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Introduction: New FDA Drug Approvals in 2012: A 15-Year High

April 2013 Vol 6 No 3 Payers’ Guide to New FDA Approvals - FDA Approvals, Drug Updates, Payers' Guide
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The year 2012 was marked by the largest number of new drug approvals in the past 15 years. The US Food and Drug Administration (FDA) issued 39 new drug approvals, of which 33 were new molecular entities and 6 were biologics (depending on how you count these).1 This number is approximately 33% higher than the average over the past 2 decades. This group of approvals included1:

  • 16 emerging sponsors (companies receiving their first FDA drug approval)
  • Nearly an 80% first-cycle approval rate (a new high)
  • 20 drugs that were first-in-class compounds
  • 13 new oncology medications
  • 8 new respiratory agents
  • The first disease-modifying therapy for cystic fibrosis
  • The first oral nontraditional disease-modifying antirheu­matic drug (DMARD) for rheumatoid arthritis (RA)
  • 13 orphan drugs.

Specialty Drugs Remained the Growth Area in 2012
The 2012 approvals continue to cast the spotlight on the growth of specialty pharmaceuticals. Among the newly approved drugs last year, 25 can be classified as specialty agents. Of those 25 specialty drugs, more than 50% (13 agents) were for cancer (Table).1 Furthermore, many of these new specialty drugs were approved as orphan drugs, including several of the cancer drugs (Table). The emphasis on specialty drug development is here to stay, and it will likely remain the major focus of pharmacy innovation in the future.

In its 2012 Drug Trend Report, Express Scripts reported an 18.4% increased spending in 2012 for biotechnology and specialty drugs, which was led by medications for cancer and for inflammatory diseases such as RA, HIV/AIDS, and multiple sclerosis (MS).2 The report further notes that specialty drug growth shows no signs of slowing in the near future, because of the combination of price inflation and new drug approvals.2

In fact, some industry experts have predicted that specialty drugs will represent 45% of pharmaceutical manufacturer sales by 2017.3 This trend is further accentuated by the Pharmaceutical Research and Manufacturers of America (PhRMA) report, which indicates that 907 new biologics are in development, including 338 treatments for cancer, 134 vaccines for infectious diseases, 71 medicines for autoimmune disorders, and 58 treatments for cardiovascular disease.4,5

Cancer Drugs Dominate 2012 FDA Approvals
For the second year in a row, cancer drugs dominated the new drug approvals. The FDA approved 13 new agents for cancer (Table), which is a significant increase from the 8 cancer drugs that were approved in 2011.

Hematologic Malignancies
Hematologic malignancies were a major area of new drug approvals in 2012. There were 3 new drugs entering the market for the treatment of chronic myelogenous leukemia (CML): ponatinib (Iclusig), a multikinase inhibitor; bosutinib (Bosulif), a BCR-ABL tyrosine kinase inhibitor/SRC family kinase inhibitor; and omacetaxine mepesuccinate (Synribo). More important, in the patients with CML who are receiving these drugs, we are seeing greater percentages of benefits from the new treatments. For example, ponatinib induces a major cytogenic response in 54% of patients overall, as well as in an astonishing 70% of patients with the T315I mutation.

Furthermore, on the leukemia front, vincristine sulfate liposome (Marqibo) was approved for patients with acute lymphoblastic leukemia (ALL) that has relapsed 2 times or more and for patients with ALL that has progressed after 2 regimens or more of antileukemia therapy. Marqibo contains vincristine, a frequently used anticancer drug, which is encased within a liposome, a drug delivery vehicle composed of material similar to that of cell membranes.

Carfilzomib (Kyprolis), a second-generation proteasome inhibitor, was approved for the treatment of patients with multiple myeloma who have received at least 2 previous therapies, including bortezomib and an immunomodulatory drug, and have demonstrated disease progression. Carfilzomib is the second proteasome inhibitor to be approved, joining bortezomib (Velcade) intravenous (IV), which was approved in 2008. In January 2012, the FDA approved the subcutaneous version of bortezomib, based on study results that showed subcutaneous bortezomib is as effective as the IV form, but subcutaneous administration reduces the frequency and seriousness of side effects, especially peripheral neuropathy, which is a common side effect of this agent.

Solid Organ Tumors
There were also significant approvals for the solid organ tumors. Several agents were approved for the treatment of metastatic colorectal cancer, including ziv-aflib­ercept (Zaltrap) and regorafenib (Stivarga)—a monoclonal antibody that binds vascular endothelial growth factor (VEGF), and a multikinase inhibitor that targets VEGF, respectively. Enzalutamide (Xtandi), an androgen receptor inhibitor, was also approved for the treatment of metastatic prostate cancer. In addition, at the end of 2012, abiraterone (Zytiga) was approved by the FDA for an expanded indication, for the treatment of patients with metastatic prostate cancer before chemotherapy. With this approval, abiraterone, in combination with prednisone, may now be used earlier in the treatment continuum for metastatic castration-resistant disease before the use of chemotherapy.

The hedgehog pathway inhibitor vismodegib (Erivedge) was approved as a first-in-class agent for the treatment of metastatic or locally advanced basal-cell carcinoma, a rare and difficult-to-treat advanced-stage skin cancer. Axitinib (Inlyta), an oral VEGF inhibitor, was approved for the treatment of advanced renal-cell carcinoma.

