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Drug Discovery 12 new drugs approved by the FDA in the third quarter of 2022

 Last Update: 2022-10-31
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The FDA approved 12 new drugs in the third quarter of this year (Table 1), with slow approval progress in the first half of the year and an increase in the pace of approvals in the second half, which many hope to maintain for the rest of the year
.

The rate of 12 new drugs has increased

Author | Small fish

author

Table 1: FDA-approved new drugs in Q3 2022

1、Betibeglogene autotemcel (ZYNTEGLO; Bluebird Bio)

1、Betibeglogene autotemcel (ZYNTEGLO; Bluebird Biology)1, Betibeglogene autotemcel (ZYNTEGLO; Bluebird Biology)1, Betibeglogene autotemcel (ZYNTEGLO; Bluebird Bio)

ON AUGUST 17, BLUEBIRD ANNOUNCED THAT THE FDA APPROVED ZYNTEGLO,® THE FIRST GENE THERAPY
FOR PATIENTS WITH β-THALASSEMIA WHO REQUIRE REGULAR RED BLOOD CELL TRANSFUSIONS.

Gene therapy for patients with β-thalassemia who require regular red blood cell transfusions

β-thalassemia is a rare inherited blood disorder caused by mutations in the β-globin gene, characterized by a significant decrease or absence of hemoglobin in adults, severe cases requiring regular blood transfusions, a lengthy process that patients usually experience every
2-5 weeks.
Data from the Cooley's Anemia Foundation suggest that the median age at death of U.
S.
transfusion-dependent β-thalassaemia patients who have died over the past decade is just 37.

The median adult with a significant reduction or deletion of hemoglobin in β-thalassaemia β-globin gene mutations was only 37 years

THE APPROVAL OF ZYNTEGLO IS THE CULMINATION
OF NEARLY 10 YEARS OF CLINICAL RESEARCH IN GENE THERAPY IN TRANSFUSION-DEPENDENT β-THALASSEMIA PATIENTS.
ZYNTEGLO works by the fact that the functional β-globin gene is added (ex vitro) to the patient's own hematopoietic stem cell (HSC) cells and then injected into the patient to allow them to reach near-normal levels of total hemoglobin
without regular red blood cell transfusions.
Risks: delayed platelet implantation; Neutrophil transplantation failure; LVV-mediated intrusive tumorigenesis; Hypersensitivity, etc
.

Functional β-globin genes are added (ex vitro) to the patient's own hematopoietic stem cell (HSC) cells and then injected into the patient to allow them to reach near-normal levels of total hemoglobin without regular red blood cell transfusions

2.
Olipudase alfa (Xenpozyme; Sanofi)

2.
Olipudase alfa (Xenpozyme; Sanofi) 2.
Olipudase alfa (Xenpozyme; Sanofi) 2.
Olipudase alfa (Xenpozyme; Sanofi)

On August 30, the FDA approved XenpozymeTM (olipudase alfa-rpcp) for the treatment of adult and pediatric patients with non-central nervous system (CNS) manifestations of acid sphingomyelinase deficiency (ASMD), the first therapy specifically designed to treat ASMD and the only currently approved therapy
for the disease.

Non-central nervous system (CNS) manifestations of acid sphingomylase deficiency (ASMD).

ASMD is a very rare progressive genetic disorder with high
morbidity and mortality.
It is estimated that fewer than 120 patients have been diagnosed with ASMD in the United States
.
In patients with ASMD, the deficiency of the ASM enzyme leads to the accumulation
of sphingomyelin in various tissues.
Signs and symptoms of ASMD can appear in infancy, childhood, or adulthood, such as an enlarged spleen or liver, difficulty breathing, lung infections, abnormal bruising, or bleeding
.
Until now, the management of ASMD has included supportive care, monitoring to detect potential complications
.

In patients with ASMD, the deficiency of the ASM enzyme leads to the accumulation
of sphingomyelin in various tissues.
Enlarged spleen or liver, difficulty breathing, lung infection, unusual bruising or bleeding

Xenpozyme is a hydrolytic lysosomal sphingomyelin-specific enzyme designed to replace the deficient or defective acid sphingomyelinase (ASM) and effectively break down lipid sphingomyelin.

Lysosomal sphingomylinin-specific enzyme designed to replace the deficient or defective acid sphingomyelinase (ASM) to effectively break down lipid sphingomyelin.

3.
Spesolimab (Spevigo; Boehringer Ingelheim)

3.
Spesolimab (Spevigo; Boehringer Ingelheim)3.
Spesolimab (Spevigo; Boehringer Ingelheim)3.
Spesolimab (Spevigo; Boehringer Ingelheim)

On September 1, Spesolimab was the first treatment approved by the FDA to treat attacks of generalized pustular eruption (GPP
).

