In February 2021, the world's first batch of 7 new drugs, the fourth CAR-T therapy Breyanzi was approved by the FDA

Author: Sea

According to the global drug database of Yaodu, in February 2021, there were 7 new drugs approved for marketing for the first time in the world, all of which were approved by the FDA.

The world’s fourth CAR-T therapy Lisocabtagene maraleucel (brand name: Breyanzi) new lymphoma drug Umbralisib (brand name: Ukoniq) chemotherapy bone marrow protection drug Trilaciclib (brand name: COSELA) lipid-lowering new drug Evinacumab (brand name: EVKEEZA) Duchenne progressive New drug for muscular dystrophy Casimersen (brand name: Ammondys 45) A new drug for molybdenum cofactor deficiency type Fosdenopterin (brand name: Nulibry) New therapy for myeloma (peptide-conjugated drug) Melflufen (brand name: Pepaxto)

01Lisocabtagene maraleucel

Lisocabtagene maraleucel (Breyanzi) is a CD19-targeted inhibitory chimeric antigen receptor (iCAR) T cell therapy developed by BMS and approved for use in adult patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) treatment.

Breyanzi is the world’s fourth approved CAR-T therapy.

02Umbralisib

Umbralisib was developed by TG Therapeutics and is approved for the treatment of patients with relapsed/refractory marginal zone lymphoma (MZL) who have previously received at least one anti-CD20-based therapy, as well as relapsed/refractory patients who have received at least third-line systemic therapy in the past Adult patients with follicular lymphoma (FL).

Umbralisib is the first approved oral PI3Kδ/CK1ε inhibitor.

03Trilaciclib

Trilaciclib is a short-acting and reversible CDK4/6 inhibitor developed by G1 Therapeutics.

Trilaciclib is the world's first and only product that is administered prophylactically during chemotherapy to protect the bone marrow and immune system.

In August 2020, Simcere Pharmaceuticals reached an exclusive license agreement with G1, and obtained the development and commercialization rights of Trilaciclib for all indications in the Greater China region.

04Evinacumab

Evinacumab was developed by Regeneron and is approved for the treatment of children 12 years and older or adults with familial homozygous hypercholesterolemia (HoFH).

Evinacumab is a fully human IgG4κ monoclonal antibody targeting angiopoietin-like protein 3 (ANGPTL3).

05Casimersen

Casimersen is an antisense oligonucleotide therapy targeting Dystroglycan.

Casimersen was approved to use the accelerated approval channel this time, and the drug has obtained the fast-track qualification granted by the FDA, the priority review qualification and the orphan drug qualification.

06Fosdenopterin

Fosdenopterin was developed by Origin Biosciences and was approved to reduce the risk of death due to type A molybdenum cofactor deficiency (MoCD).

Type A MoCD is an autosomal recessive inherited disease caused by the interruption of molybdenum cofactor synthesis, which is extremely rare.

Fosdenopterin is a substrate replacement therapy that can replace cPMP and allow the molybdenum cofactor synthesis step to continue.

07Melflufen

Melflufen (also known as Melphalan flufenamide) was developed by Oncopeptides.

This approval is based on the results of the pivotal Phase 2 clinical trial HORIZON.
In this clinical trial, 157 patients with relapsed/refractory MM received Pepaxto and dexamethasone combination therapy.
The test results showed that the combination therapy reached an overall remission rate of 23.
7%, and the median duration of remission was 4.
2 months.

Melflufen is a "first-in-class" peptide-conjugated drug.
It uses Oncopeptides' proprietary peptide-drug conjugate (PDC) platform to couple the alkylating agent felphalan with peptides targeting aminopeptidase.
together.
Melflufen can be quickly taken up by MM cells due to its lipophilicity, and then is rapidly hydrolyzed by peptidase intracellularly, thereby releasing a hydrophilic alkylating agent.

Aminopeptidase is overexpressed in tumor cells, especially in advanced cancers or tumors with high mutation complexes.
In in vitro experiments, Melflufen can increase the concentration of alkylating agent in cells, and its ability to kill MM cells is 50 times higher than that of the alkylating agent it carries.