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Central Research
FDA Novel Drug Approvals 2022
That is a wrap for 2022! And another year of relentless drug discovery.
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For you hard-core drug discovery, here are the list of 2022 novel FDA approved drugs, 37 NMEs so far, down from 2020 and 2021 (53 & 50 approvals, respectively). Before we start talking about a decline in innovation, it has been a turbulent year economically and the decline can be attributed to the COVID-19 pandemic. Total approvals have been higher over the last decade than previous.
It has been a BIG year for gene therapy. Gene therapy offers the possibility of a permanent cure for a wide range of inherited disorders. Those approvals include:
Skysona, is used to treat cerebral adrenoleukodystrophy, a rare disease in boys that allows very long chain fatty acids to accumulate in the brain which causes severe disability or death. It is made specifically for each patient, using the patient’s own blood stem cells and adds functional copies of the ABCD1 gene to the patient’s cells; bluebird bio.
Zynteglo, is used for the treatment for ß-thalassemia, an inherited blood disorder that leads the body to produce less hemoglobin. To make Zynteglo, the stem cells taken from the patient’s blood are modified by a virus that carries working copies of the beta globin gene into the cells; bluebird bio.
Hemgenix, is used for the treatment of the clotting disorder hemophilia B. It is a genetic bleeding disorder resulting from missing or insufficient levels of blood clotting Factor IX. Hemgenix delivers a working gene for the clotting protein to the liver, where it is made; CSL Behring.
Adstiladrin, is the first gene therapy for the treatment of bladder cancer; Ferring Pharmaceuticals.

New medicines which were approved in Q422
Krazati, a KRASG12C inhibitor for the treatment of NSCLC; Mirati Therapeutics.
Rezlidhia, an isocitrate dehydrogenase-1 inhibitor used to treat relapsed or refractory AML; Rigel Pharmaceuticals Inc.
Tzield, a humanized anti-CD3 mAb that is used to delay the onset of stage 3 type 1 diabetes; Provention Bio.
Elahere, a folate receptor (FR) alpha-directed antibody and microtubule inhibitor conjugate used to treat FR alpha positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer; ImmunoGen, Inc.
Tecvayli, a bispecific B-cell maturation antigendirected CD3 T cell engager used to treat R/R MM; Janssen Inc.
Imjudo, an anti-CTLA-4 antibody used to treat unresectable HCC in combination with durvalumab; AstraZeneca.
Lenacapavir, an HIV-1 capsid inhibitor for the treatment of HIV; Gilead Sciences.
Mosunetuzumab, a humanized anti-CD20/CD3 bispecific antibody used to treat R/R follicular lymphoma; Roche.
Briumvi, a monoclonal anti-CD20 antibody for the treatment of MS; TG Therapeutics, Inc.
17 of these NMEs can be classified as small molecule drugs.

First-in-Class Drugs 2022
On the other hand, CDER identified 20 of the 37 novel drugs approved in 2022 (54%) as first-in-class. These drugs have mechanisms of action different from those of existing therapies.
These novel first-in-class drugs were:
Camzyos (mavacamten), a mysosin inhibitor, used to improve functional capacity and symptoms in patients with a type of obstructive hypertrophic cardiomyopathy, in which the heart muscle thickens, making it harder to pump blood.
Mounjaro (tirzepatida), a dual GIP and GLP-1 receptor agonist injection to improve glycemic control in adults with type 2 diabetes, as an addition to diet and exercise.
Pluvicto (lutetium 177 Lu vipivotide tetraxetan), a radioligand therapeutic injection to treat adults with prostate-specific, membrane-positive, metastatic, castration-resistant prostate cancer who have received at least two prior therapies, including a chemotherapy. Pluvicto consists of a radionuclide, lutetium Lu-177, linked to a moiety that binds to PSMA, a transmembrane protein that is expressed in prostate cancer.
Sunlenca a first-in-class HIV1 capsid inhibitor, given as tablets and injection for adults with HIV who have previously received many HIV therapies and whose disease cannot be treated with other available drugs. Patients receive Sunlenca in combination with other antiretroviral(s).
Tzield (teplizumab-mzwv), a CD3-directed monoclonal antibody injection to delay onset of Stage 3 type 1 diabetes in adults and pediatric patients with Stage 2 type 1 diabetes.
Voquezna Triple Pak (vonoprazan, amoxicillin, and clarithromycin) and Voquezna Dual Pak (vonoprazan and amoxicillin) are co-packaged products containing combinations of tablets and capsules to treat adults with H. pylori infection, a bacterial infection in the stomach.
Pyrukynd (mitapivat), a pyruvate kinase activator used to treat hemolytic anemia (a disorder in which red blood cells are destroyed faster than they can be made) in adults with pyruvate kinase deficiency, an inherited disorder that causes premature red blood cell destruction.
Kimmtrak (tebentafusp-tebn), a bispecific gp100 peptide-HLA-directed CD3 T cell engager injection, used to treat metastatic or unresectable uveal melanoma, a rare cancer that develops in a part of the eye called the uvea.
Opdualag, an injection to treat patients with metastatic or unresectable melanoma. It contains nivolumab, a programmed death receptor-1 (PD-1) blocking antibody, and relatlimab, a lymphocyte activation gene-3 (LAG-3) blocking antibody.
20 of the drugs approved were to treat rare or “orphan” diseases.

Chris De Savi
CSO Partner at Curie.Bio