Patients may be asking about reports in the lay press that exenatide (Byetta - Med Lett Drugs Ther 2005; 47:45), a synthetic peptide that stimulates release of insulin from pancreatic beta cells, has been used by diabetics and non-diabetics to lose weight. Approved by the FDA to improve glycemic control in patients with type 2 diabetes not controlled by metformin, a sulfonylurea or both, it is given by subcutaneous injection before the morning and evening meals. In clinical trials, some diabetic patients treated with the drug lost weight. No data are available on use in non-diabetics....

The antibiotic aztreonam is now available as an inhalation solution (Cayston – Gilead) to improve respiratory symptoms in cystic fibrosis (CF) patients ≥7 years old colonized with Pseudomonas aeruginosa. It is the second inhaled antibiotic to be FDA-approved for this indication in CF patients; the aminoglycoside tobramycin (Tobi) was the first. Inhaled antibiotics offer the advantage of high airway concentrations while minimizing systemic side effects.

The FDA has approved incobotulinumtoxinA (Xeomin – Merz) for treatment of cervical dystonia and blepharospasm in adults. It has been commercially available in Germany since 2005. Several formulations of botulinum toxin type A (Botox; Dysport) and type B (Myobloc) are already marketed for treatment of cervical dystonia. Botox is also approved for treatment of blepharospasm.

The FDA has approved tesamorelin (Egrifta – EMD Serono), an injectable synthetic analog of growth-hormone- releasing factor (GRF), for reduction of excess abdominal fat in patients with lipodystrophy associated with HIV infection. Growth hormone (somatropin – Serostim; EMD Serono) has been available for years for treatment of HIV wasting.

The FDA has approved the opioid agonist hydromorphone in a once-daily extended-release (ER) oral tablet formulation (Exalgo – Covidien) for the management of moderate to severe pain in opioid-tolerant patients requiring continuous, long-term therapy. Another hydromorphone ER formulation (Palladone – Purdue) was available previously, but was withdrawn from the market because taking it with alcohol could interfere with the extended-release mechanism and lead to rapid release of potentially lethal amounts of the drug ("dose-dumping").

A new once-daily formulation of gabapentin (Gralise – Depomed) has been approved by the FDA for treatment of postherpetic neuralgia (PHN). Immediate-release (IR) gabapentin (Neurontin, and others), which has been available in the US since 1994, is also approved for this indication, but is taken three times a day. Extended-release gabapentin enacarbil (Horizant) was recently approved by the FDA for treatment of restless legs syndrome.

The FDA has approved glucarpidase (Voraxaze – BTG International) for treatment of toxic plasma methotrexate concentrations (>1 micromole per liter) in patients with delayed methotrexate clearance due to impaired renal function. Glucarpidase has been available in the US since 2007 under a compassionate use open-label treatment protocol (Clinical Trials and Consulting Services, 1-877-398-9829), which will remain in effect until the drug becomes commercially available later this year. There is currently a shortage of IV methotrexate in the US.

Anascorp (Rare Disease Therapeutics), an intravenously administered antivenom derived from horse serum, has been approved by the FDA for treatment of clinical signs of scorpion envenomation.

Pertuzumab (Perjeta – Roche/Genentech), a humanized monoclonal antibody, has been approved by the FDA for use in combination with trastuzumab (Herceptin) and docetaxel (Taxotere, and others) for first-line treatment of human epidermal growth factor receptor 2 (HER2)-positive metastatic breast cancer.

Linaclotide (lin´´ a kloe´ tide; Linzess – Ironwood/Forest), a guanylate cyclase-C receptor agonist, was recently approved by the FDA for oral treatment of chronic idiopathic constipation (CIC) and irritable bowel syndrome with constipation (IBS-C) in adults. It is the second drug approved for this indication; lubiprostone (Amitiza), a metabolite of prostaglandin E1, was the first.