Risdiplam Side Effects

Medically reviewed by Philip Thornton, DipPharm. Last updated on Aug 8, 2024.

Applies to risdiplam: oral powder for solution.

Serious side effects of risdiplam

Along with its needed effects, risdiplam may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor immediately if any of the following side effects occur while taking risdiplam:

More common

• body aches or pain
• chest pain or tightness
• chills
• cough
• ear congestion
• fever
• headache
• loss of voice
• runny or stuffy nose
• sneezing
• sore throat
• trouble breathing
• unusual tiredness or weakness

Less common

• bladder pain
• bloody or cloudy urine
• difficult, burning, or painful urination
• frequent urge to urinate
• lower back or side pain

Other side effects of risdiplam

Some side effects of risdiplam may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects.

Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

• canker sores
• constipation
• diarrhea
• skin rash
• sores, ulcers, or white spots on the lips or tongue or inside the mouth
• vomiting

Less common

• difficulty in moving
• joint pain
• muscle pain or stiffness

For healthcare professionals

Applies to risdiplam: oral powder for reconstitution.

General

The most frequently reported side effects included fever, diarrhea, headache, and rash. Additionally, among patients with infantile-onset spinal muscular atrophy, upper respiratory tract infection, lower respiratory tract infection, constipation, vomiting, and cough were reported.^[Ref]

Dermatologic

• Very common (10% or more): Rash (up to 17%)
• Common (1% to 10%): Eczema
• Postmarketing reports: Cutaneous vasculitis

Rash included dermatitis, rash, erythema, maculo-papular rash, erythematous rash, popular rash, allergic dermatitis, acneiform dermatitis, and folliculitis.

Cutaneous vasculitis was identified during post-marketing experience.

Endocrine

• Common (1% to 10%): Cryptorchism

Gastrointestinal

• Very common (10% or more): Constipation (up to 25%), diarrhea (up to 17%), vomiting (up to 17%)
• Common (1% to 10%): Mouth and aphthous ulcers, gastroesophageal reflux disease, gastroenteritis, nausea, upper abdominal pain

Genitourinary

• Common (1% to 10%): Urinary tract infection

Urinary tract infection included urinary tract infection and cystitis.

Musculoskeletal

• Common (1% to 10%): Arthralgia, joint dislocation

Nervous system

• Very common (10% or more): Headache (20%)

Other

• Very common (10% or more): Pyrexia (up to 54%), teething (up to 13%)
• Common (1% to 10%): Ear infection

Pyrexia included fever and hyperpyrexia.

Respiratory

• Very common (10% or more): Upper respiratory tract infection (up to 82%), pneumonia (up to 40%), cough (12.9%), bronchitis (up to 11%)
• Common (1% to 10%): Respiratory failure, respiratory distress, acute respiratory failure, atelectasis
• Frequency not reported: Lower respiratory tract infection

Upper respiratory tract infections included nasopharyngitis, respiratory tract infection, rhinitis, influenza, pharyngitis, viral respiratory tract infection, upper respiratory tract infection (bacterial and viral), and COVID-19.

Lower respiratory tract infection included pneumonia and bronchitis.

Further information

Risdiplam side effects can vary depending on the individual. Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Some side effects may not be reported. You may report them to the FDA.

Medical Disclaimer