Drug Trials Snapshots: VONJO

HOW TO USE THIS SNAPSHOT

The information provided in Snapshots highlights who participated in the key clinical trials that supported the original FDA approval of this drug, and whether there were differences among sex, race, age, and ethnic groups. The “MORE INFO” bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs.

LIMITATIONS OF THIS SNAPSHOT

Do not rely on Snapshots to make decisions regarding medical care. Always speak to your healthcare provider about the benefits and risks of a drug.

Some of the information in this Snapshot is for presentation purposes and does not represent the approved conditions of use of this drug. Refer to the VONJO Prescribing Information for all of the approved conditions of use of this drug (e.g., indication(s), population(s), dosing regimen(s), safety information).

Snapshots are limited to the information available at the time of the original approval of the drug and do not provide information on who participated in clinical trials that supported later approvals for additional uses of the drug (if applicable).

VONJO (pacritinib)
VON-joh
CTI Biopharma Corp
Approval date: February 28, 2022

DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

VONJO is a drug that is used to treat adult patients with a rare type of bone marrow disorder called myelofibrosis who also have platelet counts below 50,000/µL. Platelets are cellular components in the blood. Myelofibrosis is a chronic disorder where scar tissue forms in the bone marrow and the production of the blood cells moves from the bone marrow to the spleen and liver, causing organ enlargement.

How is this drug used?

VONJO is a capsule taken by mouth twice daily.

Who participated in the clinical trials?

The FDA approved VONJO based on evidence from one clinical trial of 63 patients with myelofibrosis who had platelet counts below 50,000/µL. The trial was conducted in the United States and countries in Europe and Asia.

How were the trials designed?

The benefit and side effects of VONJO for myelofibrosis were evaluated in one clinical trial that enrolled 311 patients. The trial enrolled adult patients with myelofibrosis and enlarged spleen who had platelet counts below 100,000/µL. The benefit of VONJO was established among 63 patients who had platelet counts below 50,000/µL. All patients had the size of their spleen measured at the beginning of the trial using either magnetic resonance imaging (MRI) or computerized tomography (CT) scan. Patients received one of two doses of VONJO (400 mg once daily or 200 mg twice daily) or best available therapy for six months. Best available therapy included any physician-selected treatment for myelofibrosis and may have included ruxolitinib, hydroxyurea, glucocorticoids, erythropoietic agents, immunomodulatory agents, mercaptopurine, danazol, interferons, cytarabine, or melphalan. Best available therapy also included no treatment (“watch and wait”) or symptom-directed treatment without myelofibrosis-specific treatment. The 400 mg dose of VONJO was to establish safety and is not an approved dosage regimen. Patients who had prior treatment for their myelofibrosis were allowed to enroll in the trial. After receiving VONJO or best available therapy for 6 months, patients had their spleen size measured again.

The benefit of VONJO was evaluated by measuring the percentage of patients who achieved at least 35% shrinkage of the spleen and comparing it to the percentage of patients who received best available therapy.

DEMOGRAPHICS SNAPSHOT

Figure 1 summarizes how many male and female patients were enrolled in the clinical trial used to evaluate the efficacy of VONJO.

Figure 1. Baseline Demographics by Sex (Efficacy Population)

_{Source: Adapted from FDA Review}

Figure 2 summarizes the percentage of patients by race enrolled in the clinical trial used to evaluate the efficacy of VONJO.

Figure 2. Baseline Demographics by Race (Efficacy Population)

_{Source: Adapted from FDA Review}

Figure 3 summarizes how many patients by age were enrolled in the clinical trial used to evaluate the efficacy of VONJO.

Figure 3. Baseline Demographics by Age (Efficacy Population)

_{Source: Adapted from FDA Review}

Who participated in the trials?

Table 1. Baseline Demographics of Efficacy Trials

Demographic	VONJO N=31 n(%)	Best Available Therapy N=32 n(%)
Age, years
<65	12 (38.7)	7 (21.9)
≥65	19 (61.3)	25 (78.1)
Sex
Female	12 (38.7)	16 (50.0)
Male	19 (61.3)	16 (50.0)
Race
White	30 (96.8)	30 (93.8)
Black/Other	1 (3.2)	1 (3.1)
Unknown	0 (0.0)	1 (3.1)

_{Source: Adapted from FDA review

*Race was not collected for patients who were not allowed to be asked per local regulations}

What are the benefits of this drug?

