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 ROMVIMZA Prescribing Information for all 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).

ROMVIMZA (vimseltinib) capsules
ROM VIM’ ZAH
Deciphera Pharmaceuticals, LLC
Original Approval date: February 17, 2025

DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

ROMVIMZA is a colony stimulating factor 1 receptor (CSF1R) inhibitor used to treat adults with a tumor in the protective layer in the tendons of the joint called tenosynovial giant cell tumor (TGCT). It should be used in patients when surgery may make the symptoms worse or cause severe problems.

How is this drug used?

ROMVIMZA is a capsule taken orally twice a week with at least 72 hours between doses, with or without food.

Who participated in the clinical trials?

The FDA approved ROMVIMZA based on safety and efficacy evidence from one clinical trial (NCT05059262) of 123 patients with TGCT. The trial was conducted at 30 sites in 13 countries including Australia, Canada, France, Germany, Hong Kong, Italy, Netherlands, Norway, Poland, Spain, Switzerland, the United Kingdom, and the United States. 

Of the 123 patients enrolled, 13 patients were enrolled in the United States and 110 patients were enrolled outside of the United States. In the patients enrolled in the United States, the median age was 41 years (range 24 to 74 years); 85% of patients were female; 69% were White, 0% were Asian, 15% were Black or African American, and the race of the other 15% was not reported or was unknown.

How were the trials designed?

The benefits and side effects of ROMVIMZA were evaluated in one clinical trial of 123 patients with TGCT. Patients received ROMVIMZA or placebo twice a week by mouth until the disease worsened or until they could not tolerate side effects. Neither the patients nor the healthcare providers knew which treatment was being given for 24 weeks. After Week 25, patients who received ROMVIMZA were allowed to continue their treatment, and patients who received placebo were allowed to receive ROMVIMZA. The treatment effect of ROMVIMZA was evaluated at Week 25 by identifying the number of patients whose tumor shrank or grew. Additionally, ROMVIMZA was also evaluated by determining the improvement in range of motion for the affected joint, change in overall physical function, and change in worst pain.

How were the trials designed?

The benefits and side effects of ROMVIMZA were evaluated in a multicenter, randomized placebo-controlled trial in patients with symptomatic TGCT for whom surgery may cause worsening functional limitation or severe morbidity. Eligible patients were randomized to receive either ROMVIMZA or placebo. Patients and healthcare practitioners did not know which treatment was being administered. 

The trial measured the initial tumor size until tumor shrinkage or until growth (overall response rate). Additional supportive measurements used in order to assess improvement were change from baseline in the range of motion of affected joints, change in overall physical function, and change in worst pain.

DEMOGRAPHICS SNAPSHOT

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

Figure 1. Baseline Demographics by Sex, Efficacy Population

Source: Adapted from FDA Review

Figure 2 summarizes how many patients by race were enrolled in the clinical trial used to evaluate the efficacy of ROMVIMZA.

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 ROMVIMZA.

Figure 3. Baseline Demographics by Age, Efficacy Population

Source: Adapted from FDA Review

Figure 4 summarizes how many patients by ethnicity were enrolled in the clinical trial used to evaluate the efficacy of ROMVIMZA.

Figure 4. Baseline Demographics by Ethnicity, Efficacy Population

Source: Adapted from FDA Review

Who participated in the trials?

Table 1 summarizes the demographics of all patients in the efficacy population.

Table 1. Baseline Demographics, Efficacy Population

Demographic ROMVIMZA
N=83
n (%)		 Placebo
N=40
n (%)
Sex

Male

 37 (44.6) 13 (32.5)

Female

 46 (55.4) 27 (67.5)
Age, years

18 to <50

 54 (65.1) 27 (67.5)

50 to <65

 24 (28.9) 9 (22.5)

65 to <75

 3 (3.6) 4 (10.0)

75 to <85

 2 (2.4) 0
Race

Asian

 1 (1.2) 4 (10.0)

Black or African American

 4 (4.8) 0

Native Hawaiian or other Pacific Islander

 0 0

White

 59 (71.1) 21 (52.5)

Not reported

 18 (21.7) 13 (32.5)

Unknown

 1 (1.2) 2 (5.0)
Ethnicity

Hispanic or Latino

 3 (3.6) 1 (2.5)

Not Hispanic or Latino

 62 (74.7) 23 (57.5)

Not reported

 17 (20.5) 15 (37.5)

Unknown

 1 (1.2) 1 (2.5)

Source: Adapted from FDA Review

What are the benefits of this drug? 

In this trial, 40% of 83 patients who were treated with ROMVIMZA experienced a complete or partial shrinkage of their tumor. In comparison, none of the 34 patients who were treated with placebo had complete or partial shrinkage of their tumor(s).

In addition, an analysis of active range of motion for the affected joint showed significant improvement for patients receiving ROMVIMIZA, demonstrating a 4.8× improvement compared to those receiving placebo. Patients receiving ROMVIMZA demonstrated a greater improvement in physical function than those receiving placebo. More patients reported at least 30% improvement in their worst pain with ROMVIMZA compared with placebo (48% compared with 23%), without increasing their use of pain medications by 30% or more.

What are the benefits of this drug (results of trials used to assess efficacy)? 

Table 2 shows the overall response rate (ORR) at Week 25 using Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 criteria assessed by independent central review (IRR) of magnetic resonance imaging (MRI) scans.

