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From 0 to 1, take stock of the "first" innovative drugs approved by the FDA in 2022

 Last Update: 2023-02-02
 Source: Internet
 Author: User

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The painful and long year of 2022 has finally passed
.
This year, the impact of the new crown epidemic continued to intensify, and the clinical trials of new drugs were deeply affected
due to transportation restrictions and patient dropout.
Compared to previous years, the number of new drugs approved by the FDA decreased in 2022, with a total of 37 new drugs
approved throughout the year.
However, the innovative content of approved new drugs is extremely high, including "first" drugs
in many fields.

1 First TCR therapy: Kimmtrak

TCR-T therapy is a type of adoptive immune cell therapy, and compared to CAR-T therapy, both modify the patient's own T lymphocytes ex vivo and infuse them back into the patient's body to kill tumor cells
.
The difference is that the mechanism of antigen recognition is different - the target antigen recognized by CAR-T is a cell surface protein, while TCR-T can target the intracellular antigen fragment presented by the MHC molecule
.
This difference in principle leads to a wider
range of targets for TCR-T therapy.
In addition, the safety profile is higher
due to less TCR-T-mediated cytokine release.
More importantly, unlike CAR-T, which mainly treats hematological tumors, TCR-T has the potential
to conquer solid tumors.

On January 26, 2022, biotechnology company Immunocore announced that its TCR-T therapy Kimmtrak (tebentafusp-tebn) has been approved by the FDA, the first FDA-approved therapy
for the treatment of unresectable or metastatic uveal melanoma.
It is also the first FDA-approved T cell receptor (TCR) therapy
.

The approval is based on a phase III clinical trial showing that Kimmtrak, as first-line therapy, reduces the risk of death by 49% in patients with untreated uveal melanoma (HR = 0.
51, 95% CI: 0.
37, 0.
71, p<0.
0001).

The median overall survival was 21.
7 months in the Kimmtrak group and 16.
0 months
in the active control group.

In fact, TCR-T therapy involves more objects, purification and testing and analysis than traditional drugs, so its development is more difficult and the failure rate is higher
.
The approval of Kimmtrak has achieved a breakthrough
of "from 0 to 1".

2 The first domestic CAR-T therapy approved by the FDA

On February 28, 2022, a CAR-T therapy developed by Nanjing Legend Biologics, Carvykti (Cedakicel), was approved by the FDA for the treatment of relapsed/refractory multiple myeloma
in adults.
This is the first domestic CAR-T cell therapy approved by the FDA
.

Carvykti is a CAR-T therapy targeting B-cell maturation antigen (BCMA) with a 4-1BB co-stimulatory domain and two BCMA-targeted single-domain antibodies
。 ONE OF THE DISCLOSED ITEMS CALLED CARTITUDE-1 criticality In the phase Ib/II study, results showed that at a median follow-up of 17.
1 months, the patient's response to Carvykti deepened over time, did not achieve median duration of response (DOR), had a median response time to first time of 1.
0 months (range, 0.
7–3.
3), and a median time to optimal response was 2.
6 At months (range, 0.
9–13.
6), the PFS rate at 15 months was 70% (95% CI, 45.
1–85.
3), and all 16 patients with evaluable samples achieved MRD negative
.

Shortly after the FDA approval, Carvykti was launched in Europe in May and September in Japan for the treatment of patients
with relapsed/refractory multiple myeloma who have received four or more lines of therapy, including proteasome inhibitors, immunomodulators, and anti-CD38 monoclonal antibodies.

Carvykti's successful "going to sea" is a milestone in the history of innovative drugs in China
.

3 The first new topical drug for psoriasis - Vtama cream

On May 25, 2022, Dermavant Sciences announced that the FDA approved its Vtama (1% tapinarof) cream is available for topical use in the treatment of adult patients
with plaque-like psoriasis.
This is the first FDA-approved topical new molecular entity
for psoriasis treatment in 25 years.
This innovative therapy was first launched in China in 2019 for the topical treatment of mild to moderate stable psoriasis vulgaris (mainly plaque psoriasis)
in adults.

Vtama is an aromatic hydrocarbon receptor modulator, and its mechanism of action is to inhibit the IL-17-mediated inflammatory response by regulating the function of aromatic hydrocarbon receptors, which is the star pathway
for the treatment of psoriasis.

In two pivotal Phase III clinical trials, Vtama met all primary and secondary endpoints
.
At week 12, approximately 35% and 40% of patients treated with Vtama met the primary endpoint of clearance of skin symptoms or near-clearance and at least grade 2 improvement, compared with only 6%
in the control group.
About 36% and 47% of patients treated with Vtama met the secondary endpoint, which was a proportion of participants ≥ 75% improvement in PASI (psoriasis area and severity index), compared with only about 10% and 7%
in the control group.
In the long-term expansion study, 81.
7% of patients considered this therapy to be more effective
than previously used topical therapy.