Generalized pustular eruption (GPP)

GPP is a rare, heterogeneous, and potentially life-threatening neutrophil skin disease
.
Due to the accumulation of neutrophils in the skin, the patient suffers from generalized pain, long sterile pustules
.
While flares can vary in severity in GPP patients, they can be life-threatening
if left untreated, causing complications such as sepsis and organ failure.
This chronic systemic disease has a significant impact on the quality of life and increases the medical burden on
patients.

Neutrophils accumulate in the skin, causing the patient to suffer from generalized pain, long sterile pustules
.

Figure 1: 12 weeks of treatment for GPP patients

Spesolimab is a novel humanized selective antibody that blocks the activation of the interleukin-36 receptor (IL-36R), a signaling pathway within the immune system involved in the pathogenesis of several autoinflammatory diseases
, including GPP.

Novel humanized selective antibodies block activation of the interleukin-36 receptor (IL-36R).

Figure 2: Spevigo pharmacology and sales packaging

4.
Daxibotulinumtoxin A (Daxxify; Revance Therapeutics)

On September 7, REVANCE announced that the FDA has approved DAXXIFY™ for injection (Daxibotulinumtoxin A-lanm) for temporary improvement of moderate to severe wrinkles in adults, especially brow wrinkles
.

It is the first and only neuromodulator to be stabilized using Peptide Exchange Technology ™ (PXT), free of human serum albumin and animal-based components for long-lasting effects
.

Neuromodulator stabilized using peptide exchange technology ™ (PXT).

Figure 3: DAXXIFY sales and usage

DAXXIFY™ is safe, well tolerated, and there are no serious treatment-related adverse events
in clinical trials.
Headache (6%) was observed in key trials, followed by ptosis (2%) and facial paralysis, including facial asymmetry (1%)
.

5.
Deucravacitinib (Sotyktu; Bristol Myers Squibb)

On September 8, the FDA approved Sotyktu™ (deucravacitinib) for oral treatment
in adults with moderate to severe plaque psoriasis.

Adults with moderate to severe plaque psoriasis

Psoriasis is a widespread chronic, systemic immune-mediated disease that severely impairs the health, quality of life and productivity of
patients.
At least 100 million people worldwide are affected by the disease, which is a serious global problem
.
Up to 90% of psoriasis patients have psoriasis vulgaris or plaque psoriasis, which is characterized by pronounced round or oval plaques, often covered with silvery-white scales
.

Psoriasis vulgaris or plaque psoriasis

Figure 4: Sotyktu sales packaging

Sotyktu™ is a selective allosteric inhibitor
of tyrosine kinase 2 (TYK2).
TYK2 is a member of
the JAK family.
Sotyktu binds to the regulatory domain of TYK2, stabilizes the regulatory domain and catalytic domain of enzymes, inhibits TYK2 activation and activation of its downstream signaling sensors and transcriptional activators (STATs
).
Currently, the exact mechanism linking inhibition of the TYK2 enzyme to the therapeutic effect in the treatment of adults with moderate to severe plaque psoriasis is unknown
.

Sotyktu, a selective allosteric inhibitor of tyrosine kinase 2 (TYK2), binds to the regulatory domain of TYK2, stabilizes the regulatory and catalytic domains of enzymes, inhibits TYK2 activation and the activation
of its downstream signaling sensors and transcriptional activators (STATs).

6.
Eflapegrastim (Rolvedon; Spectrum Pharmaceutical)

SEPTEMBER 9, THE FDA HAS APPROVED ROLVEDON™ (EFLAPEGRASTIM-XNST) INJECTION TO REDUCE THE INCIDENCE OF INFECTIONS, SUCH AS NEUTROPENIC FEVER
, IN ADULT PATIENTS RECEIVING MYELOSUPPRESSIVE ANTICANCER DRUGS FOR NON-MYELOID MALIGNANCIES.

Reduce the incidence of infection

ROLVEDON™ INJECTION IS A LONG-ACTING GRANULOCYTE COLONY-STIMULATING FACTOR (G-CSF).

Risks: splenic rupture; Acute respiratory distress syndrome; Severe allergic reactions; sickle cell crisis in patients with sickle cell disease; Glomerulonephritis; Leukocytosis; Thrombocytopenia; Capillary leakage syndrome; Potential effects of tumor growth stimulation on malignant cells, etc
.

