Casimersen

What is Casimersen?

• Casimersen AMONDYS 45 is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD).

What are the uses of this medicine?

• This medicine is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping.
• This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45.

How does this medicine work?

• Casimersen is designed to bind to exon 45 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 45 skipping.
• Exon 45 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 45 skipping.

Who Should Not Use this medicine ?

• This medicine have no usage limitation

Is this medicine FDA approved?

• It was approved for use in the United States in 2021.

How should this medicine be used?

• Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio (UPCR) should be measured before starting AMONDYS 45.
• Consider measurement of glomerular filtration rate prior to initiation of AMONDYS 45.
• Monitoring for kidney toxicity during treatment is recommended.
• Obtain the urine sample prior to infusion of AMONDYS 45 or at least 48 hours after infusion.

Recommended Dosage

• The recommended dosage of AMONDYS 45 is 30 milligrams per kilogram administered once weekly as a 35 to 60-minute intravenous infusion via an in-line 0.2 micron filter.
• If a dose of AMONDYS 45 is missed, it may be administered as soon as possible after the scheduled dose.

Administration

• Application of a topical anesthetic cream to the infusion site prior to administration of AMONDYS 45 may be considered.
• AMONDYS 45 is administered via intravenous infusion.
• Flush the intravenous access line with 0.9% Sodium Chloride Injection, USP, prior to and after infusion.
• Infuse the diluted AMONDYS 45 over 35 to 60 minutes via an in-line 0.2 micron filter.
• Do not mix other medication with AMONDYS 45 or infuse other medications concomitantly via the same intravenous access with AMONDYS 45.

What are the dosage forms and brand names of this medicine?

This medicine is available in fallowing doasage form: As Injection: 100 mg/2 mL in a single-dose vial

This medicine is available in fallowing brand namesː

• AMONDYS

What side effects can this medication cause?

The most common side effects of this medicine include:

• upper respiratory tract infection
• cough
• pyrexia
• headache
• arthralgia
• oropharyngeal pain

What special precautions should I follow?

• This medicine may cause kidney toxicity.
• Kidney function should be monitored; creatinine may not be a reliable measure of renal function in DMD patients.

What to do in case of emergency/overdose?

• In case of overdose, call the poison control helpline of your country. In the United States, call 1-800-222-1222.

• Overdose related information is also available online at poisonhelp.org/help.
• In the event that the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services. In the United States, call 911.

Can this medicine be used in pregnancy?

• There are no human or animal data available to assess the use of AMONDYS 45 during pregnancy.

Can this medicine be used in children?

• AMONDYS 45 is indicated for the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping, including pediatric patients.

What should I know about storage and disposal of this medication?

• Store AMONDYS 45 at 2°C to 8°C (36°F to 46°F). Do not freeze. Store in original carton until ready for use to protect from light.

• Dailymed label info on Casimersen
• FDA Casimersen

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