Complete adverse effect profile including incidence rates and management
Important Safety Information
This is not a complete list of all possible side effects. Contact your healthcare provider if you experience any unexpected symptoms. For serious or life-threatening side effects, seek emergency medical attention immediately.
6 ADVERSE REACTIONS Most common adverse reactions are (≥10%): abdominal pain, diarrhea, vomiting, and nausea.
( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Ultragenyx Pharmaceutical Inc.
at 1-888-756-8657 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
6.1 Clinical Trial Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.
The safety population included 79 patients with LC-FAOD exposed to DOJOLVI in two studies: one open-label 78-week study of DOJOLVI in 29 patients (Study 1) followed by an open-label extension study (Study 2).
Twenty-four patients from Study 1 continued into Study
Patients ranged from 4 months to 63 years of age and the population was 52% male.
Of the 79 patients, 87% were White, 5% were Black or African-American, 4% were Asian, and 4% other.
The daily dosage of DOJOLVI ranged between 12% and 41% DCI (which corresponds to 0.7 g/kg/day to 6.0 g/kg/day for pediatric patients and 0.5 g/kg/day to 1.3 g/kg/day for adult patients) for a mean duration of 23 months.
The most common adverse reactions to DOJOLVI reported in the pooled safety population of Study 1 and Study 2 were gastrointestinal (GI)-related, and included abdominal pain (abdominal discomfort, abdominal distension, abdominal pain, abdominal pain upper, GI pain) [60%], diarrhea [44%], vomiting [44%], and nausea [14%].
5 WARNINGS AND PRECAUTIONS Feeding Tube Dysfunction : Regularly monitor the feeding tube to ensure proper functioning and integrity.
( 5.1 ) Intestinal Malabsorption in Patients with Pancreatic Insufficiency : Low or absent pancreatic enzymes may reduce absorption of DOJOLVI.
Avoid administration of DOJOLVI in patients with pancreatic insufficiency.
( 5.2 ) 5.1 Feeding Tube Dysfunction Feeding tube performance and functionality can degrade over time depending on usage and environmental conditions.
In clinical trials, feeding tube dysfunction was reported in patients receiving triheptanoin.
Like all medications, Dojolvi can cause side effects. However, not everyone who takes this medication will experience them. Many side effects are dose-dependent and may improve as your body adjusts to the medication. Others may require dose adjustment or medical attention.
Contact your healthcare provider promptly if you experience:
Seek immediate emergency medical care if you experience signs of: