FDA Approved 49 New Therapies Last Year

The FDA’s two main drug review centers — the Center for Drug Evaluation (CDER) and Research and the Center for Biologics Evaluation and Research (CBER) — in 2022 approved a total of 49 new therapies. Of these, 37 had been approved by CDER and the remaining, including several gene therapies, were approved through CBER.

The 2022 approvals through Center for Drug Evaluation target many different disease areas, including COVID-19, HIV, smallpox, influenza, and H. pylori infection. Also approved were therapies for two severe and progressive neurological conditions, amyotrophic lateral sclerosis and spinal muscular atrophy.

Patrizia Cavazzoni, M.D.

“2022 marked the third consecutive year that COVID-19 has taken its toll on the global community,” Patrizia Cavazzoni, M.D., director, Center for Drug Evaluation and Research at the FDA, said in the report. “Mpox also emerged as a public health concern. Despite these hardships, we approved many therapies to prevent, diagnose, and treat a wide range of diseases and conditions.”

More than half of these new approvals (20) were for drugs to treat patients with rare diseases. In oncology, approvals included treatments for lung cancer, prostate cancer, uveal melanoma and types of breast cancer, among other kinds of cancers.

In 2022, CDER approved the 40th biosimilar, as well as two interchangeable biosimilars. There are now four approved interchangeable biosimilars. Additionally, 20 drugs were first in class, including:

• Camzyos (mavacamten) capsules, developed by Bristol Myers Squibb, 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. It was approved in April 2022. Read Formulary Watch’s story here.
• Mounjaro (tirzepatide) injection, developed by Lilly, to improve glycemic control in adults with type 2 diabetes, as an addition to diet and exercise. Mounjaro is a first-in-class medication that activates two hormone receptors, which leads to improved glycemic control. It was approved in May 2022. Read more here.
• Pluvicto (lutetium 177 Lu vipivotide tetraxetan) injection, developed by Lilly, to treat adults with prostate-specific, membrane-positive, metastatic, castration-resistant (cancer that grows despite reduced amounts of testosterone) prostate cancer who have received at least two prior therapies, including a chemotherapy. It was approved in March 2024. Read more here.
• Sunlenca (lenacapavir) tablets and injection from Gilead Sciences’ for adults with HIV who have previously received many HIV therapies and whose disease cannot be treated with other available drugs due to resistance, intolerance, or safety concerns. After patients complete a starting dose of Sunlenca, they receive injections once every six months. It was approved in December 2022. Read more here.
• Tzield (teplizumab-mzwv) injection, developed by Provention Bio, to delay the onset of Stage 3 type 1 diabetes in adults and pediatric patients aged 8 years and older with Stage 2 type 1 diabetes. Tzield, cleared by the FDA in November 2022, is the first drug approved for this indication. Read more here.

Of the drugs that were approved last year, 12 had been granted fast track designation, 13 had received breakthrough designation, and 21 had received priority review. Six of the approvals were accelerated approvals, which is a pathway that allows therapies for serious conditions to be approved based on a surrogate endpoint. These approvals include:

• Elahere (mirvetuximab soravtansine-gynx), developed by ImmunoGen, is a first-in-class antibody-drug conjugate targeted against folate receptor alpha, a protein on the surface of ovarian cancer cells. It was granted accelerated approval in November 2022 treat adult patients with epithelial ovarian, fallopian tube, or primary peritoneal cancer. Read more here.
• Krazati (adagrasib), developed by Mirati Therapeutics, was granted accelerated approval in December 2022 to treat patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC). Read more here.
• Lunsumio (mosunetuzumab-axgb), developed by Genentech, is a T-cell engaging bispecific antibody and targets CD20 on the surface of B cells and CD3 on the surface of T cells treat adult patients with relapsed or refractory (R/R) follicular lymphoma. It was granted accelerated approval in December 2022. Read more here.
• Lytgobi (futibatinib), developed by Taiho, was granted accelerated approval in October 2022 to treat adult patients with previously treated, unresectable, locally advanced or metastatic advanced bile duct cancer. It is approved to treat patients with the intrahepatic form of the disease who have FGFR2 gene fusions or other rearrangements. Read more here.
• Tecvayli (teclistamab-cqyv), developed by Johnson & Johnson, to treat adult patients with relapsed or refractory multiple myeloma. Tecvayli is a first-in-class, bispecific T-cell engager antibody to treat patients who have previously received four or more prior lines of therapy. It is an off-the-shelf therapy that targets the CD3 receptor expressed on the surface of T-cells and the B-cell maturation antigen (BCMA) expressed on the surface of multiple myeloma cells. It was granted accelerated approval in October 2022. Read more here.
• Vonjo (pacritinib), developed by CTI BioPharma, treat patients with myelofibrosis, a bone marrow cancer. Vonjo, which was grant accelerated approval in March 2022, is novel oral kinase inhibitor with specificity for JAK2 and IRAK1. Read more here.

The FDA’s Center for Biologics Evaluation and Research approvals in 2022, include vaccines for COVID-19 and a measles, mumps, and rubella, as well as several gene and other novel therapies. These products, however, have some of the highest list prices on new drug approvals. Some of these approvals include:

• Carvykti (ciltacabtagene autoleucel), developed by Legend Biotech and marketed by Janssen Biotech, was approved in March 2022 to treat patients with relapsed or refractory multiple myeloma. Read more here.
• Hemgenix (etranacogene dezaparvovec-drlb), developed by CSL Behring, is the first one-time gene therapy treatment for adults with hemophilia B. It will have a list price of $3.5 million, the highest ever cost for a new drug. It was approved in November 2022. Read more here.
• Rebyota (fecal microbiota, live-jslm), developed by Rebiotix, isthe first ever microbiota product to prevent recurrence of Clostridioides difficile infection (CDI) in people 18 years of age and older, following antibiotic treatment for recurrent CDI. It was approved in December 2022. Read more here.
• Skysona (elivaldogene autotemcel), developed by Bluebird Bio, is the first FDA approved therapy to slow progression of neurologic dysfunction in boys with early, active cerebral adrenoleukodystrophy (CALD). It was granted accelerated approval in September 2022 and has a list price of $3 million. Read more here.
• Zynteglo (betibeglogene autotemcel), also developed by Bluebird Bio, is the first cell-based gene therapy to treat adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions. It has wholesale acquisition cost of $2.8 million and was approved in August 2022. Read more here.