2011 FDA Biopharmaceutical Approvals: Low Number And Economic Impact, But A Record Number Of Orphan Approvals And Many Innovations

The latest annual study of FDA biopharmaceutical product approvals by the Biotechnology Information Institute (BII) reports significant advances in health care, particularly approvals for new and orphan indications, but an overall low number of approvals and industry and economic impact. According to Mr. Ronald A. Rader, President, BII, "With much of the pharmaceutical industry, particularly the major players, the largest companies, increasingly counting on biopharmaceuticals for their future profits, if recent trends of low number of approvals and their expected sales persist, there could well be insufficient new products and revenue to support the industry in the long-term." The study is derived from the BIOPHARMA database and is online at www.biopharma.com/approvals_2011.html.

Twelve biopharmaceuticals received full FDA approval in 2011, a number even lower than the poor results in the past few years. But Mr. Rader noted, "The encouraging news is that nearly all 2011 approvals were advances for patients and health care, involving indications for which either no therapeutic was previously approved or the last product approval was well over a decade ago." This included a record number (8) and percentage (66%) of approvals for orphan indications. However, associated with this, most of the approved products involve inherently limited markets (low revenue).

Therapeutic advances in 2011 included the first new treatment for lupus in over 50 years (Benlysta); the first new treatment for Hodgkin's lymphoma since 1977 (Adcetris); the first treatment for scorpion stings (Anascorp); the first treatment for Factor XIII deficiency (Corifact); the first treatment for rejection of organ transplants in more than a decade (Nulojix); the first therapeutic showing survival benefit in advanced melanoma (Yervoy); the first cord blood (hematopoietic stem) cell product for transplantation (Hemacord); the first injected live cellular product for cosmetic use (smile lines; IaViv); a new asparaginase enzyme for those allergic to the current product; and a new live oral adenovirus vaccine solely for U.S. military use. Also, the first FDA approval was granted to a biologic manufactured in Latin American (Anascorp).

There was a continuing high level of innovation in active agents. Eleven of the 12 approved products are considered New Biopharmaceutical Entities (NBEs; new unique, not me-too or follow-on products). These include the only approved immunotoxin (antibody-cytotoxin conjugate); a "trap" molecule involving cellular receptors linked by an antibody framework; two recombinant antibodies, both fully human, against new targets (CTLA-4 receptor and B-lymphocyte stimulator/BLyS); the first immunoglobulin against scorpion venom; the first plasma-derived Factor XIII product; cultured live autologous (patient's own) fibroblasts for injection; the first cord blood (hematopoietic stem) cell product approved for transplantation; fungal-derived asparaginase enzyme; and a new live oral adenovirus vaccine.

However, only 4 of the 12 (33%) approvals were for recombinant proteins, a low number and percentage. The much-anticipated growth in the number of biopharmaceuticals for cancer treatment has yet to materialize, with only 3 approvals in the past 5 years.

Yervoy (Bristol-Myers Squibb) and Benlysta (Human Genome Sciences Inc.) were the only products commonly cited as having any potential to attain blockbuster status (sales =$1B/year). However, Benlysta appears to be on track for much lower sales, with $52M sales reported for 2011, in contrast with Yervoy which was approved later and is expected to have had about $350M sales in 2011.

Other study findings include: a record number of approvals involve manufacture outside the U.S.; a high level, one-half, of approvals went to companies receiving their first FDA biopharmaceutical approvals; and none of the major independent biopharma companies, e.g., Amgen or Biogen Idec, received approvals. Similarly, only one large international (Big Pharma-type) company received any approvals, with Bristol-Myers Squibb (BMS) the only recipient of two approvals, with these expected to attain among the highest sales among 2011 approvals.

For more information, visit www.biopharma.com.

SOURCE: Biotechnology Information Institute (BII)