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  5. Drug Trial Snapshot: AMONDYS 45
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Drug Trial Snapshot: AMONDYS 45

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 AMONDYS 45 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).

AMONDYS 45 (casimersen)
(ah-MAHN-dis)
Sarepta therapeutics, Inc.

Approval date: February 25, 2021


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

AMONDYS 45 is a drug for the treatment of a particular type of Duchenne muscular dystrophy (DMD). It is to be used only in patients who have a specific mutation of the dystrophin gene.

DMD is a rare disease that primarily affects boys. It is caused by low levels of a muscle protein called dystrophin. The lack of dystrophin causes progressive muscle weakness and premature death.

How is this drug used?

AMONDYS 45 is given by a health care professional once every week directly into the bloodstream through a needle in the vein. This is known as an intravenous, or IV infusion. It takes about 35-60 minutes to receive AMONDYS 45 infusion.

What are the benefits of this drug?

AMONDYS 45 increased levels of dystrophin in the muscles of treated patients. It is believed that this increase may predict clinical benefit in patients.

AMONDYS 45 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.

A trial is ongoing to assess whether there is a clinical benefit of AMONDYS 45.

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

The trial that looked at the benefit of AMONDYS 45 consisted of boys only who were of similar age and predominantly White. It was not possible to determine if there were any differences in how well the drug worked in sex, race, and age subgroups.

What are the possible side effects?

Kidney damage was observed in animal studies with AMONDYS 45. The most common side effects of AMONDYS 45 are upper respiratory infections, cough, fever, headache, joint pain, and throat pain.

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

The trials that looked at the side effects of AMONDYS 45 consisted of boys only, who were of similar age and predominantly White. It was not possible to determine if there were any differences in side effects in sex, race, and age subgroups.

WHO WAS IN THE CLINICAL TRIALS?

Who participated in the clinical trials?

The data for approval of AMONDYS 45 came from 43 male patients with Duchenne muscular dystrophy (DMD) in an ongoing clinical trial (Trial 1). The trial was conducted at 7 sites in 5 countries (United States, Canada, Germany, Spain, Czech Republic). Demographics of the trial population (called efficacy population) are presented in Table 3 under MORE INFO.

The ongoing clinical trial also provided data for the evaluation of safety in 57 male subjects receiving AMONDYS 45 and 31 receiving placebo for 96 weeks (called the safety population). Demographics from the safety population are presented below and in Table 3 under MORE INFO.

Figure 1 summarizes the percentage of patients by sex.

Figure 1. Baseline Demographics by Sex (Safety Population)

Baseline Demographics by Sex (Safety Population)

Figure 1 is a pie chart summarizing the percentage of participants by sex in the population evaluated for safety in Trial 1.  All 88 participants (100%) were male.

Source: Adapted from FDA Review

Figure 2 summarizes the percentage of patients by race.

Figure 2. Baseline Demographics by Race (Safety Population)

Figure 2 is a pie chart summarizing the percentage of participants by race in the population evaluated for safety in Trial 1.  In total, safety was assessed for 78 White (89%), 5 Asian (6%) and 1 (1%) Native Hawaiian or Pacific Islander participants; race was reported as “Other” for 4 (4%) volunteers.

Source: Adapted from FDA Review

Figure 3 summarizes the percentage of patients by age.

Figure 3. Baseline Demographics by Age (Safety Population)

Figure 3 is a pie chart summarizing the percentage of participants by age in the population evaluated for safety in Trial 1.  In total, efficacy was assessed for 44 (50%) participants between 6 – 9 years of age, 31 (35%) participants between 9 – 12 years of age, and in 13 (15%) participants > 12 years of age.]

Source: Adapted from FDA Review

Figure 4 summarizes the percentage of patients by ethnicity.

Figure 4. Baseline Demographics by Ethnicity (Safety Population)

Figure 4 is a pie chart summarizing the percentage of participants by ethnicity in the population evaluated for safety in Trial 1.  In total, safety was assessed for 79 (90%) Hispanic or Latino participants and 5 (6%) not Hispanic or Latino participants; ethnicity was reported as “Unknown” for 4 (4%) volunteers.

Source: Adapted from FDA Review

How were the trials designed?

Trial 1 is an ongoing clinical trial which enrolled patients with a particular type of Duchenne muscular dystrophy, who were randomly assigned to receive either AMONDYS 45 or placebo once a week for 96 weeks. Neither the patients nor the healthcare providers knew which treatment was being given. The benefit was evaluated by measuring the level of dystrophin in muscle biopsies in 43 patients (Efficacy Population) before treatment and at week 48, in an interim analysis. In this ongoing trial, 88 patients (Safety Population) have received AMONDYS 45 or placebo for up to 96 weeks.

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.

AMONDYS 45 Prescribing Information

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