A Banner Year for New Drugs

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The FDA ended 2012 with a bang, approving eight new drugs in December and pushing the year's total to 39, its highest level since 1996 when the agency cleared a backlog of applications.

This article published with permission from The Burrill Report.

The U.S. Food and Drug Administration (FDA) ended 2012 with a bang, approving eight new drugs in December and pushing the year’s total to 39, its highest level since 1996 when the agency cleared a backlog of applications.

Though December’s newly approved drugs cover a broad range of conditions, the agency had granted orphan drug status to six of the eight medicines. Orphan drug status confers financial and other benefits to a drug’s sponsor to encourage the development of drugs to treat patient populations of 200,000 or less in the United States. In fact, nearly half of the 39 drugs approved in 2012 had orphan drug designations.

The approvals included many new cancer therapies, as well as treatments for cystic fibrosis, HIV, macular degeneration, Alzheimer’s disease, blood disorders, meningitis and Gaucher’s disease.

Roche’s Erivedge, approved in January 2012, became the first FDA-approved drug for late-stage basal cell cancer, the most common form of skin cancer. January also brought approval of BTG International’s Voraxaze, an enzyme that lowers toxicity associated with the common chemotherapy drug, methotrexate.

Attacking a public health priority, Xtandi was approved in August 2012 to treat men with late-stage castration-resistant prostate cancer. Medivation and Astellas Pharma co-developed the drug. Meanwhile, Stivarga, Bayer’s therapy for late-stage colorectal cancer, joined Zaltrap in September 2012 in the arsenal of therapies against colorectal cancer treatment capable of extending patient’s lives.

Also in September, Pfizer delivered Bosulif for chronic myelogenous leukemia, a therapy that the FDA recognized as reflecting an improvement in the treatment of the leukemia based on a better understanding of the molecular basis of the disease.

The 20% of breast cancer patients with HER2 positive cancers gained access to Roche’s Perjeta in June 2012. It joined Vertex Pharmaceuticals’ Kalydeco, a targeted therapy for cystic fibrosis patients who have a specific gene mutation that drives their disease approved in January 2012, in bolstering the roster of personalized therapies approved by the FDA during the year.

Pfizer won approval for Xeljanz, the first oral disease-modifying drug for rheumatoid arthritis in more than a decade.

There were also some notable innovations in 2012 in the way approved drugs were made. Pfizer, along with its partner Protalix Biotherapeutics, won approval for the Gaucher disease drug Elelyso, the first drug to be manufactured using genetically engineered plant cells. Novartis, won approval for Flucelvax, the first seasonal influenza vaccine licensed in the United States produced using cultured animal cells instead of fertilized chicken eggs.

In a time that the FDA is striving to project leadership, the agency’s banner year of approvals give it positive momentum headed into 2013.