title: [Two news drugs (ivacaftor & bedaquiline), one biomarker (florbetapir) and a re-positioned drug (propranolol) on the market].
abstract: Among the new molecular entities approved by the EMEA and the FDA in 2012, four have caught our attention for their significant contribution to the health of patient. First of all, among the notable 2012 approvals, is ivacaftor or Kalydeco®. This is the first treatment that targets one of the gene defects that is underlying cause of cystic fibrosis. This is also an example of the promise of personalized medicine. The benefits with bedaquiline or Sirturo® are its ability to likely provide clinically relevant activity as part of multi-drug regimens against tuberculosis (TB) based on clinical data in multi-drug resistant tuberculosis (MDR TB) patients, who were defined as being at least resistant against the two major tuberculostatic medicines (isaoniazide and rifampicine). On December 2012 and then, on December 2013, the FDA and European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended granting a conditional marketing authorization for Sirturo® (bedaquiline), respectively, for use as part of a combination therapy for pulmonary multidrug resistant tuberculosis in adult patients when an effective treatment regimen cannot otherwise be composed for reasons of resistance or tolerability. Amyvid®, which is a solution for injection that contains the active substance florbetapir (18F), is a radiopharmaceutical that emits low amounts of radiation and works by targeting and attaching to β-amyloid plaques in the brain. This enables doctors to know whether or not significant amount of plaques are present in order to know if the patient is unlikely or not, to have Alzheimer's disease. Finally, the last topics addresses the propranolol, which is a beta-blocker, used alone or together with other medicines to treat high blood pressure. Propranolol is gaining a new lease of life for treating infantile hemangioma.
authors.affiliation: Institut Curie, 26, rue d'Ulm, 75248 Paris cedex 05, France. Electronic address: claude.monneret@curie.fr.
authors.forename: C
authors.initials: C
authors.lastname: Monneret
mesh_terms: D000319:Adrenergic beta-Antagonists / Q000627:therapeutic use*; D000328:Adult; D000544:Alzheimer Disease / Q000000981:diagnostic imaging; D000627:Aminophenols / Q000627:therapeutic use*; D000814:Aniline Compounds; D000995:Antitubercular Agents / Q000627:therapeutic use*; D003550:Cystic Fibrosis / Q000188:drug therapy*; D064687:Diarylquinolines / Q000627:therapeutic use*; D024881:Drug Resistance, Bacterial; D005026:Ethylene Glycols; D006391:Hemangioma / Q000188:drug therapy; D006801:Humans; D007223:Infant; D011433:Propranolol / Q000627:therapeutic use*; D015363:Quinolones / Q000627:therapeutic use*; D011877:Radionuclide Imaging; D019275:Radiopharmaceuticals; D014376:Tuberculosis / Q000188:drug therapy / Q000382:microbiology; D018088:Tuberculosis, Multidrug-Resistant
chemical_list: D000319:Adrenergic beta-Antagonists; D000627:Aminophenols; D000814:Aniline Compounds; D000995:Antitubercular Agents; D064687:Diarylquinolines; D005026:Ethylene Glycols; D015363:Quinolones; D019275:Radiopharmaceuticals; C545203:ivacaftor; C545186:florbetapir; C493870:bedaquiline; D011433:Propranolol
keywords: Alzheimer disease; Bedaquiline; Bédaquiline; Florbetapir F-18; Hémangiomes infantiles; Infantile hemangioma; Ivacaftor; Kalydeco(®); Maladie d’Alzheimer; Mucoviscidose; Mucoviscidosis; Propranolol; Sirturo(®); Tuberculose; Tuberculosis