The "first" new drug approved globally in 2021

According to incomplete statistics, in 2021, a number of drugs that can be labeled "first" were approved globally, including new target drugs and new therapies
.

In 2021, at least 7 new target drugs will be approved globally
.

See the table below for details .

Vyvgart (efgartigimod) is the world's first neonatal Fc receptor (FcRn) antagonist approved by the FDA in December for the treatment of adult patients with systemic myasthenia gravis (gMG) with positive anti-acetylcholine receptor antibodies (AChR Ab+)
.

Tezspire (tezepelumab) is the first approved anti-thymic interstitial lymphopoietin (TSLP) monoclonal antibody.
It was approved by the FDA in December as an additional maintenance therapy for the treatment of pediatric patients and adults with severe asthma ≥12 years of age Patient
.

Tavneos is the first approved orally administered selective complement C5a receptor (C5aR) inhibitor.
It was approved in Japan in September in combination with standard therapies to assist in the treatment of two major anti-neutrophil cytoplasmic autoantibodies ( ANCA) related vasculitis: Microscopic polyangiitis (MPA) and granuloma with polyangiitis (GPA)
.

Lumakras/Lumykras (sotorasib) is the first approved targeted therapy for KRAS G12C mutations.
It was approved in the United States in May for the treatment of at least one systemic therapy that has been previously received.
The FDA-approved test method confirms the presence of KRAS G12C.
Adult patients with mutant, locally advanced or metastatic NSCLC
.

Scemblix's active pharmaceutical ingredient asciminib is a STAMP inhibitor that specifically targets the myristoyl pocket (STAMP) of BCR-ABL1 protein and locks BCR-ABL1 into an inactive conformation
.

Competitive drugs currently on the market bind to the ATP binding site of the BCR-ABL1 protein

Saphnelo is the first type I interferon receptor (IFNAR) antibody.
It was approved by the FDA in August for the treatment of moderate to severe adult patients with systemic lupus erythematosus (SLE) who are receiving standard treatment.
It is also the only FDA approval in the past 10 years.
New treatment for SLE
.

Evkeeza is the first drug approved by the FDA to target and block the function of angiopoietin-like protein 3 (ANGPTL3).
It was approved by the FDA in February as an adjuvant therapy for other lipid-lowering therapies for the treatment of homozygous ≥12 years of age.
Familial hypercholesterolemia (HoFH) children and adults
.

Moreover, CAR-T therapy and ADC drugs have also ushered in new targeted drugs
.

Abecma is the world’s first B-cell maturation antigen (BCMA) targeted CAR-T therapy.
It was approved by the FDA in March for the treatment of adult relapsed/refractory multiple myeloma (R/ R MM)
.

Zynlonta is the first approved CD19-targeted antibody conjugate (ADC).
It was approved by the FDA in April for the treatment of relapsed or refractory (r/r) big B that has received 2 or more systemic therapies.
Adult patients with cell lymphoma (LBCL), including diffuse large B cell lymphoma (DLBCL), DLBCL originating from low-grade lymphoma and high-grade cell lymphoma
.

Tivdak is the world's first tissue factor (TF) targeted antibody drug conjugate (ADC).
It was approved by the FDA in September for the treatment of adult patients with recurrent or metastatic cervical cancer who progressed during or after chemotherapy
.

In addition, drugs with new mechanisms of action have been approved for some diseases in 2021
.

Empaveli is the first C3 targeted therapy to treat PNH with regulatory approval.
It was approved by the FDA in May for PNH adult patients who have not previously been treated, and PNH adult patients who switched from C5 inhibitors (Soliris and Ultomiris).

.

Zeposia is the first oral sphingosine-1-phosphate (S1P) receptor modulator approved for the treatment of ulcerative colitis (UC), and was approved by the FDA in May for the treatment of moderate to severe active colitis (UC) in adults
.

Previously, the drug was approved by the FDA in March 2020 for the treatment of adult relapsing multiple sclerosis (RMS), including clinically isolated syndrome, relapsing-remitting disease and active secondary progressive disease

Farxiga/Forxiga was approved by the FDA in April for the treatment of adult patients with chronic kidney disease (CKD), regardless of whether they have type 2 diabetes (T2D), and is the first approved treatment of chronic kidney disease (CKD) patients (whether or not There are T2D) SGLT2 inhibitors
.

