Valoctocogene roxaparvovec-rvox (Roctavian – Biomarin), an adeno-associated virus (AAV) vector-based gene therapy, has been approved by the FDA for one-time treatment of severe hemophilia A in adults without pre-existing antibodies to AAV serotype 5. It is the first gene therapy to be approved in the US for treatment of hemophilia A.

Isatuximab-irfc (Sarclisa – Sanofi), a CD38-directed cytolytic antibody, has been approved by the FDA for treatment of newly diagnosed multiple myeloma in adults who are not eligible for autologous stem cell transplantation (ASCT). The drug was approved earlier for treatment of relapsed or treatment-refractory multiple myeloma.

Vimseltinib (Romvimza – Deciphera), an oral kinase inhibitor, has been approved by the FDA for treatment of symptomatic tenosynovial giant cell tumor in adults for whom surgical resection would cause worsening functional limitation or severe morbidity. The kinase inhibitor pexidartinib (Turalio) was approved for a similar indication in 2019.

View the Flowchart: Rheumatoid Arthritis Treatment

Relyvrio, the fixed-dose combination of sodium phenyl-butyrate and taurursodiol that received accelerated approval for treatment of amyotrophic lateral sclerosis (ALS) in 2022 based on a phase 2 trial showing that it slowed functional decline, has voluntarily been withdrawn from the market. According to the manufacturer (Amylyx), the decision to withdraw Relyvrio was based on the results of a phase 3, 48-week trial (PHOENIX). The change from baseline on the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale, which measures fine motor, gross motor, bulbar, and respiratory...

Two types of oral contraceptives are available in the USA: combination oral contraceptives that contain both an estrogen and a progestin, and progestin-only contraceptives, or "mini-pills."Used as directed, all oral contraceptives marketed in the USA are similarly effective in preventing pregnancy.

Patients with lung cancer may be asking their physicians about gefitinib (ge fi' tye nib; ZD1839; Iressa -- AstraZeneca), because it has been the subject of positive coverage in the media. An inhibitor of the epidermal growth factor receptor (EGFR) tyrosine kinase, this oral drug has not been approved by the FDA, but is in clinical trials in the US for treatment of non-small cell lung cancer (NSCLC) and some other solid tumors. Iressa was recently approved in Japan, and is available from the manufacturer on a "compassionate-use" basis in the US (800-236-9933).

Acetaminophen (paracetamol; Tylenol, and others) taken in overdosage can cause severe hepatic injury and death. Acetaminophen poisoning is treated with (N-)acetylcysteine. Available for many years in the US as a solution for inhalation therapy (Mucomyst, and others), it subsequently became available for oral use and was recently marketed in the US for intravenous (IV) use as Acetadote (Cumberland).

A large mumps outbreak that began in Iowa in December 2005 has spread. About 40% of the cases have been in people 18-25 years old, many of whom are college students and had been vaccinated against the disease.

Health care professionals may be asked about lowvoltage electronic devices now being used to diagnose and/or treat a number of health problems. Most are galvanometers that measure skin resistance to an electric current. The FDA classifies "devices that use resistance measurements to diagnose and treat various diseases" as Class III devices that require FDA approval prior to marketing. None of the devices reviewed here has such approval.