The end of the blockbuster era
"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.
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 in 2013.
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.