Table 2. Efficacy Results, Efficacy Population

Efficacy Parameter ROMVIMZA
N=83		 Placebo
N=40
ORR per RECIST   v1.1, % (95% CI) 40 (29, 51) 0 (0, 9)

Complete response, %

 5 0

Partial response, %

 35 0

p-value

 <0.0001
DOR1

Median (range in months)2

 NR (2.5+, 19.4+) N/A

DOR ≥6 months

 28 (85%) -

DOR ≥9   months

 19 (58%) -
Active ROM3

Mean   change from baseline4 (95% CI)

 18.4 (5.6, 31.2) 3.8 (-10.5, 18.0)
PROMIS-PF (15-item score; ranges from 0 to 100)5    

Mean change from baseline4 (95% CI)

 4.6 (2.7, 6.5) 1.3 (-0.5, 3.0)
BPI-30 response6

Patients with data at baseline and Week   25, n

 68 31

Responders, n (%)

 40 (48.2) 9 (22.5)

(95%   CI)

 (37.1, 59.4) (10.8, 38.5)

Source: Adapted from ROMVIMZA Prescribing information
1 DOR results are based on an additional 6 months of follow-up from the time of ORR analysis.
2 The median DOR was estimated using the Kaplan-Meier method. “+” indicates that the patient’s response was ongoing at last assessment as of the data cutoff date.
3 Active ROM was normalized to the AMA reference standard.
4 Mean change from baseline was estimated from a statistical model for each corresponding endpoint. Baseline means presented include all patients and not only the ones with data at baseline and Week 25.
5 Higher scores of PROMIS-PF indicate better physical functioning.
6 BPI response in Worst Pain is defined as at least a 30% improvement in the mean BPI Worst Pain NRS score (0 to 10 NRS) without a 30% or greater increase in narcotic analgesic use at Week 25.

Abbreviations: AMA, American Medical Association; BPI, Brief Pain Inventory; CI, confidence interval; DOR, duration of response; n, number of patients in the category; N, sample size; N/A, not applicable; NR, not reached; ORR, overall response rate; PROMIS-PF, Patient-reported Outcomes Measurement Information System-Physical Function; ROM, range of motion

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

  • Sex: ROMVIMZA worked similarly in males and females.
  • Race: The number of patients of races other than White was small; therefore, differences in how ROMVIMZA worked among races could not be determined.
  • Age: The majority of patients were adults younger than 65 years of age. The number of patients older than 65 years was limited; therefore, differences in how well ROMVIMZA worked between patients younger and older than 65 years of age could not be determined.

What are the possible side effects?

The most common side effects of ROMVIMZA occurring in ≥20% of patients who received ROMVIMZA are increased liver enzymes, swelling around the eyes, tiredness, rash, increased cholesterol levels in the blood, swelling of the hands or feet, swelling of the face, decreased white blood cells, and itchy skin. 

ROMVIMZA can cause serious side effects, including liver problems (increased liver enzymes). 

What are the possible side effects (results of trials used to assess safety)? 

Table 3 and Table 4 summarize adverse reactions and laboratory abnormalities that occurred in the trial.

Table 3. Adverse Reactions Occurring in ≥10% of Patients Receiving ROMVIMZA With a Difference Between Arms of >5% Compared to Placebo Through Week 25

Adverse Reaction* ROMVIMZA,   N=83 Placebo, N=39
All Grades % Grade 3 or 4 % All Grades % Grade 3 or 4 %
Eye disorders

Periorbital edema1

 60 3.6 21 0

Lacrimation increased

 12 0 0 0

Dry eye1

 10 0 0 0
General disorders and administration site conditions

Fatigue1

 59 1.2 38 2.6

Peripheral edema1

 33 1.2 8 0

Face edema

 31 1.2 8 0
Skin and subcutaneous tissue disorders

Rash1

 47 3.6 5 0

Pruritus

 29 2.4 8 0
Vascular disorders

Hypertension

 17 4.8 10 2.6
Nervous system disorders

Neuropathy1

 12 1.2 2.6 0

Source: Adapted from FDA Review
* The severity of adverse reactions was assessed using CTCAE v5.0
1 Includes multiple related terms
Abbreviations: CTCAE, Common Terminology Criteria for Adverse Events

Table 4. Laboratory Abnormalities Worsening From Baseline in ≥10% of Patients Receiving ROMVIMZA With a Difference Between Arms of >5% Compared to Placebo Through Week 25

Laboratory Abnormality ROMVIMZA,   N=83 Placebo, N=38
All Grades % Grade 3 or 4 % All Grades % Grade 3 or 4 %
Chemistry

AST increased

 92 0 11 0

Cholesterol increased

 43 0 16 0

ALT increased

 24 0 16 0

Creatinine increased

 17 0 2.6 0

ALP increased

 14 0 8 0

Magnesium increased

 13 1.2 2.6 0

Calcium   decreased

 13 0 2.6 0
Hematology

Neutrophils decreased

 31 1.2 2.6 0

Leukocytes decreased

 29 0 8 0

Source: Adapted from FDA Review
Abbreviations: AST, aspartate aminotransferase; ALT, alanine aminotransferase; ALP, alkaline phosphatase

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

  • Sex: The occurrence of side effects was similar in males and females.
  • Race: The number of patients of races other than White was small; therefore, differences in the occurrence of side effects among races could not be determined.
  • Age: Most patients were adults younger than 65 years of age. The number of patients older than 65 years of age was limited; therefore, differences in the occurrence of side effects between patients younger and older than 65 years of age could not be determined.

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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