Axatilimab-csfr (Niktimvo – Incyte), a colony stimulating factor-1 receptor-blocking antibody, has been approved by the FDA for intravenous treatment of chronic graft-versus-host disease (cGVHD) after failure of at least 2 lines of systemic therapy in patients weighing ≥40 kg. Corticosteroids are generally the first-line treatment for cGVHD. The oral kinase inhibitors ibrutinib (Imbruvica), ruxolitinib (Jakafi), and belumosudil (Rezurock) are also approved for treatment of cGVHD. Extracorporeal photopheresis has been used off-label.

The FDA has approved Zunveyl (Alpha Cognition), a delayed-release formulation of the prodrug benzgalantamine, for treatment of mild to moderate dementia of Alzheimer's disease (AD). Immediate- and extended-release formulations of the acetylcholinesterase inhibitor galantamine have been available in the US for years for treatment of AD.

On April 23, 2021, on advice from the Advisory Committee on Immunization Practices (ACIP), the FDA and CDC recommended that use of the Johnson & Johnson adenovirus-based COVID-19 vaccine resume despite its association with development of thrombosis with thrombocytopenia syndrome (TTS). Administration of the vaccine had been paused on April 13 because of 6 reports of TTS, but after completing a data review that identified a further 9 cases associated with the formulation, the agencies concluded that the benefits of the vaccine outweighed its risks. About 7.98 million doses of the...

The ophthalmic H1-antihistamine alcaftadine 0.25%, which has been available by prescription since 2011 for use in patients with allergic conjunctivitis, is now available without a prescription as Lastacaft Once Daily Relief (Allergan) for temporary relief of itchy eyes due to pollen, ragweed, grass, animal hair, and dander in adults and children ≥2 years old. It is the third ophthalmic antihistamine to be approved for over-the-counter (OTC) use (see Table 1).

The FDA has approved idecabtagene vicleucel (Abecma – BMS), a B-cell maturation antigen (BCMA)-directed genetically-modified cellular product, for treatment of relapsed or refractory multiple myeloma in adults who received ≥4 prior lines of therapy, including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 monoclonal antibody. Abecma is an individualized cellular product prepared from the patient’s own T cells, which are genetically modified to express chimeric antigen receptors (CAR) and then infused back into the patient. Ciltacabtagene autoleucel...

A reader of our article on the 2024-2025 formulations of the mRNA COVID-19 vaccines manufactured by Pfizer/BioNTech (Comirnaty) and Moderna (Spikevax) asked us to provide more information on the data that supported their licensure.

On February 27, 2021, the FDA issued an Emergency Use Authorization (EUA) for the Johnson & Johnson adenovirus-based vaccine for prevention of COVID-19 in persons ≥18 years old. It is the third COVID-19 vaccine to become available in the US and the first to require only a single dose. Two-dose mRNA-based vaccines manufactured by Pfizer-BioNTech and Moderna received EUAs in December 2020.

The FDA has approved the complement C5 inhibitor avacincaptad pegol (Izervay – Iveric) for intravitreal treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Avacincaptad pegol is the second complement inhibitor to be approved in the US for this indication; pegcetacoplan (Syfovre), a complement C3 and C3b inhibitor, was approved earlier.

The FDA has approved a sublingual fi lm formulation of the nonergot dopamine agonist apomorphine (Kynmobi – Sunovion) for acute, intermittent treatment of "off" episodes in patients with Parkinson's disease (PD). A subcutaneous formulation of apomorphine (Apokyn) has been available for years for the same indication in patients with advanced PD.

Myfembree (Myovant/Pfizer), an oral fixed-dose combination of the gonadotropin-releasing hormone (GnRH) receptor antagonist relugolix, the estrogen estradiol, and the progestin norethindrone acetate, has been approved by the FDA for management of heavy menstrual bleeding associated with uterine leiomyomas (fibroids) in premenopausal women. It is the second product to be approved in the US for this indication; Oriahnn, which contains the GnRH receptor antagonist elagolix in combination with estradiol and norethindrone acetate, was approved earlier. Relugolix was approved for...