Vtama was first isolated
from a symbiotic bacterial metabolite of a soil nematode.
Its molecular weight is only 254 Da, with good skin permeability
.
The approved treatment indications cover all psoriasis patients, and there are no restrictions
on the duration of use and body use.

4 The first approved TYK2 allosteric inhibitor - Sotyktu

On September 10, 2022, Bristol-Myers Squibb (BMS) announced that the FDA has approved its oral selective TYK2 inhibitor Sotyktu (deucravacitinib) for the treatment
of adults with moderate to severe psoriasis.
This is the first FDA-approved TYK2 inhibitor
.

TYK2, a member of the JAK family, is an intracellular signaling kinase
that mediates IL-23, IL-12, and type I interferon (IFN) signaling.
As an orally selective TYK2 inhibitor, Sotyktu selectively inhibits the activity
of TYK2 by "locking" TYK2 in an inactivated state by binding to the regulatory domain of TYK2.
Sotyktu is a safer and more effective treatment option
than JAK inhibitors.

In pivotal phase III clinical trials, the results showed that after 16 weeks of treatment, PASI was achieved in patients in the Sotyktu group The proportions of 75 were 58.
7% and 53.
6%, respectively, which were significantly better than the active control group and the placebo group
.
This proportion increased further with the continuation of treatment, rising to 69.
0% and 59.
3%
at 24 weeks of treatment.
After 60 weeks of treatment, PASI is achieved The proportion of patients with 75 criteria was 77.
7%.

5 The first type 1 diabetes drug – Tzield

On November 18, 2022, the FDA approved Provention Bio's drug Tzield (teplizumab) is marketed to delay disease progression
in people at high risk of type 1 diabetes.
Tzield is an anti-CD3 monoclonal antibody that works by binding to specific immune cells so that they no longer attack insulin-producing cells
.

The results showed that among people at high risk of type 1 diabetes treated with Tzield, the proportion of patients with type 1 diabetes who did not develop clinical symptoms reached 50% at a median follow-up of 2.
5 years, more than
twice that of the placebo group (22%).
In addition, the Tzield treatment group significantly improved the function of islets β cells, as well as the ability to
secrete insulin.

Type 1 diabetes is a chronic disease caused by damage to the β cells of the pancreas, resulting in very little insulin secreted in the body, which in turn leads to abnormally high blood sugar levels and abnormal glucose metabolism
.
At present, the mechanism of type 1 diabetes has not been fully studied, and there is no cure
for the disease.
Tzield is the first drug
to delay the onset of type 1 diabetes.

6 The first fecal microbiome drug, Rebyota

On December 1, 2022, Rebiotix Corporation announced that its fecal microbiome therapy, Rebyota (RBX2660), was approved by the US FDA for the purpose of preventing the recurrence
of infection in adult patients with Clostridium difficile infection (CDI) after receiving antibiotic therapy.

Rebyota is made from the feces of eligible donors, does not contain any antibiotics, is administered rectally in a single dose, and is designed to help patients restore their gut microbiome and avoid reinfection
with CDI.
In the comprehensive analysis of two clinical trials, Rebyota was shown to be effective in avoiding CDI recurrence in patients, and the rate of successful avoidance of CDI recurrence in patients in the Rebyota group was 70.
6%, which was significantly better than 57.
5%
in the placebo group.
Rebyota, the FDA's first approved fecal microbiome therapy, represents an important milestone and offers another treatment option
to avoid relapse treatments for CDI.

The above are several "firsts" approved by the FDA in 2022, involving TCR-T, TYK2 inhibitors, intestinal microbial therapy, etc
.
The past year has been a year of stricter FDA approvals, and it has also been a year of ups and downs for pharmaceutical companies, but in the end
, it has been full of harvests.
We are confident
in the upcoming new cycle.

Reference:

1.
Immunocore announces FDA approval of KIMMTRAK® (tebentafusp-tebn) for the treatment of unresectable or metastatic uveal melanoma, Retrieved January 26, 2022, from ；

2.
Nathan et al.
, (2021).
Overall Survival Benefit with Tebentafusp in Metastatic Uveal Melanoma.
NEJM, DOI: 10.
1056/NEJMoa2103485；

3.
；

4.
FDA Approves First Drug That Can Delay Onset of Type 1 Diabetes, Retrieved November 17, 2022, from ；

5.
FDA Approves First Fecal Microbiota Product.
Retrieved November 30, 2022 from

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