2007 Medco Health Solutions, Inc.
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