1. 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."
“Xeljanz provides a new treatment option for adults suffering from the debilitating disease of RA who have had a poor response to methotrexate,” said Badrul Chowdhury, M.D., Ph.D., director of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA’s Center for Drug Evaluation and Research.
2. 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."
The medicines, which are man-made antibodies, lowered "bad" cholesterol by more than 40 percent and up to 80 percent in small Phase 2 studies released by Amgen Inc and Pfizer Inc on Monday at the annual scientific meeting of the American Heart Association in Los Angeles.
3. Kalydeco (ivacaftor)
"The first drug designed to treat the underlying cause of cystic fibrosis won approval in 2012 from both European and US regulators."
“Kalydeco is an excellent example of the promise of personalized medicine – targeted drugs that treat patients with a specific genetic makeup,” said FDA Commissioner Margaret A. Hamburg, M.D. “The unique and mutually beneficial partnership that led to the approval of Kalydeco serves as a great model for what companies and patient groups can achieve if they collaborate on drug development.”
4. Glybera (alipogene tiparvovec)
"European regulators in November approved the first gene therapy product to be used in the Western world."
The benefits with Glybera are its ability to allow expression of the lipoprotein lipase protein in deficient patients suffering from severe or multiple pancreatitis attacks.
5. 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."
The two new studies, published online in The New England Journal of Medicine, found that the drug BG-12, developed by Biogen Idec, reduced relapse rates in patients with relapsing M.S. by about 50 percent.
6. T-DM1 (trastuzumab emtansine) [for advanced HER2-positive breast cancer]
"In November, the FDA granted priority-review designation to an antibody-drug conjugate developed in a partnership between Roche and ImmunoGen."
“We believe that antibody-drug conjugates have the potential to change the future treatment of cancer, and we look forward to working with regulatory authorities in the hope of bringing another potential treatment option to people with HER2-positive metastatic breast cancer.”
7. 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"
“Today’s approval of Stribild will provide physicians and their patients an effective new single tablet treatment option for individuals starting HIV therapy for the first time.”
"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."
"Odanacatib works differently than other treatments for osteoporosis by targeting cat-K, a specific enzyme within bone cells," said Albert Leung, M.D., Ph.D., executive director, clinical research, Merck Research Laboratories.
9. Elelyso (taliglucerase alfa)
"The first pharmaceutical manufactured using genetically engineered plant cells, an enzyme replacement therapy for the treatment of Gaucher’s disease from Protalix BioTherapeutics and Pfizer, won approval this year from regulators in both the US and Israel. Yet the European Medicines Agency (EMA) rejected the drug in November because a competing agent from Shire has orphan market exclusivity in the EU until 2020."
Following the CHMP recommendation, on 29 October 2012, the European Commission adopted a decision refusing the granting of a marketing authorisation
10. Belviq (lorcaserin) and Qsymia (phentermine/topiramate)
"For the first time in over a decade, the FDA approved new weight-loss pills: Belviq, a serotonin receptor activator from Arena Pharmaceuticals and Eisai, and Qsymia, a combination of an appetite suppressant and an anticonvulsant from Vivus. Across the Atlantic, however, the EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended against approving Vivus’s agent, citing potential cardiovascular and central nervous system effects."
Why has it been 13 years since the last diet drug was approved?
Eric Colman, M.D., deputy director of DMEP, says that drug companies have been testing potential new weight loss drugs, but none had proven effective and safe for consumers until now.
11. CETP inhibitors
"In May, Roche announced plans to discontinue development of its cholesteryl ester transfer protein (CETP) inhibitor dalcetrapib after an interim review of the company’s phase 3 trial failed to show any signs of clinically meaningful efficacy."
“Lowering cardiovascular risk beyond that which is achieved with intensive statin treatment is a very challenging goal and while we have always stated that dalcetrapib is a high-risk project, we are disappointed by the fact that this drug didn't provide benefit to the patients in our study,” said Hal Barron M.D., Chief Medical Officer and Head, Global Product Development.
12. Dengue vaccine
"The world’s most advanced vaccine against dengue fever proved safe and 60–90% effective against three of the four genetic forms of the dengue virus, scientists reported in September. However, in the trial of 4,000 schoolchildren in Thailand, the Sanofi product offered no protection from the most common dengue strain circulating in the country at the time."
