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New Drugs, Indications, Dosage Forms, and Approval Recommendations

  • Acetaminophen intravenous (Ofirmev) received a complete response letter in February from the FDA related to facility manufacturing deficiencies. The FDA did not cite any safety or efficacy issues and is not requiring any additional studies to be done prior to approval.1 The third-party manufacturer has submitted its response to the FDA and is ready to resubmit their new drug application (NDA) for this agent. It is being investigated to treat fever and pain in adults and children.2
  • Ciprofloxacin dry powder inhaler (DPI) has received orphan drug status from the FDA for treating pulmonary infections in cystic fibrosis (CF) patients.3,4 It is in clinical trials to determine if it can improve pulmonary function in CF patients with Pseudomonas aeruginosa infections.
  • Carglumic acid (Carbaglu) has been approved by the FDA to treat the metabolic disorder N-acetylglutamate synthetase (NAGS) deficiency.5 NAGS deficiency is an extremely rare genetic disorder that presents shortly after birth. It results in hyperammonemia, and can be fatal if not rapidly detected and managed. Carglumic acid treats the hyperammonemia within three days, with a lowering of the ammonia level within 24 hours. In clinical trials, a small number of patients (n=23) received the drug from six months to 21 years; the majority of patients were able to maintain normal ammonia levels long-term with continued treatment. It is recommended that carglumic acid only be administered by physicians who have experience dealing with metabolic disorders. The starting dose is between 100 mg/kg/day and 250 mg/kg/day for treatment of acute hyperammonemia. Using other agents to lower the ammonia level during acute episodes is recommended. Dosing should be based on the ammonia level and the patient’s symptoms.
  • CK-2017357 has received orphan drug status for treating amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease.6
  • Desirudin injection (Iprivask), a direct thrombin inhibitor, has been approved by the FDA for the prevention of DVT.7 In clinical trials, it was superior to enoxaparin and unfractionated heparin for preventing proximal DVT and prevention of major venous thromboembolic events following elective hip replacement surgery. Desirudin is administered as a fixed subcutaneous dose. It does not cause thrombocytopenia, is relatively short-acting, and is easy to monitor. Some of the adverse reactions in clinical trials were thrombosis, hypotension, lower-extremity edema, fever, decreased hemoglobin level, and hematuria.8 Also known as Revasc, this medication has been available in Europe for more than 10 years.
  • Doxepin tablets (Silenor) have been approved by the FDA for the treatment of short-term and chronic insomnia distinguished by difficulty with sleep maintenance in adults and elderly patients.9 Sleep maintenance includes difficulty staying asleep, waking up too much or too early, and not being able to fall back asleep. In clinical trials, adverse reactions were similar to placebo, there was a low-therapy discontinuation rate, and no evidence of amnesia, tolerance, or complex sleep behaviors such as sleep eating or sleep driving.10 It will be available in 3-mg and 6-mg tablets. It is not designated as a controlled substance.
  • GVAX pancreas vaccine has received orphan drug status as a potential treatment for pancreatic cancer.11 It also is being investigated for other cancers, including those of the breast and for leukemias.
  • Ritonavir (Norvir) has been approved by the FDA in a new formulation, which is heat-stable and can be stored at room temperature rather than in the refrigerator.12 The rate of drug absorption with the new formulation is different but does not require a dosage change.
  • Somatropin [rDNA origin] prefilled injection pen (Norditropin FlexPro) has been approved by the FDA to treat adults and children with growth hormone disorders.13 The pen has an audible click and does not require any reconstitution or cartridge loading. After initial use, the pen can be left at room temperature for up to three weeks without worry of drug degradation.

Pipeline

  • Agalsidase (Replagal) has received fast-track status from the FDA for treating Fabry disease.14 It is an enzyme replacement therapy.
  • Exenatide LAR (Bydureon), the once-weekly version of exenatide (Byetta), is in final discussions at the FDA. The FDA has asked for additional information related to the product label, risk mitigation, and manufacturing, which the manufacturer is addressing.15
  • Insulin powder for inhalation, ultra-rapid-acting (Afrezza), has been reviewed by the FDA, and the agency has requested additional information related to safety and labeling.16
  • A combination therapy of saxagliptin/metformin has been submitted to the FDA as a once-daily treatment of Type 2 diabetes mellitus as an adjunct to diet in adults who cannot adequately control their diabetes on metformin monotherapy, or in treatment-naïve patients.17
  • Vilanterol/fluticasone is a combination of the inhaled corticosteroid fluticasone and the long-acting beta-agonist (LABA) vilanterol, which is currently in Phase 3 clinical trials for treating asthma.18 The trial will compare the combination’s efficacy and safety to fluticasone/salmeterol (Advair). TH

Michele B. Kaufman, PharmD, BSc, RPh, is a freelance medical writer based in New York City and a clinical pharmacist at New York Downtown Hospital.

