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In 2022, FDA's Center for Drug Evaluation and Research (CDER) approved a total of 37 new drugs, including 22 new molecular entities and 15 biologics
.
In addition, the Center for Biologics Evaluation and Research (CBER) approved 2 vaccines, 1 cell therapy and 4 gene therapies, as well as 1 microbiome therapy
.
Compared with previous years, the number of new drugs approved by the FDA this year has decreased, but the innovation content is extremely high, and the types of approved biological products are rich, including monoclonal antibodies, dual antibodies, ADCs, TCR therapies, enzyme replacement therapies, etc
.
The number of new drugs approved by the FDA in recent years
In terms of disease areas, neocology drugs accounted for the highest proportion (27%) among new drugs (excluding vaccines, gene therapy and cell therapy) approved by the FDA in 2022, followed by neurological and skin drugs
.
From the perspective of review methods, 13 of these new drugs were marketed through priority review, and 5 were marketed through the accelerated approval process, of which 18 were granted orphan drug status by the FDA, accounting for 49%, and 13 were recognized
as breakthrough therapies.
Distribution of CDER-approved drug disease areas (left) and number of qualifications granted (right)
In terms of innovation, 21 of the 37 new drugs approved by the FDA are "first-in-class" therapies, accounting for 57%, of which 10 have also been recognized
by the FDA as breakthrough therapies.
10 drugs approved by the FDA as breakthrough therapies
From the company's point of view, BMS is the biggest winner of new drug approval in 2022, including 3 new drugs, including the melanoma drug Opdualag (nivolumab+).
relatlimab), hypertrophic cardiomyopathy drug Camzyos (mavacamten) and psoriasis drug Sotyktu (deuterium colexitinib).
Roche and Sanofi also each received 2 new drugs
.
In 2022, the FDA approved an unprecedented number of gene therapies, up to 4, mostly for rare disease treatment
.
Due to factors such as the narrow population of rare diseases and the high superimposed cost, Bluebird Bio's Zynteglo is priced at $2.
8 million in the U.
S.
and Skysona at $3 million, CSL
Behring's Hemgenix is priced at $3.
5 million, making it the most expensive drug
in the world.
In addition to gene therapy, the FDA has also approved 1 cell therapy, 1 microbiome therapy and 2 vaccines, of which the cell therapy is from the Chinese local company Legend Biotechnology, which is also the first domestic CAR-T therapy
approved by the FDA.
8 new therapies approved by CBER
Due to space limitations, this article selects 10 blockbuster new drugs for introduction
.
The world's first TCR therapy: Kimmtrak
The world's first TCR therapy: KimmtrakIndications: HLA-A*02:01 Positive adult metastatic uveal melanoma On January 25, the FDA approved the innovative bispecific fusion protein Kimmtrak (tebentafusp-tebn) for marketing
.
This is the first FDA-approved therapy for the treatment of unresectable or metastatic uveal melanoma, the first approved T cell receptor (TCR) therapy, and the first bispecific T cell adaptor protein
for the treatment of solid tumors.
Tebentafusp is the first biologic developed based on the Immunocore core technology platform ImmTACs, which is an engineered high-affinity T cell receptor (TCR) binding domain at one end that can target gp100 on the surface of tumor cells; At the other end is a single-chain antibody fragment (scFv) that targets CD3 on the surface of
T cells.
The TCR domain first recognizes and binds to the peptide-HLA
on the surface of tumor cells.
The anti-CD3 antibody fragment then recruits and redirects the T cells around the tumor cells
.
In this way, ImmTACs build a bridge between cancer cells and T cells, forming immune synapses.
Activates T cells and releases lytic granules, leading to cancer cell death
.
Tebentafusp kills cancer cell processes
The first domestic CAR-T cell therapy: Carvykti
The first domestic CAR-T cell therapy: CarvyktiIndications: Relapsed/refractory multiple myeloma February 28, BCMA jointly developed by Legend Biologics and Johnson & Johnson
The CAR-T therapy Carvykti (cilta-cel) was approved by the FDA
.
