Novartis AG (NYSE:NVS) affiliate Novartis Pharmaceuticals Corp. said
its Afinitor (everolimus) drug to treat patients with non-cancerous
kidney tumors associated with tuberous sclerosis complex was approved by
the US Food and Drug Administration (FDA), sending its shares up 0.71%
FDA approved Afinitor (everolimus) tablets for the treatment of adult
patients with kidney tumors known as renal angiomyolipomas and tuberous
sclerosis complex (TSC), who do not require immediate surgery. This
marks the first approval of a medical treatment in this patient
The accelerated approval was based on the Phase 3 EXIST-2 (Examining
everolimus In a Study of TSC) trial, which found that 42% of patients on
everolimus experienced an angiomyolipoma response versus 0% of patients
in the placebo arm.
The time to angiomyolipoma progression was also statistically
significantly longer in patients on everolimus. Among the 97% of trial
patients with skin lesions, one of the key concerns for the majority of
patients with TSC, a 26% response rate was seen with everolimus versus
0% with placebo.
Novartis previously received approval for everolimus for the
treatment of adult and pediatric patients, aged three or older, with
subependymal giant cell astrocytoma (SEGA) associated with TSC who
require therapeutic intervention but are not candidates for curative
surgical resection, in the U.S. and in more than 40 additional
countries. Filings for renal angiomyolipoma are under way in multiple
countries outside of the U.S.
Affecting about 25,000 to 40,000 people in the U.S., TSC can affect
many different parts of the body, including the kidneys and brain, as
well as the heart, lungs and skin.
Tuberous sclerosis complex is
associated with a variety of resulting disorders, including skin
lesions, seizures, swelling in the brain (hydrocephalus), kidney
failure, developmental delays and behavioral issues.
NVS closed Thursday's regular session up 0.47% at $55.11. The stock
has been trading between $51.60 and $64.82 for the past 52 weeks.