Carvykti (ciltacabtagene autoleucel) for Relapsed/Refractory Multiple Myeloma

Carvykti is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T-cell therapy indicated for the treatment of adults with relapsed or refractory multiple myeloma after 4 or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. The treatment is provided as a single dose for infusion containing a suspension of CAR-positive viable T cells in 1 infusion bag. Approval was based on data from the open-label, single-arm, phase 1b/2 CARTITUDE-1 study in which 98% of patients responded to the one-time therapy.

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Enjaymo (sutimlimab-jome) for Cold Agglutinin Disease

Enjaymo, an IgG4 monoclonal antibody, is the first treatment approved to decrease the need for red blood cell transfusion due to hemolysis in adults with cold agglutinin disease (CAD), a rare autoimmune hemolytic anemia. The approval was based on data from the phase 3 CARDINAL study; 54% (n=13/24) of patients achieved the composite primary endpoint (defined by an increase in hemoglobin (Hgb) of at least 2g/dL from baseline or reaching a Hgb level of at least 12g/dL at the 26-week treatment assessment timepoint, as well as the absence of blood transfusions from weeks 5 to 26 or any other CAD-related treatments). The treatment is administered via intravenous infusion.

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Nalmefene HCl Injection for Opioid Overdose Treatment

The Food and Drug Administration (FDA) approved Purdue Pharma’s Nalmefene Hydrochloride Injection for the complete or partial reversal of opioid drug effects, including respiratory depression, induced by either natural or synthetic opioids, and in the management of known or suspected opioid overdose. The drug was previously marketed under the brand name Revex (Baxter) but was discontinued in 2008. The product is expected to be available by mid-2022. In a press release, Purdue stated that it will be distributing the drug for no profit.

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Pyrukynd (mitapivat) for Hemolytic Anemia Due to Pyruvate Kinase Deficiency

Pyrukynd is a first-in-class, oral, selective pyruvate kinase (PK) activator indicated to treat hemolytic anemia in adults with PK deficiency, a rare, inherited disease caused by mutations in the PKLR gene. The approval was based on data from two phase 3 studies, ACTIVATE and ACTIVATE-T, which evaluated mitapivat in adults with PK deficiency not regularly transfused and regularly transfused, respectively. Results from ACTIVATE showed that 40% (n=16) of patients treated with mitapivat had a hemoglobin response. Findings from ACTIVATE-T showed that 33% (n=9) of patients treated with mitapivat had a transfusion reduction response. The oral treatment is the first disease-modifying therapy approved for PK deficiency.

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Vonjo (pacritinib) for Myelofibrosis With Severe Thrombocytopenia

Granted accelerated approval by the FDA, Vonjois indicated for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50×109/L. It is the first approved therapy specifically addressing cytopenic myelofibrosis. The oral kinase inhibitor was approved based on data from the phase 3 PERSIST-2 trial. Among 31 patients who received pacritinib and had a platelet count less than 50×109/L, findings showed that 29% achieved a reduction in spleen volume of at least 35% at week 24 vs 3.1% of those treated with best available therapy (included ruxolitinib, watchful waiting, and hydroxyurea).

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  1. Carvykti. Package insert. Janssen Biotech, Inc.; 2022. Accessed March 2, 2022.
  2. Enjaymo. Package insert. Sanofi; 2022. Accessed March 2, 2022.
  3. Nalmefene Hydrochloride Injection. Package insert. Purdue Pharmaceuticals, LP; 2022. Accessed March 2, 2022.
  4. Pyrukynd. Package insert. Agios Pharmaceuticals, Inc.; 2022. Accessed March 2, 2022.
  5. Vonjo. Package insert. CTI BioPharma Corp.; 2022. Accessed March 2, 2022.