Another common solid organ tumor, breast cancer, strikes more than 250,000 women annually in the United States. Approximately 20% of these patients have tumors that overexpress the HER2 protein, which is a significant contributor to cancer-cell growth. Pertuzu­mab (Perjeta) was approved in June 2012 and is intended for patients who have not received previous treatment for metastatic breast cancer with an anti-HER2 therapy or chemotherapy. Pertuzumab is used in combination with trastuzumab, another anti-HER2 therapy, and docetaxel, a type of chemotherapy.

Overall, 2012 was a very active year for cancer drug launches, and this trend shows no signs of change. According to the PhRMA report from March 2013, 337 of the drugs currently in development are targeted for cancer.4 Based on these numbers, we can anticipate significant numbers of cancer drugs to be approved well into the foreseeable future.

New Oral Therapies for RA and MS
Two new agents were approved in 2012 for disease categories that traditionally have been dominated by injectable medications—RA and MS.

Tofacitinib (Xeljanz) is the first oral, nontraditional DMARD to be approved by the FDA; its approval has been highly anticipated by patients and physicians. This new RA agent belongs to a class of drugs known as Janus kinase inhibitors. Tofacitinib blocks a signaling molecule that is released when a cytokine binds to the surface of a cell, thereby inhibiting the activity of the cytokine. Based on its mechanism of action, the clinical features of this medication are very similar to those of the interleukin-6 receptor antagonists. Because the FDA approved tofacitinib as a second-line agent for RA, treatment with a biologic agent is not required before a physician can prescribe this agent. Tofacitinib is indicated for the treatment of adults with moderately to severely active RA who have had an inadequate response to or intolerance to methotrexate.

The second oral agent approved in 2012 for the treatment of MS is teriflunomide (Aubagio). Teriflunomide is a once-daily oral agent for the treatment of relapsing forms of MS. Teriflunomide, a pyrimidine synthesis inhibitor that inhibits the function of T- and B-cells, is related to leflunomide, a drug used for the treatment of RA.

Significant Advances in Other Therapeutic Categories
Many other important approvals or expanded indications for existing drugs occurred in 2012. For example, adalimumab (Humira), which has been approved for the treatment of RA since 2002, saw its multiple indications expanded to include the treatment of moderate-to-­severe ulcerative colitis in adults. Adalimumab was approved to control ulcerative colitis when immunosuppressant medicines, such as corticosteroids, azathioprine, and 6-mercaptopurine, have not worked.

From a therapeutic and scientific standpoint, one of the most interesting new products of 2012 is ivacaftor (Kaly­deco), which is the first disease-modifying agent to be approved for a select group of patients with cystic fibrosis. Ivacaftor is a transmembrane conductance regulator stabilizer, and it was designated as an orphan drug and a “breakthrough drug” that could ultimately lead to the development of additional treatments for cystic fibrosis.

Antiobesity drugs were also prominent in 2012, with the approval of 2 new therapies. Qsymia, a new combination of phentermine and topiramate, which are 2 existing drugs, was approved in July 2012, to be used in combination with diet and exercise by adults with body mass index (BMI) >30 kg/m2, or BMI >27 kg/m2 along with a weight-related condition, such as type 2 diabetes, high blood pressure, or high cholesterol levels. The second antiobesity agent approved in 2012, lorcaserin (Belviq), is a serotonin 2C receptor agonist that is indicated for chronic weight management in adults when used in conjunction with a caloric restriction diet and increased physical activity.

To date, the uptake of the phentermine plus topiramate combination has been gradual, and lorcaserin has not yet launched. Other 2012 approvals of note include mirabegron (Myrbetriq) for overactive bladder, linaclotide (Linzess) for irritable bowel syndrome, and apixaban (Eliquis), the second factor Xa inhibitor.

Looking Forward
For the drug industry and for all healthcare stakeholders, 2012 certainly was an exciting year. The approvals of new drugs reached a 15-year high, with many of these approvals being for novel agents that offer first-in-class treatments for patients with chronic or life-threatening diseases. For the second year in a row, a significant increase was seen in the number of FDA approvals.

This trend of novel drug approvals and industry growth, which became evident in 2011, is expected to continue. As we enter 2013 with a robust pipeline, we can anticipate another year of significant growth for the drug industry and for patients.

Author Disclosure Statement
Dr Owens is consultant to Allergan, Biogen Idec, Boston Scientific, CardioDx, Crescendo Biosciences, Genzyme, Iroko, Johnson & Johnson, Lilly, and Novocure.


  1. Mullard A. 2012 FDA drug approvals. Nat Rev Drug Discov. 2012;12:87-90.
  2. Express Scripts. 2012 Drug Trend Report. March 2013. Accessed March 7, 2012.
  3. Artemetrx. Specialty drug trend across the pharmacy and medical benefit. 2013. Accessed March 12, 2013.
  4. Dearment A. PhRMA: 907 biotech drugs, vaccines under development. March 11, 2013. Drug Store News. vaccines-under-development. Accessed March 14, 2013.
  5. Pharmaceutical Research and Manufacturers of America. Biologics research promises to bolster future of medicine: more than 900 biologic medicines and vaccines in development. March 11, 2013. promises-bolster-future-medicine. Accessed March 17, 2013.
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Last modified: August 30, 2021