FDA's first batch of drugs will be ushered in in the fourth quarter of 2021

According to incomplete statistics, at least 16 drugs will be approved by the FDA in the fourth quarter of this year (see the table below for details), of which 9 are small molecule chemical drugs, and their indications involve tumors, autoimmune diseases, and rare diseases.

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Fyarro (ABI-009) is a suspension of sirolimus albumin-bound nanoparticles for injection.
Sirolimus is an mTOR inhibitor
.
Compared with the marketed mTOR inhibitors, Fyarro has superior PK characteristics, wider therapeutic window, higher tumor tissue drug exposure, stronger target cell inhibitory effect and better safety
.
In July 2021, the FDA accepted Fyarro's NDA for the treatment of advanced malignant perivascular epithelioid cell tumors and granted priority review to the application.
The PDUFA date is November 26, 2021
.
In addition, in January this year, Eton King Ang Pharmaceutical Co.
, Ltd.
reached an exclusive license cooperation with Aadi, and obtained Fyarro's development and commercialization rights in Greater China (Mainland China, Hong Kong, Macau and Taiwan)
.

9.
Cilta-cel

Cilta-cel is a CAR-T cell therapy with a differential structure.
It contains a 4-1BB costimulatory domain and two antibody domains targeting BCMA.
It has the ability to promote the expansion of CD8-positive T cells
.
In December 2017, Janssen and Legend Bio reached an exclusive global license and cooperation agreement to jointly develop and commercialize cilta-cel
.

In May 2021, Janssen announced that the FDA has accepted Cilta-cel for the treatment of multiple myeloma (MM) BLA and granted priority review to the BLA
.
The key phase 1b/2 study CARTITUDE-1 of Cilta-cel in the treatment of MM showed that the median follow-up time was 12.
4 months, and the ORR of Cilta-cel reached 97%, and the patient’s remission degree further deepened over time.
67% of patients achieved a strict complete remission
.

10.
Palovarotene

Palovarotene is a selective RARγ inhibitor obtained by Ipsen (Ipsen) in its US$1.
3 billion acquisition of Clementia Pharmaceuticals.
It has been developed for the treatment of progressive muscular ossification (FOP), a variety of osteochondroma, dry eye and other diseases , Its global multi-center phase 3 clinical trial MOVE data for the treatment of FOP showed that compared with untreated patients in the natural history study, patients treated with palovarotene reduced the volume of new heterotopic ossification by an average of 62% each year (p= 0.
0292)
.

In June 2021, the FDA accepted Palovarotene's NDA for the treatment of FOP and granted priority review qualifications for the application.
The PDUFA date is November 30, 2021
.
If approved, palovarotene will become the world's first treatment for FOP
.

11.
Pacritinib

Pacritinib is an oral inhibitor that specifically inhibits JAK2, IRAK1 and CSF1R.
Because it does not inhibit JAK1 activity, it avoids the potential side effects of JAK1 inhibition.
It can treat myelofibrosis, acute myeloid leukemia (AML), and myelodysplastic syndromes.
The potential for diseases such as chronic myelogenous leukemia (MDS), chronic myelomonocytic leukemia (CMML) and chronic lymphocytic leukemia (CLL)
.
The results of a phase 2 clinical trial and two phase 3 clinical trials for the treatment of myelofibrosis showed that 29% of patients receiving pacritinib had a reduction in spleen volume by at least 35% (Vs active control group 3%), 23 % Of patients’ total symptom score was reduced by at least 50% (13% in the Vs active control group)
.

In June 2021, the FDA accepted pacritinib for the treatment of NDA for myelofibrosis with severe thrombocytopenia, and granted the application priority review.
It is expected that the FDA will respond before November 30 this year
.

12.
Nefecon

Nefecon is a targeted-release oral formulation of budesonide.
It uses Calliditas' TARGIT technology to allow the drug to pass through the gastrointestinal tract without being absorbed, and to be absorbed only when it reaches the lower part of the small intestine
.
In April 2021, the FDA accepted Nefecon's NDA for the treatment of primary IgA nephropathy (IgAN) and granted the NDA priority review status.
The PDUFA target date is September 15, 2021
.
Since the FDA believes that the further data analysis of NeflgArd submitted by Calliditas is a major revision of the NDA, Nefecon's PDUFA date has been delayed, and the PDUFA date has been postponed to December 15, 2021
.

13.
maribavir

Maribavir belongs to the benzimidazole nucleoside drugs, which can target to inhibit the UL97 protein kinase of cytomegalovirus (CMV), thereby potentially affecting several key processes of CMV replication, including viral DNA replication, viral gene expression, encapsidation and maturation The capsid escapes from the nucleus of the infected cell
.
Its key phase 3 study TAK-620-303 in CMV infection/disease transplant recipients with refractory, with or without drug resistance showed that: in the eighth week of the study, they received antiviral therapy, with or without resistance Among transplant recipients with medicinal properties and CMV disease/infection, 55.
7% of patients in the maribavir treatment group achieved confirmed CMV viremia clearance, while 23.
9% in the conventional treatment group reached the primary study endpoint
.

In May 2021, Takeda announced that the FDA will accept maribavir treatment of refractory, with or without drug-resistant CMV infection NDA, and grant the application priority review
.
If approved, maribavir will be the first and only drug used in transplant recipients to treat R/R CMV infection after transplantation
.

14.
balstilimab

Balstilimab is a new fully human monoclonal immunoglobulin G4 (IgG4) designed to block the interaction of PD-1 with its ligands PD-L1 and PD-L2
.
In June 2021, the FDA granted the drug priority review status for the treatment of recurrent or metastatic cervical cancer that has progressed during or after chemotherapy.
The PDUFA date is December 16, 2021
.

15.
efgartigimod

Efgartigimod is an antibody fragment under development based on the ABDEG Fc engineering technology platform.
It is a pioneering new IgG transport receptor (FcRn) antagonist.
FcRn is widely expressed throughout the body.
Blocking FcRn can reduce the expression of IgG antibodies.
Level, treatment of autoimmune diseases known to be driven by pathogenic IgG antibodies
.
The results of the key phase 3 study ADAPT for the treatment of systemic myasthenia gravis (gMG) showed that efgartigimod treatment can significantly improve the strength and quality of life of patients with gMG
.

Currently, the drug is under review in the United States, and the PDUFA date is December 17, 2021
.
If approved, efgartigimod will become the first and only FcRn antagonist to receive regulatory approval
.

16.
gefapixant

Gefapixant (MK-7264) is an oral, selective P2X3 receptor antagonist.
In March 2021, the FDA accepted the drug for the treatment of refractory chronic cough (RCC) or unexplained chronic cough (UCC) in adult patients NDA
.
If it goes well, gefapixant will become the first drug specifically for the treatment of RCC and UCC
.