The FDA has approved a 0.016% gel formulation of the nitrogen mustard mechlorethamine (Valchlor – Actelion) for second-line topical treatment of patients with stage IA or IB mycosis fungoides, the most common type of cutaneous T-cell lymphoma. Topical nitrogen mustard has been used off-label for decades for this indication, usually as a pharmacy-compounded ointment, but no clinical trials evaluating its efficacy and safety have been done. Mechlorethamine is also available in an injectable formulation (Mustargen – Recordati) for the same and other indications.FDA approval of Valchlor was...

In 2007, an FDA advisory committee asked that placebo-controlled, dose-ranging trials be conducted to establish the efficacy of the oral decongestant phenylephrine (Sudafed PE, and others), which is sold over the counter (OTC) as a single agent and in combination with other drugs for treatment of cold and allergy symptoms. Phenylephrine replaced pseudoephedrine (Sudafed, and others) in many OTC formulations when access to pseudoephedrine-containing products was restricted in an effort to reduce their use in the synthesis of methamphetamine.CLINICAL STUDIES — In a randomized, open-label,...

The FDA has approved Lonsurf (Taiho Oncology), a combination of the thymidine-based nucleoside analog trifluridine and the thymidine phosphorylase inhibitor tipiracil, for oral treatment of metastatic colorectal cancer. Trifluridine is incorporated into DNA, interfering with DNA synthesis and inhibiting cell proliferation. Tipiracil inhibits the metabolism of trifluridine. The combination is only approved for use in patients who were previously treated with a fluoropyrimidine (fluorouracil or capecitabine), oxaliplatin, irinotecan, an anti-VEGF biological such as bevacizumab, and, if the tumor...

The FDA has approved an oral suspension formulation of nitisinone (Orfadin – Sobi) for treatment of hereditary tyrosinemia type 1 (HT-1). Orfadin has been available in a capsule formulation in Canada since 1994 and in the US since 2002.HT-1 is an autosomal recessive genetic disorder (estimated birth prevalence: 1:100,000) caused by mutations in the gene responsible for formation of fumarylacetoacetate hydrolase (FAH), which catalyzes the last step in tyrosine metabolism. FAH deficiency results in accumulation of upstream toxic metabolites, causing liver and kidney failure and developmental...

The FDA has approved adjunctive treatment with Dartisla ODT (Edenbridge), a new orally disintegrating tablet (ODT) formulation of the anticholinergic drug glycopyrrolate, to reduce symptoms of a peptic ulcer. Glycopyrrolate oral tablets (Robinul, Robinul Forte, and generics) were approved for the same indication in 1961.

Six vaccines are recommended for routine use in adults living in the USA (Guide for Adult Immunization, 2nd ed, Philadelphia:American College of Physicians, 1990). Immunization recommendations for travel outside the USA were published in The Medical Letter, volume 32, page 33, April 6, 1990.

Two new drugs alendronate (Fosamax - Merck) and salmon calcitonin nasalspray (Miacalcin - Sandoz) are now available in the USA for treatment of postmenopausalosteoporosis. A third drug for treatment of osteoporosis, a slow-release fluoridepreparation (Slow Fluoride -Mission Pharmacal), has been recommended for approvalby an advisory committee of the US Food and Drug Administration (FDA). Various formulationsof fluoride have been available in Europe for this indication for many years.

Atorvastatin (Lipitor - Parke-Davis), an HMG-CoA reductase inhibitor (or 'statin'), was recently approved by the US Food and Drug Administration for treatment of primary hypercholesterolemia and mixed dyslipidemia. A single stereoisomer of a pyrrole derivative, the new drug is chemically different from other statins.

Panitumumab (Vectibix - Amgen), a fully human IgG₂ monoclonal antibody that inhibits the epidermal growth factor receptor (EGFR), has been approved by the FDA for treatment of patients with EGFR-expressing metastatic colorectal cancer that has progressed despite standard chemotherapy. It is the second monoclonal antibody EGFR inhibitor to be approved for metastatic colorectal cancer; cetuximab (Erbitux), a human-murine chimeric IgG₁ monoclonal antibody, was approved in 2004.

The FDA has approved Aptensio XR (Rhodes), an extended-release formulation of methylphenidate hydrochloride, for treatment of attention-deficit/hyperactivity disorder (ADHD). The Aptensio XR capsules contain multilayer beads designed to provide both a rapid onset and a long duration of action. This formulation of methylphenidate has been available in Canada as Biphentin since 2006.