PHARMA & HEALTHCARE FIND THIS ARTICLE HERE: http://www.forbes.com/sites/matthewherper/2012/12/27/the-most-important-new-drug-of-2012/
The Food and Drug Administration looks set for a
great 2012; with a few days left to go, it has approved 40 new drugs and
vaccines, one of the most impressive totals ever, according to data from
Pharmaceutical Approvals Monthly and FDA press releases. In this haul, one
medicine stands out for its scientific and medical importance.
Kalydeco, for cystic fibrosis, is a triumph of
genetics and drug development, the first medicine to directly affect the genetic
defect that causes the disease. It will only help 4% of the 70,000 people who
suffer from declining lung function, damaged pancreases, and shortened lives
due to CF worldwide, but in those few it has a dramatic effect. It makes
medical history for three reasons:
·
It’s a genomics triumph: Francis Collins, later famous for heading the Human Genome Project
and then the National Institutes of Health, discovered the gene that, when
mutated, causes cystic fibrosis 23 years ago. Kalydeco is the first drug to
directly affect the defects caused by these mutations, leading to improvements
in patients’ lung function.
·
A patient group powered its development:Kalydeco would probably not exist were it not
for the Cystic Fibrosis Foundation, which funded its early development at
Vertex and gets a royalty on the drug. This success paved the way for other
disease foundations including the Michael J. Fox Foundation, Myelin Repair, and
the Multiple Myeloma Research Foundation.
·
Its price: Kalydeco, given alone,
will only help a few thousand patients the world over. Like other drugs for
very rare diseases, its price is very high: $294,000 per patient per year.
Vertex shares have fallen 37% from their high
earlier this year because of doubts by investors that Vertex will succeed in
its attempts to dramatically expand Kalydeco’s use by combining it with a
second drug that will make it work in CF patients whose disease is caused by
other, more common, mutations. Initial results were very promising, but then
Vertex had to restate them. Sales of its best-seller, Incivek for hepatitis C,
are dropping. But whatever you think of Vertex shares, Kalydeco is already a
success, with $113 million in sales in the first nine months of 2012.
Kalydeco
was not the only important drug this year, in which the FDA also approved the
first flu vaccine made in cells, not chicken eggs (that’s a Novartis product)
and several important cancer drugs including Onyx’s Kyprolis, Medivation’s Xtandi, and Roche’s
Perjeta. Nor is it the most commercially important — that honor goes to
Gilead’s Stribild combination pill for HIV, which could help preserve that
company’s HIV franchise through patent expirations. But it’s probably the most
exciting as a harbinger of drugs to come.
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