Expanding options for Duchenne in patients amenable to exon 45 skipping.

Meet Levi, age 10. Amenable to exon 45 skipping

Meet Colin, age 11. Amenable to exon 45 skipping

AMONDYS 45 is an FDA-approved Duchenne muscular dystrophy treatment for patients who have a confirmed genetic mutation in the dystrophin gene that can be treated by skipping exon 45. AMONDYS 45 helps the body make a shorter form of the 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.

APPROVED UNDER ACCELERATED APPROVAL

Accelerated approval allows for drugs to be approved based on a marker that is considered reasonably likely to predict a clinical benefit. AMONDYS 45 treatment increased the marker, dystrophin, in skeletal muscle.

Accelerated approval also requires that, after approval, additional adequate and well-controlled studies called confirmatory trials are conducted to verify and describe the clinical benefit.

Discover AMONDYS 45

Who Can Take It?

Learn the importance of understanding your genetic mutation and what it means to be amenable to treatment with AMONDYS 45.

Learn About Amenability

Starting Treatment

Starting treatment with AMONDYS 45? Let us take you through the process so you know what to expect.

Explore the Process

Getting Support

With the SareptAssist patient support program, we’ll help you navigate the process of starting therapy with AMONDYS 45.

How We Can Help

“SareptAssist has done an outstanding job of putting our minds at ease throughout this entire journey. They answered any questions we had while providing useful resources.”

Jake, Colin's father

Ongoing support and information from Sarepta.

Receive future tips and support about treatment with AMONDYS 45.

Stay informed about SareptAssist services.

Find out about patient resources that are available.

Get updates about news and research on Duchenne from Sarepta.

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).