The end of the blockbuster era
Realising that the era of blockbuster drugs is coming to an end, Big Pharma has turned its eyes to focus on a new game — orphan
drugs. The pharma industry's new fascination with these drugs is highlighted in Thomson Reuters' The Ones To Watch report for October–December 2009, which examines some of the most promising drugs launched or receiving approval, and moving
through each of the clinical phases, between October and December 2009.
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"As we see from this quarter's selection, drugs for small patient populations have already reached the market," says the report.
"Meanwhile, drugs for urea cycle disorder and multiple myeloma are moving apace through the pipeline, giving hope to patients
diseases that are unlikely to ever deliver a blockbuster drug."
The report also added that, according to a January 2010 report by the Tufts Center for the Study of Drug Development, the
number of drugs receiving orphan status has doubled since 2000. Meanwhile, research from BCC Research also shows that companies
migrating away from blockbusters to orphans are likely to be rewarded— the orphan drugs market is expected to grow from $58.7
billion in 2006 to $81.8 billion in 2011.
Of Thomson Reuters' predicted five most promising drugs launched or receiving approval, three are treatments for rare conditions.
Dyax's Kalbitor, a treatment for hereditary angioedema, which affects between 1 in 10000 and 1 in 50000, has been forecast
by Thomson Pharma to achieve sales of approximately $150 million in 2013.
Meanwhile, Folotyn, developed by Allos, was granted accelerated approval by the FDA in September 2009 and launched in the
US in October, and is the first treatment for relapsed or refractory periphereal Tcell lymphoma (an often aggressive type
of nonHodgkins lymphoma). The report also highlighted Istodax, developed by Celgene for the treatment of cutaneous Tcell lymphoma
(CTCL). The drug is the second anticancer histone deactylase inhibitor to be approved after Merck's Zolinza (also for CTCL),
which, according to the report, suggests "significant potential in this class of compounds".
The other two drugs highlighted were Novartis' Onbrez Breezhaler treatment for chronic obstructory pulmonary disease, which
is expected by Thomson Pharma to achieve sales of $397 million in 2013, and Theravance and Astellas' Vibativ — a treatment
for complicated skin structure infections caused by Gram positive bacteria that is forecast to reach sales of $128.2 million
Thomson Reuters has also identified some of the promising drugs entering the various stages of clinical testing. Again, many
treatments for niche conditions are featured including a treatment developed by ERYtech for acute lymphoblastic leukemia,
which is entering Phase III trials and has orphan status in the US and the EU. A treatment for thymic cancer, which currently
has no standard approved treatment at all, is also being investigated in a Phase II trial by Nerviano Medical Sciences
As well as orphan drugs, Thomson Reuters added that drugs for cancer continue to attract significant attention, with 25% of
the quarter's promising drugs being for oncological indications. Interestingly, the report also showed that companies are
looking at lifestyle fatcors that may lead to cancer; for instance, Nabi Biopharmaceuticals' NicVAX for Nicotine addiction
has entered Phase III clinical trials, while a treatment for alcohol addiction, developed by Alkermes under license from Rensselaer
Polytechnic Institute, has entered Phase II trials.