A long-acting granulocyte colony-stimulating factor (G-CSF)

Figure 5: ROLVEDON sales packaging

7.
Terlipressin (Terlivaz; Mallinckrodt Pharmaceuticals)

On September 14, the FDA approved Terlivaz (Terlipressin) for the treatment of hepatorenal syndrome (HRS).

Hepatorenal syndrome (HRS), which involves a rapid decline in kidney function, is an acute and life-threatening condition that occurs in patients with
advanced liver disease.
HRS is divided into two distinct types: a rapidly progressive type that leads to acute renal failure and patients are usually hospitalized; Chronic type, which progresses
over weeks to months.
HRS, which involves a rapid decline in kidney function, affects 30,000 to 40,000 Americans each year, with a median survival time of about 2 weeks and a mortality rate of more than 80 percent
within 3 months if left untreated.

Rapid decline in kidney function

Figure 5: Terlivaz chemical formula and packaging

Terlivaz is a synthetic vasopressin analogue, which is both a predrug of lysine-vasopressin and has its own pharmacological activity
.
Increases renal blood flow
in patients with hepatorenal syndrome by reducing blood circulation in portal hypertension and portal vein vessels, as well as increasing effective arterial volume and mean arterial pressure (MAP).
Risks: severe or fatal respiratory failure, ischemia, fetal toxicity, etc
.

Synthetic vasopressin analogues increase renal blood flow
in patients with hepatorenal syndrome by reducing blood circulation in portal hypertension and portal vein vessels, as well as increasing effective arterial volume and mean arterial pressure (MAP).

8.
Futibatinib（Lytgobi; Otsuka Holdings)

8.
Futibatinib（Lytgobi; Otsuka Holdings)8.
Futibatinib（Lytgobi; Otsuka Holdings)8.
Futibatinib（Lytgobi; Otsuka Holdings)

On September 14, the FDA approved the FGFR inhibitor Futibatinib for the treatment of adult patients with treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma (iCCA) containing fibroblast growth factor receptor 2 (FGFR2) gene fusion or recombination
.

Fibroblast growth factor receptor 2 (FGFR2) gene fusion or recombination

In the United States, about 8,000 people are diagnosed with cholangiocarcinoma (intrahepatic, extrahepatic) each year, about 20% of CCA diagnosed patients have intrahepatic disease, and of this 20%, about 10-16% of patients have FGFR2 gene fusion or recombination, which promotes tumor proliferation
.

FGFR2 gene fuses or recombinates to promote tumor proliferation
.

Figure 6: Lytgobi chemical formula

Futibatinib (TAS-120) selectively binds to the ATP binding bag of FGFR1-4, inhibits FGFR-mediated signal transduction pathways, reduces tumor cell proliferation and promotes tumor cell death
.

ATP binding bags that selectively bind FGFR1-4 inhibit FGFR-mediated signal transduction pathways, reduce tumor cell proliferation and promote tumor cell death

9.
Elivaldogene autotemcel（Skysona; Bluebird Bio)

On September 16, the FDA approved SKYSONA®, also known as eli-cel, to slow the progression
of neurological dysfunction in boys ages 4-17 with early active cerebral adrenal dysplasia (CALD).

CALD is a progressive and irreversible neurodegenerative disease manifested by irreversible destructive neurological decline, major functional disabilities such as loss of communication, cortical blindness, tube feeding, complete incontinence, wheelchair dependence, complete loss of voluntary movement, etc
.
Early diagnosis and treatment of CALD is critical because nearly half of untreated patients die
within five years of symptom onset.

Progressive and irreversible neurodegenerative diseases

The disease is caused by a mutation in the ABCD1 gene, which affects the production of adrenal leukocyte dystrophy protein (ALDP), which subsequently leads to the accumulation of very long-chain fatty acids (VLCFA), mainly in
the white matter of the brain and spinal cord.
This accumulation leads to the breakdown of the myelin sheath, which is the protective sheath needed for nerve cells to function effectively, especially for thinking and muscle control
.

Caused by mutations in the ABCD1 gene

Figure 6: Skysona gene therapy

Skysona is a single-use gene therapy that uses Lenti-D lentiviral vectors (LVVs) for in vitro transduction, introducing a functional copy of the ABCD1 gene into the patient's own hematopoietic stem cells in vitro, and then infusing it back into the patient to produce the ALDP protein and break down VLCFAs
.
Risk: hematological malignancy; Severe infections; Long-term cytopenia; delayed platelet implantation; risk of neutrophil transplant failure; Hypersensitivity, etc
.

