The US Food and Drug Administration recently announced (pdf) that it had cleared 35 new drugs during 2012, of which 31 were novel therapies. This is in addition to the literally hundreds of approvals for changes in already approved drugs for changes in packaging, manufacturing, and dozens of other reasons.
In no particular order, here are the top 10 most interesting of the approvals based on my subjective viewpoint, which includes innovativeness, seriousness of disease, and other random factors. In others, no different in importance than all those end-of-year top 10 movie lists. So here we go:
- AUBAGIO (teriflunomide). Manufactured by Sanofi-Aventis, it is indicated relapsing forms of multiple sclerosis (MS). According to research, eriflunomide inhibits rapidly dividing cells, including activated T cells, which are thought to drive the disease process in MS. Furthermore, it may decrease the risk of infections compared to chemotherapy-like drugs because of its more-limited effects on the immune system.
- KALYDECO (ivacaftor). Manufactured by Vertex Pharmaceuticals, it is indicated for cystic fibrosis (CF) patients with G551D gene mutation, . One of the most innovative drugs for patients suffering from CF, it directly addresses the genetic defect that causes CF by improving flow through ion channels and reducing the symptoms. It’s an incredibly expensive drug, for patients with this gene mutation, it is literally a life-saver.
- ELELYSO (taliglucerase alfa). Manufactured by Israeli-base Protalix Biotherapeutics. It is indicated for type-1 Gaucher’s disease, a rare genetic disorder in which a fatty substance (lipid) accumulates in cells and certain organs. The drug is produced in genetically modified (GMO) carrots to reduce contamination and improve efficacy. Let’s hope that the anti-GMO crowd don’t decide to fight this life-saving drug because of their anti-scientific beliefs about GMO crops.
- KYPROLIS (carfilzomib). Manufactured by Onyx Pharmaceuticals it is indicated for treatment of multiple myeloma, a cancer of plasma cells, a type of white blood cell normally responsible for producing antibodies. Carfilzomid, which inhibits the growth of the cancer, was fast-tracked by the FDA based on highly promising results which showed that it was safe, that it didn’t interact with other drugs, and it had improved outcome against the cancer relative to currently available therapies.
- MENHIBRIX (Meningococcal serogroups C and Y, Haemophilus influenzae type b, and Tetanus Toxoid Conjugate Vaccine). Manufactured by GlaxoSmithKline, it is a combination vaccine, indicated for active immunization to prevent invasive disease caused by Neisseria meningitides serogroups C and Y and Haemophilus influenzae type b.
- STENDRA (avanafil). Manufactured by Vivus Inc, and it is indicated for the treatment of erectile dysfunction (ED). I know, what’s the big deal with another ED drug? Well, this one has fewer side effects and is faster working. And despite the beliefs that people take them just for fun, for those men with reduced blood flow as a result of diabetes, vascular disease, and other real medical conditions, ED medications can improve the quality of life and can reduce depression.
- SURFAXIN (lucinactant). Manufactured by Discovery Laboratories, it is indicated to prevent Infant Respiratory Distress Syndrome (RDS) in premature infants, but it also can be used up to two years of age and is specified for children who are diagnosed with acute respiratory failure following exposure to a pathogen such as RSV or influenza, including H1N1. The best news about this new drug is that it may have improved outcomes compared to previously available drugs, but also may have strong advantages in reduced costs and timelines of treatment.
- BOSULIF (bosutinib). Manufactured by Pfizer, it is indicated for the treatment of adult patients with chronic, accelerated, or blast phase Philadelphia chromosome-positive (Ph+) chronic myelogenous leukemia (CML) with resistance, or intolerance to prior therapy. This drug is part of the armamentarium of medications to treat CML.
- EYLEA (aflibercept). Manufactured by Regeneron Pharmaceuticals, it is indicated for wet, age-related macular degeneration, a medical condition which usually affects older adults and results in a loss of vision in the center of the visual field (the macula) because of damage to the retina.
- STRIBILD (elvitegravir, cobicistat, emtricitabine, tenofovir). Manufactured by Gilead Sciences, it is indicated for treatment of HIV infections and AIDS. The cobicistat component increases the effectiveness of the combination by inhibiting liver enzymes that metabolize the other components.
These new drugs (out of many that got approved) save lives. They make patient’s quality of life higher. And the FDA along with the pharmaceutical companies work to bring out new pharmaceuticals for rare diseases, or those diseases that do not get a lot of research effort, to help people. So whenever I hear that oft-repeated myth that “Big Pharma never helps anyone”, let’s show them 2012. Or 2011. Or 1998. Or whatever year you want over the past 20.
- Aviezer D, Brill-Almon E, Shaaltiel Y, Hashmueli S, Bartfeld D, Mizrachi S, Liberman Y, Freeman A, Zimran A, Galun E. A plant-derived recombinant human glucocerebrosidase enzyme–a preclinical and phase I investigation. PLoS One. 2009;4(3):e4792. doi: 10.1371/journal.pone.0004792. Epub 2009 Mar 11. PubMed PMID: 19277123; PubMed Central PMCID: PMC2652073.
- Garner SS, Cox TH. Lucinactant: new and approved, but is it an improvement? J Pediatr Pharmacol Ther. 2012 Jul;17(3):206-10. doi: 10.5863/1551-6776-17.3.206. PubMed PMID: 23258962; PubMed Central PMCID: PMC3526923.
- Guardia CG, Moya FR, Sinha S, Simmons PD, Segal R, Greenspan JS. A pharmacoeconomic analysis of in-hospital costs resulting from reintubation in preterm infants treated with lucinactant, beractant, or poractant alfa. J Pediatr Pharmacol Ther. 2012 Jul;17(3):220-7. doi: 10.5863/1551-6776-17.3.220. PubMed PMID: 23258964; PubMed Central PMCID: PMC3526925.
- Jones AM, Helm JM. Emerging treatments in cystic fibrosis. Drugs. 2009 Oct 1;69(14):1903-10. doi: 10.2165/11318500-000000000-00000. PubMed PMID: 19747007.
- Magne D, Mézin F, Palmer G, Guerne PA. The active metabolite of leflunomide, A77 1726, increases proliferation of human synovial fibroblasts in presence of IL-1beta and TNF-alpha. Inflamm Res. 2006 Nov;55(11):469-75. PubMed PMID: 17122964.
- Perelman MA. Erectile dysfunction and depression: screening and treatment. Urol Clin North Am. 2011 May;38(2):125-39. doi: 10.1016/j.ucl.2011.03.004. Review. PubMed PMID: 21621079.
- Wang Z, Yang J, Kirk C, Fang Y, Alsina M, Badros A, Papadopoulos K, Wong A, Woo T, Bomba D, Li J, Infante JR. Clinical pharmacokinetics, metabolism, and drug-drug interaction of carfilzomib. Drug Metab Dispos. 2013 Jan;41(1):230-7. doi: 10.1124/dmd.112.047662. Epub 2012 Nov 1. PubMed PMID: 23118326.
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