FDA Novel Drug Approvals: February 2022

This article reviews the novel drugs approved by the FDA in February 2022.

together with Dr. Naveed Yasin, Dr. Vinicius Texeira, and Dr. Jennifer Huen

Three novel molecules were approved in February 2022:

Small molecules:

• Pyrukynd (mitapivat): An oral pyruvate kinase activator approved for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency.
• Vonjo (pacritinib): An oral (200 mg BID) JAK2/IRAK1 kinase inhibitor with selectivity over JAK1 for treatment of myelofibrosis

Large molecule:

• Enjaymo (sutimlimab): An injectable classical complement pathway inhibitor (IgG mAb targeting C1s) approved for decreasing red blood cell (RBC) transfusions in adults with cold agglutinin disease (CAD).

We recently covered mitapivat, the allosteric pyruvate kinase activator.

Pacritinib has had a long clinical journey, which included a full clinical hold in 2016. More discussion of the strategy used to clinically differentiate it over previous JAK inhibitors appears below.

Sutimlimab was acquired by Sanofi in its $11.6 billion buyout of Bioverativ in 2018. Bioverativ acquired sutimlimab in a $400M deal with True North Therapeutics in 2017. More details about the first-in-class sutimlimab also appears in the premium section below.

Enjaymo (sutimlimab)

Cold agglutinin disease (CAD) is a difficult-to-treat autoimmune disorder in which red blood cells (RBCs) are destroyed prematurely by cold agglutinin autoantibodies (usually IgMs), leading to hemolysis via the classical complement pathway. Cold agglutinins are usually not problematic since the optimal temperatures at which they react with RBC antigens are outside the range of normal human body temperature (28-30°C). However in CAD, active cold agglutinins are pathogenic, causing agglutination (formation of particle-sized clumps) of RBCs, and hemolytic anemia by activating the classical complement pathway via binding the C1 complex.

See our overview of all 36 of the novel small molecule and large molecule FDA approvals for 2022 here.

You can find the novel drug approvals of January 2022, the small molecule drug approvals of 2021, the large molecule drug approvals of 2021, the first-in-class small molecule drugs of 2021, and more on drughunter.com.