Sodium phenylbutyrate, an "orphan drug,"has recently been marketed for the treatment of patients with urea cycle disorders caused by a deficiency in one of the following hepatic enzymes: carbamyl phosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). Urea cycle disorders are rare; they occur in one of every 10,000 births.

The FDA has approved a conjugated polysaccharide vaccine, Menactra (Sanofi-Pasteur), for protection against disease caused by Neisseria meningitidis in people 11-55 years old, and the manufacturer has applied for approval for use in children 2 to 10 years old. An unconjugated meningoccocal polysaccharide vaccine (Menomune - Sanofi-Pasteur) has been licensed in the US since 1981.

As cocaine continues to be widely available in the USA (Medical Letter, 28:69, 1986), new problems associated with its use continue to emerge. Some recent reports indicate that use of the drug during pregnancy can damage the fetus, lead to abortion or premature labor, and cause toxic effects in the newborn infant.

A reader asked whether healthcare providers should avoid prescribing the angiotensin receptor blocker (ARB) olmesartan medoxomil (Benicar, and others) because it can cause severe GI adverse effects.In 2013, the FDA warned that olmesartan can cause sprue-like enteropathy, a condition characterized by intestinal villous atrophy, severe chronic diarrhea, and significant unintended weight loss. The warning was based on 23 cases of serious sprue-like enteropathy associated with use of olmesartan, some occurring years after starting the drug. All patients improved after stopping olmesartan; 10 had a...

The FDA has approved the oral tyrosine kinase inhibitor neratinib (Nerlynx – Puma Biotechnology) for extended adjuvant treatment of adults with early-stage, human epidermal growth factor receptor 2 (HER2)-positive breast cancer, following adjuvant trastuzumab (Herceptin)-based therapy. HER2 is overexpressed in about 20% of breast cancers. Up to 30% of early-stage, HER2-positive breast cancer cases treated with trastuzumab-based adjuvant therapy recur.

The oral kinase inhibitor capivasertib (Truqap – AstraZeneca), a first-in-class AKT inhibitor, has been approved by the FDA for use in combination with the selective estrogen receptor degrader (SERD) fulvestrant (Faslodex, and generics) for treatment of hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer in adults with one or more PIK3CA/AKT1/PTEN-alterations who had disease progression on at least one endocrine-based regimen for metastatic disease or recurrence on or within 12 months of completing...

Development of abuse-deterrent opioid products, including reformulation of existing products, has become a priority for drug manufacturers and public health advocates. Three available opioid formulations, OxyContin (Purdue), Embeda (Pfizer), and Hysingla ER (Purdue), now include claims of abuse deterrence in their package inserts.

The FDA has approved sacituzumab govitecan-hziy (Trodelvy – Immunomedics), a trophoblast cell-surface antigen-2 (Trop-2)-directed antibody and topoisomerase inhibitor conjugate, for treatment of adults with metastatic triple-negative breast cancer who have received ≥2 prior therapies for metastatic disease. It is the first Trop-2-directed antibody-drug conjugate to become available in the US.

Four new drugs have become available in the USA for HIV infection or its complications. Nevirapine (Viramune - Roxane), intravenous cidofovir (Vistide - Gilead), and amphotericin B oral suspension (Fungizone Oral Suspension - Bristol-Myers Squibb) have been approved by the US Food and Drug Administration; nelfinavir (Viracept - Agouron) is currently available only through an "Expanded Access Program."

The FDA has approved voretigene neparvovec-rzyl intraocular suspension (Luxturna – Spark), an adeno-associated virus vector-based gene therapy, for treatment of confirmed biallelic RPE65 mutation-associated retinal dystrophy in patients who have viable retinal cells. It is the first gene replacement therapy approved in the US that targets a disease caused by mutations in a specific gene and the first pharmacologic treatment to be approved for this disorder.