This is the first domestic CAR-T therapy
approved by the FDA.
This approval is also a new milestone and breakthrough
in the internationalization of innovative drugs in China after BeiGene's zebratinib.
Independently developed by GenScript subsidiary Legend Biologics, Cilta-cel is a structurally differentiated CAR-T cell therapy with a 4-1BB co-stimulatory domain and two BCMA-targeted single-domain antibodies
designed to improve affinity.
The drug has successively obtained breakthrough therapy designation and priority review qualification from the FDA, priority drug qualification from EMA, and is also the first drug
in China to obtain breakthrough therapy qualification.
At the end of December 2022, the product also submitted a marketing application
to CDE.
The first new diabetes drug type in nearly 10 years: Mounjaro
The first new diabetes drug type in nearly 10 years: MounjaroIndications: Supplemental diet and exercise to improve glycemic control in adult patients with type 2 diabetes On May 13, the FDA approved the marketing of Mounjaro (tirzepatide
), a dual agonist of Eli Lilly's glucose-dependent insulin stimulating peptide (GIP) and glucagon-like peptide-1 (GLP-1) receptor.
Lilly said this is the first new diabetes drug type
in nearly 10 years.
Tirzepatide is a weekly injection of GIP and GLP-1 receptor agonists that integrate the effects of two incretin insulins into
a new molecule.
GIP is a hormone that complements the effects of
GLP-1 receptor agonists.
Preclinical models have shown that GIP reduces food intake and increases energy expenditure, leading to weight loss
.
And GIP, when used in combination with GLP-1 receptor agonists, may have a greater impact
on blood sugar and body weight.
The first new topical drug for psoriasis in 25 years: Vtama
The first new topical drug for psoriasis in 25 years: VtamaIndications: Local treatment of adult plaque psoriasis On May 23, the FDA approved Vtama (benvimod, tapinarof, 1%) cream for the treatment of adult plaque psoriasis
.
This is the first and only steroid-free topical drug of its kind approved by the FDA and the first topical new molecular solid drug
approved in the United States in 25 years for the treatment of psoriasis.
Benvimod is a novel, once-daily therapeutic aromatic hydrocarbon receptor modulator (TAMA) and is a non-steroidal topical cream
.
It is developed by the local enterprise Tianji Pharmaceutical, and is a national innovative drug
with independent intellectual property rights in China.
In 2012, GSK acquired Benvimod offshore development rights for a down payment of nearly US$200 million, and in July 2018, GSK sold Benvimod offshore development rights to Dermavant for US$330 million
.
The world's first TYK2 inhibitor: Sotyktu
The world's first TYK2 inhibitor: SotyktuIndications: Plaque psoriasis On September 9, the marketing application of deuterium colexitinib (deucravacitinib), a TYK2 inhibitor of BMS, for the treatment of plaque psoriasis was approved by the FDA
.
This is the world's first approved TYK2 inhibitor and has been declared for marketing
in China.
Deuterium colesicitinib is an orally highly selective tyrosine kinase 2 (TYK2) allosteric inhibitor
with a unique mechanism of action.
It inhibits signaling
of key cytokines (such as IL-23, IL-12, and type I interferons) involved in multiple immune-mediated disease pathogenesis by selectively targeting TYK2.
Deuterium colesicitinib achieves high selectivity by binding to the regulatory domain of TYK2, contributing to the allosteric inhibition
of TYK2 and its downstream functions.
Deuterium colesicitinib selectively inhibits TYK2 in the range of physiological concentrations and does not inhibit JAK1, JAK2, or JAK3
at therapeutic doses.
Deucravacitinib mechanism of action
A new treatment for a century-old disease: Relyvrio
A new treatment for a century-old disease: RelyvrioIndications: Amyotrophic lateral sclerosis (ALS) On September 29, Amylyx Relyvrio (AMX0035) was approved by the FDA for the treatment of adult patients with amyotrophic lateral sclerosis (ALS), trade name Relyvrio
.
