US FDA approvals of new biopharmaceutical products increased significantly in 2009, reversing a trend of low numbers and a decline in approvals in recent years. US FDA granted 19 full approvals for biopharmaceuticals in 2009. This was the highest number of new product approvals since 2005, and considerably more than the 11 and 10 approvals in 2008 and 2007, respectively.
Eliminating approvals for products that are not really new, 16 New Biopharmaceutical Entities (NBEs; new products) received approval in 2009, another recent record. A record number of products were also approved for orphan indications. However, continuing a trend in recent years, no biopharmaceuticals received approval for cancer indications, contrary to the many reports that cancer treatment is where much of the growth in biopharmaceuticals is taking place. Compared to recent years:
There was a further increase in the number of recombinant protein/antibody approvals (7); 2009 new products will collectively attain significantly higher peak sales levels and have more impact on health care; and In 2009, there were even fewer new products from US-based and smaller biotechnology-type companies.
This study was prepared by Ronald A Rader, president, Biotechnology Information Institute, and author of BIOPHARMA: Biopharmaceutical Products in the US and European Markets, the only information resource specializing in biopharmaceuticals. Rader remarked, “Overall, 2009 was a good year for FDA biopharmaceutical approvals, with 19 approvals rising from the abysmally low levels of recent years to levels in line with approvals over the past decade. And biopharmaceutical approvals will further increase in coming years, with a large number of filings pending and many more expected, including for new classes of products, such as gene therapies, stem and other cellular therapies, and cancer vaccines."
There were 19 full approvals for biopharmaceutical products in 2009. Eight of these were for recombinant proteins/antibodies, up from only two in 2007. All approvals involved products regulated by US FDA as biologics, with these receiving BLAs, except for one (Creon) that received approval as a drug (NDA).
Among the 19 biopharmaceuticals receiving full approval in 2009, 16 are considered New Biopharmaceutical Entities (NBEs), i.e., are new, unique, distinct products. This is a significant increase, up from 10, 11 and 11 in 2008, 2007 and 2006, respectively. Unlike in recent years, there were no biogeneric/biosimilar-like [505(b)(2) generic drug] approvals.
No biopharmaceuticals received approval in 2009 for cancer indications, despite a large number reported to be in development. This continues a trend with none also approved in either 2008 or 2007. New biopharmaceuticals, including recombinant antibodies, for cancer treatment are widely extolled as on tract to revolutionize patient care. So far, in terms of approvals, this is not the case.
Three recombinant monoclonal antibodies, all fully human, were approved in 2009, a significant increase from only one each in 2006-2008.
In terms of economic impact, peak sales for 2009-approved products are expected to cumulatively be greater than for products approved in 2008 and 2007. However, at best, only a few products may achieve blockbuster status (sales over US$ 1 billion/year).
Many of these newly-approved products will significantly improve patient care. However, nearly all of these therapeutic advances are for orphan indications, so the number of patients affected and the economic impacts of these products will be somewhat limited.
2009 provided some notable technological advances, including Cervarix, the first insect cell cultured recombinant biopharmaceutical, also the first vaccine approved in the US with a totally new adjuvant AS(04), composed of 3-deacylated monophosphoryl lipid A (MPL; derived from Salmonella minnesota); and ATryn, recombinant antithrombin-III expressed in the milk of transgenic goats, became the first transgenic protein to receive approvals in the US and European Union, a significant precedent for transgenic animal-derived therapeutic proteins.
The Biopharma Web database reports at least 30 biopharmaceutical filings currently pending with US FDA and at least 30 expected to be filed in 2010. Although it is too early to conclude that problems and delays with US FDA reviews of biopharmaceutical filings have subsided and whether these were attributable to the sponsors and/or US FDA, further increases are expected in 2010 and coming years.