Pegaspargase (peg as par jase; PEG-L-asparaginase; Oncaspar - Rh ne-Poulenc Rorer), a polyethylene glycol (PEG) conjugate of L-asparaginase, has been approved by the US Food and Drug Administration for treatment of acute lymphoblastic leukemia (ALL), the most common malignancy of childhood. The new drug is recommended only for patients who have had allergic reactions to asparaginase. Asparaginase is available commercially as Elspar, which is derived from Escherichia coli. A formulation derived from Erwinia chrysanthemi is available on an investigational basis (Ogden Bioservices,...

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.

The FDA has approved the neoadjuvant (preoperative) use of pertuzumab (Perjeta – Genentech) in combination with trastuzumab (Herceptin) and docetaxel (Taxotere, and generics) for treatment of locally advanced, inflammatory, or early-stage HER2 (human epidermal growth factor receptor 2)-positive breast cancer patients with tumors >2 cm in diameter or node-positive disease. Pertuzumab in combination with trastuzumab and docetaxel was approved earlier for treatment of HER2-positive metastatic breast cancer. Pertuzumab is the first drug to be approved for neoadjuvant treatment of...

The FDA has issued an Emergency Use Authorization (EUA) for a lower-strength (10 mcg/0.2 mL) formulation of the mRNA-based COVID-19 vaccine manufactured by Pfizer/BioNTech (Comirnaty) for use in children 5-11 years old. The original formulation of (Comirnaty) (30 mcg/0.3 mL) is FDA-licensed for use in persons ≥16 years old and available under an EUA for use in adolescents 12-15 years old.

The FDA has issued an Emergency Use Authorization (EUA) for the long-acting investigational IV monoclonal antibody pemivibart (Pemgarda – Invivyd) for pre-exposure prophylaxis of COVID-19 in persons ≥12 years old (weight ≥40 kg) who have moderate to severe immune compromise and are unlikely to respond adequately to COVID-19 vaccination (see Table 1). Pemgarda is the only drug that is currently authorized in the US for pre-exposure prophylaxis of COVID-19. Tixagevimab/cilgavimab (Evusheld) was previously available under an EUA for this indication, but it lacks activity against...

The FDA has expanded its Emergency Use Authorizations (EUAs) for the mRNA COVID-19 vaccines manufactured by Pfizer/BioNTech (Comirnaty) and Moderna (Spikevax) to allow for their use in children as young as 6 months old. The Pfizer vaccine was previously authorized for use in persons ≥5 years old, and the Moderna vaccine was authorized for use in adults ≥18 years old.

The FDA has approved the subcutaneously injected interleukin (IL)-4 receptor alpha antagonist dupilumab (Dupixent – Sanofi/Regeneron) for treatment of eosinophilic esophagitis (EoE) in patients ≥12 years old who weigh ≥40 kg and treatment of prurigo nodularis in adults. Dupilumab is the first drug to be approved in the US for each of these indications. It is also FDA-approved for treatment of atopic dermatitis, asthma, and chronic rhinosinusitis with nasal polyposis.

Endoscopic injection of sclerosing agents is now widely accepted as a primary treatment for bleeding esophageal varices (J Terblanche et al, N Engl J Med, 320:1393, 1469, May 25 and June 1, 1989). Ethanolamine oleate 5% (Ethamolin - Glaxo), a sclerosing agent that has been available in Canada and abroad for many years, was recently approved for this indication by the US Food and Drug Administration (FDA).