Disposable gene therapy, using Lenti-D lentiviral vector (LVV) for in vitro transduction, introduces a functional copy of the ABCD1 gene into the patient's own hematopoietic stem cells in vitro, and then infuses it back into the patient to produce ALDP protein and break down VLCFAs

10.
Gadopiclenol (Elucirem; Guerbet)

On September 21, the FDA approved Elucireem™, a new macrocyclic gadolinium contrast agent
for contrast-enhanced magnetic resonance imaging (MRI), after priority review.

Gadolinium cresol, the active substance of Elucirem™, is designed to have two water molecule exchange sites to increase relaxation (relaxation is a physics term that refers to the process of gradually returning from a state to equilibrium in a gradual physical process), using only half
the dose of conventional gadolinium compared to other non-specific GAGBAS.

Figure 7: Magnetic resonance imaging (MRI) equipment

The product is used to detect and visualize lesions of vascular abnormalities in the
central nervous system (brain, spine and related tissues) and body (head and neck, chest, abdomen, pelvis and musculoskeletal system).

Detect and visualize lesions of abnormal blood vessels in the central nervous system (brain, spine, and related tissues) and body (head and neck, chest, abdomen, pelvis, and musculoskeletal system).

11.
Omidenepag isopropyl (Omlonti； Santen Pharmaceutical/UBE Corporation)

SEPTEMBER 26, THE FDA APPROVED OMLONTI® AS AN EYE DROP TO LOWER INTRAOCULAR PRESSURE FOR PATIENTS WITH
OPEN-ANGLE GLAUCOMA AND HIGH INTRAOCULAR PRESSURE.

Glaucoma causes damage to the optic nerve, leading to vision defects, and in many countries, glaucoma is the leading cause of visual impairment, including vision loss and blindness
.
Since it is usually gradual and irreversible, in treatment, it is crucial to control progression through early detection and early treatment, and lowering intraocular pressure is an effective way to
avoid optic nerve damage.
The number of glaucoma patients worldwide was 76 million in 2020 and is expected to increase to 95 million by 2030
.

Glaucoma causes optic nerve damage, causing vision defects and reducing intraocular pressure is an effective way to avoid optic nerve damage, the number of glaucoma patients worldwide was 76 million in 2020 and is expected to increase to 95 million by 2030

Figure 8: Open-angle glaucoma

OMLONTI® is an eye drop developed by Santen and UBE to treat glaucoma and high intraocular pressure
.
Among them, the active ingredient Omidenepag isopropyl is a selective EP2 receptor agonist, which stimulates EP2 receptors and promotes the outflow of aqueous humor through the fibrous band channel and the uveoscleral channel, and is the only product
with this pharmacological effect.

Selective EP2 receptor agonist, stimulates EP2 receptors and promotes the outflow of aqueous humor through the fibrous band channel and uveal scleral channel

FIGURE 9: OMLONTI eye drops

12.
Sodium phenylbutyrate and taurine glycol (Relyvrio; Emilix Pharmaceutical)

SEPTEMBER 29, THE FDA APPROVED RELIVRIO™ TO TREAT ADULTS
WITH AMYOTROPHIC LATERAL SCLEROSIS (ALS, FROSTBITE).

ALS is a progressive neurodegenerative disease caused
by the death of motor neurons in the brain and spinal cord.
The loss of motor neurons in ALS patients leads to deterioration of muscle function, inability to move and speak, paralysis of breathing, and eventual death
.
More than 90% of patients with ALS have sporadic disease and no family history
.

ALS is a progressive neurodegenerative disease caused
by the death of motor neurons in the brain and spinal cord.

Figure 10: ALS Ice Bucket Challenge

RELYVRIO, an oral fixed-dose drug, is a combination of two drugs, Sodium Phenylbutrate and Taurursodiol,™ and the team is exploring its potential
for the treatment of other neurodegenerative diseases.
Risks: increased levels of bile acids, worsening of diarrhea; salt (sodium) retention, etc
.

A combination of sodium phenylbutrate and taurursodiol for oral administration

Figure 11: RELYVRIO packaging

brief summary

Part of the reason for the slowdown in FDA drug approvals may be related to the coronavirus pandemic, which restricted agency staff from traveling abroad to inspect overseas manufacturing facilities, leading to delays
in some resolutions.
Other potential reasons include FDA staffing shortages and tighter approval standards
.
However, we can see that gene therapy, combination preparations, monoclonal antibodies, small molecule inhibitors, etc.
have shown their magic in rare and common diseases, bringing a ray
of hope to the majority of patients.

The new crown pneumonia pandemic resolution postponed the FDA's staff shortage approval standards, tightened gene therapies, combination preparations, monoclonal antibodies, small molecule inhibitors, etc.
in rare and common diseases, bringing a ray of hope to the majority of patients

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