Of the 31 patients who received VONJO, 29.0% had a decrease in the size of their spleen by 35% or more in comparison to 3.1% percent of the 32 patients who received other treatments for myelofibrosis (which is called best available therapy).

VONJO was approved under FDA’s accelerated approval program, which provides earlier patient access to a promising new drug while the company continues to conduct clinical trials to confirm that the drug works well.

An additional trial is ongoing to assess whether there is a clinical benefit.

Were there any differences in how well the drug worked in clinical trials among sex, race, and age?

Sex: VONJO worked similarly in males and females.
Race: The number of patients of races other than White was small; therefore, differences in how VONJO worked among races could not be determined.
Age: VONJO worked better in patients 65 years of age and older compared to those younger than age 65.

Were there any differences in how well the drug worked in clinical trials among sex, race, and age groups?

Table 2. Proportion of Patients With at Least 35% Reduction in Spleen Volume From Baseline to Week 24 by Sex

Parameter	VONJO	Best Available Therapy
Male	N=19	N=16
With ≥35% reduction, n (%)	6 (31.6)	1 (6.3)
95% CI¹	12.6, 56.6	0.2, 30.2
Difference (97.5% CI)²	25.3 (-6.2, 50.6)
Female	N=12	N=16
With ≥35% reduction, n (%)	3 (25.0)	0
95% CI¹	5.5, 57.2	0.0, 20.6
Difference (97.5% CI)2	25.0 (-6.6, 52.7)

_{Source: Adapted from FDA review

¹95% CIs are based on the Clopper-Pearson method.

² 97.5% CIs are based on the Agresti-Caffo method.

Note: Percentages in each subgroup are based on the number of patients in that subgroup.

Abbreviations: CI, confidence interval}

Table 3. Proportion of Patients With at Least 35% Reduction in Spleen Volume From Baseline to Week 24 by Age

Parameter	VONJO	Best Available Therapy
<65 years	N=12	N=7
With ≥35% reduction, n (%)	2 (16.7)	0
95% CI¹	2.1, 48.4	0.0, 41.0
Difference (97.5% CI)²	16.7 (-23.7, 44.3)
≥65 years	N=19	N=25
With ≥35% reduction, n (%)	7 (36.8)	1 (4.0)
95% CI¹	16.3, 61.6	0.1, 20.4
Difference (97.5% CI)²	32.8 (4.4, 57.0)

What are the possible side effects?

VONJO may cause bleeding; diarrhea; low platelet counts; abnormal heart rhythm; major adverse cardiac events including cardiovascular death, myocardial infarction, and stroke; blood clots; secondary cancers; and infection.

Other serious side effects include anemia, pneumonia, heart failure, disease progression, and fever.

The most common side effects are diarrhea, low platelet counts, nausea, anemia, and swelling in the legs, feet, and hands.

What are the possible side effects?

Table 4. Adverse Reactions Reported in ≥10% Patients Receiving VONJO 200 mg Twice Daily or Best Available Therapy During Randomized Treatment

	VONJO 200 mg BID N=106		Best Available Therapy N=98
	All Grades^a	Grades ≥3	All Grades^a	Grades ≥3
Adverse Reaction	%	%	%	%
Diarrhea	48	4	15	0
Thrombocytopenia	34	32	23	18
Nausea	32	1	11	1
Anemia	24	22	15	14
Peripheral edema	20	1	15	0
Vomiting	19	0	5	1
Dizziness	15	1	5	0
Pyrexia	15	1	3	0
Epistaxis	12	5	13	1
Dyspnea	10	0	9	3
Pruritus	10	2	6	0
Upper respiratory tract infection	10	0	6	0
Cough	8	2	10	0

_{Source: VONJO Prescribing Information

^aGrade by CTCAE version 4.03

Abbreviations: BID, twice daily; CTCAE, Common Terminology Criteria for Adverse Events}

Were there any differences in side effects among sex, race and age?

Sex: Differences in the occurrence of side effects between males and females could not be determined because of the small number of patients in each treatment group.

Race: The number of patients of races other than White was small; therefore, differences in side effects related to VONJO treatment among races could not be determined.

Age: The majority of patients were adults 65 years of age and older. There were not enough patients younger than 65 years old to determine whether there was any difference in side effects between older and younger patients.

GLOSSARY

Clinical Trial: Voluntary research studies conducted in people and designed to answer specific questions about the safety or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments.

Comparator: A previously available treatment or placebo used in clinical trials that is compared to the actual drug being tested.

Efficacy: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial.

Placebo: An inactive substance or “sugar pill” that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.

Subgroup: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.

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