References

  1. Cadence pharmaceuticals receives complete response letter from FDA for intravenous acetaminophen NDA. Cadence Pharmaceuticals website. Available at: http://cadx.client.shareholder.com/releasedetail.cfm?ReleaseID=444303. Accessed March 23, 2010.
  2. Cadence pharmaceuticals reports fourth quarter and full year 2009 financial results. Cadence Pharmaceuticals website. Available at: http://files.shareholder.com/downloads/CADX/874963043x0x359109/1cf00f72-0872-4d4e-b27d-bde1c03d625a/CADX_News_2010_3_15_General_Releases.pdf. Accessed March 23, 2010.
  3. Bayer lung infection drug gets orphan status. Forbes website. Available at: http://www.forbes.com/feeds/ap/2010/03/11/business-energy-us-bayer-healthcare-orphan-drug_7427368.html. Accessed March 23, 2010.
  4. FDA grants orphan status for Bayer’s ciprofloxacin for cystic fibrosis. The Pharma Letter website. Available at: http://www.thepharmaletter.com/file/4e4fb33313eddf122cdb730e3ea71840/fda-grants-orphan-status-for-bayers-ciprofloxacin-for-cystic-fibrosis.html. Accessed March 23, 2010.
  5. Burgess S. FDA approves drug to treat condition that causes elevated ammonia levels. U.S. Food and Drug Administration website. Available at: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm205150.htm. Accessed March 23, 2010.
  6. UPDATE 1: Cytokinetics’ Lou Gehrig’s drug gets orphan status. Reuters website. Available at: http://www.reuters.com/assets/print?aid=USSGE6290J820100310. Accessed March 23, 2010.
  7. Iprivask available for prevention of deep vein thrombosis. Monthly Prescribing Reference website. Available at: http://www.empr.com/iprivask-available-for-prevention-of-deep-vein-thrombosis/article/164779/. Accessed March 23, 2010.
  8. First direct thrombin inhibitor for DVT prevention now available from Canyon Pharmaceuticals. Canyon Pharmaceuticals website. Available at: http://www.canyonpharma.com/newsexpand.aspx?id=7. Accessed March 23, 2010.
  9. FDA approves Silenor. Drugs.com website. Available at: http://www.drugs.com/newdrugs/somaxon-announces-fda-approval-silenor-doxepin-insomnia-2070.html?printable=1. Accessed March 23, 2010.
  10. UPDATE 1: Somaxon gets FDA nod for insomnia drug, shares soar. Reuters website. Available at: http://www.reuters.com/assets/print?aid=USSGE62H0KI20100318. Accessed March 21, 2010.
  11. BioSante announces FDA orphan drug designation for GVAX pancreatic cancer vaccine. The New York Times website. Available at: http://markets.on.nytimes.com/research/stocks/news/press_release.asp?docTag=201003150755BIZWIRE_USPRX____BW5298&feedID=600&press_symbol=64917. Accessed March 23, 2010.
  12. Abbott receives U.S. FDA approval for heat-stable Norvir (ritonavir) tablets. Abbott website. Available at: http://www.abbott.com/global/url/pressRelease/en_US/60.5:5/Press_Release_0820.htm. Accessed March 23, 2010.
  13. Novo Nordisk receives FDA approval for Norditropin FlexPro for growth hormone treatment. Novo Nordisk website. Available at: http://press.novonordisk-us.com/index.php?s=43&item=239. Accessed March 23, 2010.
  14. Dane L. Shire receives FDA fast-track designation for Replagal. FirstWord website. Available at: http://www.firstwordplus.com/Fws.do?articleid=97AF48EBA7054EF0857F055877E82B2C&logRowId=352323. Accessed March 23, 2010.
  15. Amylin shares up on FDA response for diabetes drug. Forbes website. Available at: http://www.forbes.com/feeds/ap/2010/03/15/business-health-care-us-amylin-mover_7435579.html. Accessed March 23, 2010.
  16. Russell J. MannKind drug fails to win FDA approval. Los Angeles Business Journal website. Available at: http://labusinessjournal.com/article.asp?aid=4998235.0597195.1894204.8500584.7421642.713. Accessed March 23, 2010.
  17. U.S. Food and Drug Administration accepts NDA for once-daily fixed dose combination of Onglyza (saxagliptin) and extended-release metformin for the treatment of type 2 diabetes mellitus in adults. AstraZeneca website. Available at: http://www.astrazeneca-us.com/about-astrazeneca-us/newsroom/product/8804120?itemId=8804120#. Accessed March 23, 2010.
  18. Dennis M. GlaxoSmithKline begins late-stage clinical programme for asthma drug Relovair. FirstWord website. Available at: http://www.firstwordplus.com/Fws.do?articleid=E256469FBD8F4A2F80C5DD3E844CC1E1&logRowId=356423. Accessed March 23, 2010.

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