This is the first treatment to significantly delay ALS disease progression and prolong survival in a randomized, placebo-controlled clinical trial, and the third FDA-approved ALS treatment in 116 years
.
AMX0035 is a proprietary oral fixed-dose combination consisting of sodium phenylbutyrate and diol taurine
.
The combination of the two targets endoplasmic reticulum and mitochondria-dependent neuronal degenerative pathways in amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, reducing neuronal death and dysfunction
.
AMX0035 composition and mechanism of action
The world's first BCMA/CD3 dual antibody: Tecvayli
The world's first BCMA/CD3 dual antibody: TecvayliIndications: Relapsed or refractory multiple myeloma On October 25, Johnson & Johnson's BCMA/CD3 bispecific antibody Tecvayli (teclistamab) was approved by the FDA for the treatment of relapsed or refractory multiple myeloma (r/r
MM）
。 This is the world's first commercially available BCMA/CD3 dual antibody
.
BCMA is a member of the tumor necrosis factor receptor (TNF) superfamily and is highly expressed in cancer cells of MM patients, and CD3 is involved in activating the immune system to fight infection
.
Teclistamab is a Genmab that uses Genmab
IgG4 dual antibody developed by DuoBody® technology that simultaneously targets T cell CD3 and MM cell BCMA, which redirects CD3+ T cells to BCMA-expressing myeloma cells to induce cytotoxicity
against target cells.
The world's most expensive drug: Hemgenix
The world's most expensive drug: HemgenixIndications: Hemophilia B November 22, CSL
Behring and unique's hemophilia B gene therapy Hemgenix (etranacogene
dezaparvovec) was approved by the FDA, becoming the first gene therapy
approved to treat this rare disease.
The drug is reported to cost as much as $3.
5 million per dose, more than Bluebird
Bio's gene therapy Skysona, which sold for $3 million, is the world's most expensive drug
.
Hemgenix uses AAV5 vector delivery expressing coagulation factor IX
Transgenic of the Padua variant, which is 5-8 times more active than ordinary coagulation factor IX, can complete normal coagulation function
at lower expression levels.
The therapy delivers genes to the liver, and the newly synthesized clotting factor IX promotes clotting and reduces bleeding events
.
The first fecal microbiome therapy: Rebyota
The first fecal microbiome therapy: RebyotaIndications: On December 1, Ferring's microbiome therapy, Rebyota (RBX2660), was approved by the FDA for the prevention of recurrent Clostridium difficile infection
in patients 18 years of age and older.
This is the first FDA-approved fecal microbiome therapy
.
Rebyota is a microbiome therapy for the prevention of Clostridium difficile infection that does not contain any antibiotics and is designed to help patients restore their gut microbiome and avoid reinfection
with CDI.
Rebyota is administered as a single dose rectally and is made from the feces of an eligible donor, and before the donated feces are used, the donor and the feces undergo a series of tests to confirm that they contain pathogenic bacteria
that can cause infection.
First CD20/CD3 dual antibody: Lunsumio
First CD20/CD3 dual antibody: LunsumioIndications: On December 22, the marketing application of Roche's Genentech Lunsumio was approved
by the FDA.
This is the first FDA-approved CD20/CD3 dual antibody for the treatment of non-Hodgkin lymphoma and represents a chemotherapy-free, off-the-shelf new tumor immunotherapy option that allows patients to receive intravenous infusion therapy in an outpatient setting without
waiting 。 Lunsumio is a first-in-class bispecific antibody targeting CD20/CD3, designed to target CD20 on the surface of B cells and CD3 on the surface of T cells, activate and redirect the patient's existing endogenous T cells by dual targeting, and release cytotoxic proteins into B cells to eliminate malignant B cells
.
Attached: New drugs approved by the FDA in 2022
