What Is AMONDYS 45

Meet Beck, age 13. Amenable to exon 45 skipping

An exon-skipping therapy used to treat Duchenne.

AMONDYS 45 is used to treat patients with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the dystrophin gene that can be treated by skipping exon 45.

This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45.

Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

Beck’s Journey with AMONDYS 45

Embark on Beck’s journey with AMONDYS 45 as he and his family share their treatment goals and experiences, how AMONDYS 45 fits into their lives, and the vital role SareptAssist plays in their journey.

How does it work?

AMONDYS 45 is an exon-skipping therapy that helps the body make a shorter form of the dystrophin protein.^a Find out more about AMONDYS 45 and the results from an ongoing clinical study.

^aBoys treated with AMONDYS 45 showed varying levels of increased dystrophin production after 48 weeks. In an ongoing clinical study, 27 boys (median age 9 years) receiving AMONDYS 45 had an average dystrophin level of 1.74% of normal, compared to 0.76% in 16 boys who received a placebo infusion.

Get the Details

A lack of dystrophin protein

Exon-skipping technologies strive to address the underlying issue with Duchenne—a lack of the protein dystrophin. Many people with Duchenne have a genetic mutation in which one or more exons in the dystrophin gene are missing. This causes errors in the instructions for making dystrophin, leaving the body unable to produce the protein. Exon skipping tells the body to hide an exon so the whole section can be skipped over and the remaining exons can fit together. AMONDYS 45 is designed to skip over an exon. AMONDYS 45 helped boys with DMD amenable to skipping exon 45 to make a shorter form of dystrophin protein.^a

^aBoys treated with AMONDYS 45 showed varying levels of increased dystrophin production after 48 weeks. In an ongoing clinical study, 27 boys (median age 9 years) receiving AMONDYS 45 had an average dystrophin level of 1.74% of normal, compared to 0.76% in 16 boys who received a placebo infusion.

See How It Works

Graphic of train cars representing exons in the dystrophin gene, with exon 43 and 46 highlighted

Informed decisions.

There are certain risks and side effects associated with AMONDYS 45. As with any medication, you should discuss risks and side effects with your child's doctor.

See Important Safety Information

Meet Levi, age 10. Amenable to exon 45 skipping

What is AMONDYS 45 (casimersen)?

AMONDYS 45 is used to treat patients with Duchenne muscular dystrophy (DMD) who have a confirmed mutation of the dystrophin gene that can be treated by skipping exon 45.

This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

IMPORTANT SAFETY INFORMATION

Contraindications: Do not receive AMONDYS 45 if you are allergic to casimersen or any of the ingredients in AMONDYS 45. Serious allergic reactions to casimersen have included anaphylaxis, which may include difficulty breathing, tightness in the chest, and angioedema which may include swelling of the mouth, face, lips, or tongue.

Hypersensitivity Reactions: Serious allergic reactions, including angioedema and anaphylaxis, have occurred in patients who were treated with AMONDYS 45. Patients should seek immediate medical care should they experience signs and symptoms of allergic reactions. Your doctor will institute appropriate medical treatment which may include slowing, interrupting, or discontinuing the AMONDYS 45 infusion. Your doctor will monitor you until the condition resolves.

Kidney Toxicity and Monitoring: Damage to the kidneys was seen in animals who received casimersen. Although damage to the kidneys was not seen in clinical studies with AMONDYS 45, potentially fatal kidney damage has occurred with other drugs that work in a similar way. Your doctor may recommend urine and blood testing before starting treatment followed by urine testing every month and a blood test every 3 months to monitor your kidneys.

Adverse Reactions: Side effects occurring in at least 20% of patients treated with AMONDYS 45 and at least 5% more frequently than in patients who received an inactive intravenous (IV) infusion were (AMONDYS 45, placebo): upper respiratory tract infection (65%, 55%), cough (33%, 26%), fever (33%, 23%), headache (32%, 19%), joint pain (21%, 10%), and pain in mouth and throat (21%, 7%).

Other side effects that occurred in at least 10% of patients treated with AMONDYS 45 and at least 5% more frequently than patients who received an inactive IV infusion were: ear pain, nausea, ear infection, pain after injury, and dizziness and light-headedness.

What do I do if I have side effects?

Ask your healthcare provider for advice about any side effects that concern you.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782).

The information provided here does not include all that is known about AMONDYS 45. To learn more, talk with your healthcare provider.

Before receiving this infusion, please see the full Prescribing Information for AMONDYS 45 (casimersen).