Cosela is a pioneering short-acting CDK4/6 inhibitor.
It was approved by the FDA in February for use in adult patients with extensive-stage non-small cell lung cancer (ES-SCLC).
Kang’s chemotherapy regimen was previously administered to reduce the incidence of chemotherapy-induced bone marrow suppression, becoming the world's first bone marrow protective therapy that can reduce the incidence of chemotherapy-induced bone marrow suppression
.

Verquvo is the first soluble guanylate cyclase (sGC) stimulator approved for the treatment of heart failure.
It was approved by the FDA in January for symptomatic chronic heart failure patients with an ejection fraction <45%, reducing the incidence Exacerbating heart failure events (defined as: heart failure hospitalization or outpatient intravenous [IV] diuretics for heart failure without hospitalization) risk of cardiovascular death and heart failure hospitalization
.

Moreover, in 2021, a variety of diseases around the world ushered in new therapies
.

Rybrevant (Amivantamab-vmjw), an EGFR-MET bispecific antibody with immune cell targeting activity, was approved by the FDA in May for the treatment of disease progression after failure of platinum-containing chemotherapy and positive EGFR gene exon 20 insertion mutation ( Adult patients with metastatic non-small cell lung cancer (mNSCLC) with EGFRex20ins+ have become the world's first targeted therapy approved for the treatment of EGFRex20ins+ NSCLC
.

Voxzogo is a C-type natriuretic peptide (CNP) analog that is injected once a day.
It was approved by the FDA in November for the treatment of pediatric patients with achondroplasia who are 5 years old and older with open growth plates.
It increases linear growth.
The world's first drug approved to treat children with achondroplasia can treat the underlying cause of the disease
.

Keytruda was approved by the FDA in November for the adjuvant treatment of renal cell carcinoma (RCC) patients who had a medium-to-high or high risk of recurrence after nephrectomy (surgical removal of the kidney) or nephrectomy and resection of metastatic lesions.
It was the first Approved for the adjuvant treatment of immunotherapy for the above-mentioned RCC patients
.

Besremi is an innovative mono-PEGylated long-acting interferon.
It was approved by the FDA in November for the treatment of adult patients with polycythemia vera (PV), becoming the first drug approved by the FDA to treat PV (regardless of whether the patient has a history of treatment) Can be used), and is also the first interferon therapy specifically approved for the treatment of PV
.

Libtayo is the first immunotherapy approved for the treatment of advanced basal cell carcinoma (BCC).
It was approved by the FDA in February as a monotherapy for the treatment of a hedgehog pathway inhibitor (HHI) treatment but the disease progressed or the class Adult patients with locally advanced or metastatic BCC who are drug intolerant
.

The active ingredient of Ryplazim is plasminogen, which is extracted and purified from human plasma
.

In June, the drug was approved by the FDA to treat patients with type 1 plasminogen deficiency (hypoplasminogenemia), becoming the first drug approved by the FDA for the treatment of plasminogen deficiency

The active pharmaceutical ingredient maralixibat of Livmarli oral liquid is an orally administered ileal bile acid transporter (IBAT) inhibitor with minimal absorption.
It was approved by the FDA in October for the treatment of patients with Alagille syndrome (ALGS) aged ≥ 1 year.
Cholestatic pruritus is the first drug approved for the treatment of ALGS cholestatic pruritus
.

Avnibuchen is the world's first drug approved by the FDA for the targeted therapy of patients with IDH1 mutant cholangiocarcinoma.
It was approved by the FDA in August for previously treated patients with IDH1 mutations detected by the FDA approved detection method.
Adult patients with locally advanced or metastatic cholangiocarcinoma
.

Korsuva active drug difelikefalin is a pioneering peripheral κ opioid receptor (KOR) agonist.
It was approved by the FDA in August for patients undergoing hemodialysis to treat moderate to severe pruritus (CKD-aP) associated with chronic kidney disease.
The first drug approved by the FDA to treat chronic kidney disease-related itching in adult patients on hemodialysis has the potential to fundamentally change the treatment model of CKD-aP in adult patients undergoing dialysis
.

