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Drug Trial Snapshot: PEPAXTO

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 PEPAXTO 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).

PEPAXTO (melphalan flufenamide)
(Peh-PAX-toe)
Oncopeptides AB

Approval date: February 26, 2021


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

PEPAXTO is a prescription medicine used in combination with the medicine dexamethasone to treat adults with multiple myeloma who did not respond to or stopped responding to at least four prior medicines including at least one proteasome inhibitor, one immunomodulatory agent and one CD38-directed antibody.

How is this drug used?

PEPAXTO is given by a healthcare provider into the vein through an intravenous line (IV). PEPAXTO is usually given 1 time every 28 days.

What are the benefits of this drug?

In the trial, 23.7% of patients responded to treatment with PEPAXTO. Responses lasted between 3.2 and 7.6 months (median 4.2 months).

PEPAXTO 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.

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

  • Sex: PEPAXTO appeared to work similarly in men and women.
  • Race: The number of patients of races other than White was small; therefore, differences in how PEPAXTO worked among races could not be determined.
  • Age: No difference in response to PEPAXTO was detected across age groups. The observed differences in overall response rate were not greater than one could expect given the variability of patients and the number of patients in the trial.

What are the possible side effects?

The most common side effects include low blood cell counts, fatigue, nausea, diarrhea, fever, and cold-like symptoms (respiratory tract infection).

The most common laboratory abnormalities were low blood cell counts, including:

  • low platelet counts which may cause bleeding or bruising under the skin,
  • low red blood cell counts which may cause a sense of weakness or tiring easily, paleness or feeling short of breath, and
  • low white blood cell counts which can increase the risk of infections.

Secondary cancers, such as myelodysplastic syndrome or acute leukemia have been reported. In addition, taking PEPAXTO can cause fetal harm and breastfeeding is not advised.

Were there any differences in side effects of the clinical trials among sex, race, and age?

  • Sex: The occurrence of side effects appeared to be similar in men and women.
  • 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: The occurrence of sides effects appears to be similar across the age groups.

WHO WAS IN THE CLINICAL TRIALS?

Who participated in the clinical trials?

The FDA approved PEPAXTO based on evidence from a clinical trial of 157 adult patients with multiple myeloma. The trial was conducted at 17 sites in 4 countries in Spain, France, Italy and the US.

Figure 1 summarizes how many men and women were enrolled in the clinical trial used to evaluate the safety of PEPAXTO.

Figure 1: Baseline Demographics by Sex (Safety Population)

Figure 1 is a pie chart summarizing how many participants by sex were evaluated for safety in the HORIZON clinical trial.  Of the 157 participants, 89 (57%) were male and 68 (43%) were female.

Source: FDA Review

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

Figure 2: Baseline Demographics by Race (Safety Population)

Figure 2 is a pie chart summarizing how many participants by race were evaluated for safety in the HORIZON clinical trial.  Of the 157 participants, 135 (86%) were White, 11 (7%) Black or African American, and Other races accounted for 4 (3%) of volunteers; race was Unknown for 7 (4%) participants.

Source: FDA Review

The figure below summarizes how many patients by age were in the trial used to evaluate the side effects of PEPAXTO.

Figure 3: Baseline Demographics by Age (Safety and Efficacy Population)

Figure 3 is a pie chart summarizing how many participants by age were evaluated for safety in the HORIZON clinical trial.  Of the 157 participants, 78 (50%) were <65 years, 54 (34%) were between 65 to <75 years, and 25 (16%) were ≥75 years.

Source: FDA Review

How were the trials designed?

PEPAXTO was evaluated in 1 single-arm, open-label clinical trial of 157 adult patients with relapsed refractory multiple myeloma who were treated with at least 2 prior lines of therapy. The efficacy was measured by overall response rate.

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 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.

PRESCRIBING INFORMATION

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