These data show for the first time that a safe vaccine against dengue is possible.
13. Tresiba (insulin degludec) and Ryzodeg (insulin degludec/insulin aspart)
"Novo Nordisk’s ultra-long-acting insulins, Tresiba and Ryzodeg, won approval from Japanese regulators and received positive reviews from Europe’s CHMP and an FDA advisory committee this year. [...] However, they have also been linked to an increased risk of heart disease, which led the FDA panel to vote unanimously in favor of Novo conducting further cardiovascular outcome studies."
The two new products are expected to make headway in the long-acting basal insulin sector, currently dominated by Sanofi's $5bn-a-year blockbuster product Lantus (insulin glargine), thanks to a low tendency to cause low blood sugar.
14. Vyndaqel (tafamidis meglumine)
"The FDA rejected Pfizer’s rare-disease drug in June and asked for another trial to demonstrate that the selective stabilizer of the transthyretin protein helps patients with a neurodegenerative disease known as transthyretin familial amyloid polyneuropathy, which affects an estimated 8,000 people worldwide."
Pfizer said the FDA has requested the completion of a second efficacy study to establish substantial evidence of effectiveness prior to an approval. The Agency also asked for additional information on the data within the current tafamidis NDA.
15. Warfarin replacements
"Injury lawsuits began to mount this year against Boehringer Ingelheim’s blood thinner Pradaxa (dabigatran), although the FDA said in November that rates of gastrointestinal bleeding and brain hemorrhage were no higher in people taking the drug than in those on warfarin."
FDA has not changed its recommendations regarding Pradaxa. Pradaxa provides an important health benefit when used as directed. Healthcare professionals who prescribe Pradaxa should carefully follow the dosing recommendations in the drug label, especially for patients with renal impairment (when kidneys don’t function normally) to reduce the risk of bleeding. Patients with atrial fibrillation should not stop taking Pradaxa without first talking to their healthcare professional. Stopping use of anticoagulant medications such as Pradaxa can increase the risk of stroke. Strokes can lead to permanent disability and death.
"Bristol-Myers Squibb halted development of this hepatitis C drug in August after a trial participant died of heart failure following drug administration."
While the cause of these unexpected events, which involve heart and kidney toxicity, has not been definitively established, the Company has determined that it is in the best interest of patients to halt development of BMS-986094.
17. Bapineuzumab and solanezumab
"Two antibody drugs designed to target the amyloid-β plaques in the brains of people with Alzheimer’s disease both flunked in late-stage trials this year. "
Scientists and researchers widely believe any treatment of the disease should begin early or even before a high-risk patient begins to notice the symptoms. As such, many of these researchers and scientists had expected the late-stage trials to fail, saying the companies were treating patients who had already suffered damage to their brains.
"A kinase inhibitor designed to target the tau deposits associated with Alzheimer’s disease also flopped this year."
It is a blow for Zeltia given that in experimental models, tideglusib (also known as NP-12), the only GSK-3 inhibitor nowadays in clinical development for AD, "has demonstrated ability to act on the most relevant histopathological lesions associated with AD-reducing tau protein phosphorylation, accumulation of amyloid plaques in the brain, oligomeric amyloid toxicity, neuritic dystrophies, and hippocampal and entorhinal cortex neuron loss", the company says.
19. Bardoxolone methyl
"In October, doctors involved in testing a new chronic kidney disease therapy from Abbott Laboratories and Reata Pharmaceuticals received an email telling them to contact their patients and tell them to stop taking the drug."
"This decision was made based upon a recommendation of the Independent Data Monitoring Committee (IDMC) to stop the trial 'for safety concerns due to excess serious adverse events and mortality in the bardoxolone methyl arm'," a statement from Reata said.
"AstraZeneca’s and BTG’s experimental sepsis agent became the latest in a long line of drugs to prove no better than placebo in human testing."
"These results are obviously disappointing, as the treatment of severe sepsis remains a major unmet need. Our core business and trading continue on track, as described in our recent interim management statement," said BTG's Chief Executive Officer Louise Makin.