Opdivo is the first PD-1 inhibitor approved for the treatment of urothelial carcinoma (UC) in adjuvant therapy.
It was approved by the FDA in August for the adjuvant treatment of patients with muscle invasive urothelial carcinoma (MIUC).
For: MIUC patients who have a high risk of recurrence after radical resection, regardless of whether they have previously undergone neoadjuvant chemotherapy, lymph node involvement, or PD-L1 status
.

Darzalex Faspro was accelerated by the FDA in January for the combination of bortezomib, cyclophosphamide, and dexamethasone (D-VCd regimen) for the treatment of newly diagnosed adult patients with light chain (AL) amyloidosis, becoming the first Drugs for the treatment of AL amyloidosis
.

Carbaglu, as a substitute for N-acetylglutamate (NAG), improves or restores urea cycle function by activating carbamoyl phosphate synthase (CPS 1), and promotes ammonia detoxification and urea production
.

In January, the drug was approved by the FDA as an adjunct to standard care therapy for pediatric and adult patients to treat acute hyperammonemia caused by propionic acidemia (PA) or methylmalonic acidemia (MMA) , Becoming the first drug approved by the FDA to treat acute hyperammonemia caused by PA and MMA

Bylvay is a potent, non-systemic ileal bile acid transport inhibitor, approved by the European Union in July, becoming the world's first drug approved for the treatment of all subtypes of progressive familial intrahepatic cholestasis (PFIC)
.

Xywav is a product developed specifically to provide low-sodium oxybutyric acid therapy for narcolepsy patients.
It has a unique cationic component (calcium magnesium potassium potassium sodium), which can greatly reduce the chronic sodium load of patients after taking the drug.
The FDA approved the treatment of idiopathic narcolepsy in adults and is the first new drug approved by the FDA for the treatment of idiopathic narcolepsy
.

Tyvaso active drug Treprostinil is a synthetic prostacyclin mimic, which mainly acts by directly dilating the pulmonary and systemic arterial beds and inhibiting platelet aggregation
.

In June, Tyvaso inhalation solution was approved by the FDA for the treatment of patients with interstitial lung disease-related pulmonary hypertension (PH-ILD; WHO Group 3) to improve exercise capacity and become the first approved treatment for PH-ILD in the United States

Arcalyst is a weekly, subcutaneously injected recombinant fusion protein that can block interleukin-1α (IL-1α) and interleukin-1β (IL-1β) signals.
It was approved by the FDA for treatment in March 12 It is the first drug approved by the FDA for the treatment of recurrent pericarditis in patients with recurrent pericarditis and to reduce the risk of recurrence of pericarditis
.

Yescarta is a CD19-targeted CAR-T therapy.
It was approved by the FDA in March for the treatment of relapsed or refractory (R/R) follicular lymphoma (FL) that had previously received two or more systemic therapies.
) Adult patients, becoming the first CAR-T therapy approved for the treatment of indolent FL
.

Moreover, some diseases have also ushered in new ways of administration
.

Exkivity is an oral tyrosine kinase inhibitor (TKI) specifically designed for EGFRexon20 insertion mutations.
It was approved by the FDA in September for the treatment of disease progression during or after previous platinum-containing chemotherapy.
The FDA-approved test method proved to be Adult patients with locally advanced or metastatic non-small cell lung cancer carrying EGFRex20ins mutations are the first oral targeted therapy approved for the treatment of EGFRexon20 NSCLC
.

Tyrvaya nasal spray is a highly selective cholinergic agonist.
It was approved by the FDA in October to treat the symptoms and signs of dry eye (DED), becoming the first nasal spray approved for the treatment of dry eye.
Agent
.

Qulipta is the first and only oral calcitonin gene-related peptide (CGRP) receptor antagonist specifically developed for the preventive treatment of migraine
.

It was approved by the FDA in October for the preventive treatment of episodic migraine in adults .

Opzelura is the first topical Janus kinase inhibitor approved by the FDA.

Lupkynis (voclosporin) is a new and potentially best-in-class calcineurin inhibitor (CNI).