Another year, another round of approvals, mixed reviews and high-profile failures. We look back on which medicines made the headlines.
GREEN LIGHT
Xeljanz (tofacitinib)
In November, the US Food and Drug Administration (FDA) approved the first oral disease-modifying drug for rheumatoid arthritis in more than a decade. Pfizer’s twice-daily pill—which works by blocking enzymes, called Janus kinases, that are involved in joint inflammation—is also being tested as a treatment for psoriasis, colitis and other diseases.
PCSK9 inhibitors
A new class of agents directed against proprotein convertase subtilisin/kexin type 9 (PCSK9), a key regulator of cholesterol metabolism, is moving into the last stage of clinical development. In phase 2 trials reported this year, experimental PCSK9 inhibitors from Amgen, Pfizer and Regeneron Pharmaceuticals (in partnership with Sanofi) each reduced low-density lipoprotein levels by 40–60%, on average, in various patient populations.
Kalydeco (ivacaftor)
The first drug designed to treat the underlying cause of cystic fibrosis won approval in 2012 from both European and US regulators. Although the approved therapy will only benefit those 4% of cystic fibrosis sufferers who have a specific genetic defect known as the G551D mutation, Vertex Pharmaceuticals reported interim data this year showing that the drug, marketed as Kalydeco, helped improve lung function in people with the most common mutation associated with the disease when administered in combination with an experimental agent called VX-809.
Glybera (alipogene tiparvovec)
European regulators in November approved the first gene therapy product to be used in the Western world. UniQure’s Glybera relies on an adeno-associated viral vector to deliver a functional copy of the gene that is deficient in individuals with a rare lipid-processing disease called lipoprotein lipase deficiency, which affects around one in a million people worldwide.
Aubagio (teriflunomide) and BG-12 (dimethyl fumarate)
People with multiple sclerosis now have a second oral drug option—and a third could be just around the corner. In September, the FDA approved Sanofi’s once-a-day tablet Aubagio for the treatment of relapsing forms of the disease. A week later, Biogen Idec posted positive phase 3 trial data for its own oral agent, BG-12. Both pills face competition from Novartis’s Gilenya (fingolimod), which hit the market in 2010.
T-DM1 (trastuzumab emtansine)
In November, the FDA granted priority-review designation to an antibody-drug conjugate developed in a partnership between Roche and ImmunoGen. Earlier in the year, phase 3 data showed that the therapy—a combination of the monoclonal antibody Herceptin (trastuzumab) linked to the cytotoxic agent mertansine—prolonged median survival by about six months longer than standard therapy did among women with advanced HER2-positive breast cancer.
Truvada (enofovir/emtricitabine) and Stribild
Gilead’s combination antiretroviral pill Truvada received the blessing of US authorities in July as a strategy for reducing the risk of HIV infection among adults at high risk of sexually acquired infection. A month later, Gilead scored another FDA blessing for its four-in-one drug Stribild, which packages Truvada together with two newer ingredients, elvitegravir and cobicistat, to combat the virus in HIV-positive individuals who have not received prior treatment.
Odanacatib
In July, Merck announced an early close to a 16,000-person clinical trial of odanacatib after a review of early results reportedly showed that the experimental osteoporosis drug significantly reduced the risk of bone fractures among post-menopausal women. Phase 2 data released this year similarly demonstrated that the investigational cathepsin K inhibitor increased bone mineral density by significantly more than standard therapy. Merck plans to file for regulatory approval early next year.
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