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DOJOLVI (triheptanoin) is the first and only FDA-approved treatment for LC-FAOD.

Select a type to learn more

CPT I
(carnitine palmitoyltransferase I) deficiency2,3
Cause

Mutation in the CPT1A gene; prevents long-chain fatty acids from being transported into the cells’ mitochondria for breakdown

Estimated Incidence
1:750,000 to 1:2,000,000
CACT
(carnitine-acylcarnitine translocase) deficiency2,4,5
Cause

Mutation in the SLC25A20 gene; prevents long-chain fatty acids from being transported into the cells’ mitochondria for breakdown

Estimated Incidence
1:750,000 to 1:2,000,000
CPT II
(carnitine palmitoyltransferase II) deficiency2,3
Cause

Mutation in the CPT2 gene; prevents long-chain fatty acids from being transported into the cells’ mitochondria for breakdown

Estimated Incidence
1:750,000 to 1:2,000,000
VLCAD
(very long-chain acyl-CoA dehydrogenase) deficiency2,3
Cause

Mutation in the ACADVL gene; prevents long-chain fatty acids from being broken down via fatty acid beta-oxidation

Estimated Incidence
1:85,000
TFP
(trifunctional protein) deficiency2,3
Cause

Mutations in both the HADHA and HADHB genes; leads to defects in the entire TFP complex and prevents long-chain fatty acids from being broken down via fatty acid beta-oxidation

Estimated Incidence
1:750,000
LCHAD
(long-chain 3-hydroxyacyl-CoA dehydrogenase) deficiency2,3
Cause

Mutation in the HADHA gene; encodes for a subunit of TFP and prevents long-chain fatty acids from being broken down via fatty acid beta-oxidation

Estimated Incidence
1:250,000

LC-FAOD are a group of rare, often serious, and life-threatening autosomal recessive disorders that result from defective enzymes involved in the mitochondrial transport and catabolism of long-chain fatty acids (LCFAs). Each type of LC-FAOD is named for the specific enzyme that is affected.3,6-8

DOJOLVI IS A UNIQUE, ODD-CHAIN, MEDIUM-LENGTH FATTY ACID

DOJOLVI (triheptanoin) is a synthetic medium odd-chain (C7) triglyceride consisting of three odd-chain, 7-carbon-length fatty acids.

It is the first and only FDA-approved treatment for patients of all ages diagnosed with LC-FAOD.

DOJOLVI (triheptanoin) is a synthetic medium odd-chain (C7) triglyceride consisting of three odd-chain, 7-carbon-length fatty acids.

It is the first and only FDA-approved treatment for patients of all ages diagnosed with LC-FAOD.


dojolvi molecule

Each 7-carbon fatty acid (heptanoate) in DOJOLVI (triheptanoin) provides a source of calories and fatty acids to bypass the enzyme deficiencies in LC-FAOD for energy production and replacement.

Dosing calculator

CALCULATE DOJOLVI

Use the DOJOLVI Dosing Calculator to:

  • Determine appropriate dosing for each patient
  • Generate dosing reports and schedules for both you and your patients

Calculate DOJOLVI now

Prescribe DOJOLVI

Complete the Start Form with your patient and fax it to 14157237474.

For additional support, you may also call our UltraCare Guides at 1-888-756-8657.


Access DOJOLVI

UltraCare® Patient Services provides a suite of services to help patients and caregivers:

  • Gain access to DOJOLVI (triheptanoin)
  • Determine eligibility for financial and patient assistance programs
  • Utilize patient support program resources
  • Gain access to DOJOLVI (triheptanoin)
  • Determine eligibility for financial and patient assistance programs
  • Utilize patient support program resources

Visit UltraCare

Ultracare.com homepage on a laptop

References:

  1. DOJOLVI (triheptanoin) US Prescribing Information; November 2021.
  2. Lindner M, Hoffmann GF, Matern D. J Inherit Metab Dis. 2010;33(5):521-526.
  3. Vockley J. Am J Manag Care. 2020;26(suppl 7):S147-S154.
  4. Pennisi EM, Garibaldi M, Antonini G. J Clin Med. 2018;7(12):E472.
  5. Vitoria I, Martín-Hernández E, Peña-Quintana L, et al. JIMD Rep. 2015;20:11-20.
  6. Knottnerus SJG, Bleeker JC, Wüst RCI, et al. Rev Endocr Metab Disord. 2018;19(1):93-106.
  7. Wajner M, Amaral AU. Biosci Rep. 2015;36(1):e00281.
  8. Wanders RJ, Ruiter JP, IJLst L, Waterham HR, Houten SM. J Inherit Metab Dis. 2010;33(5):479-494.

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FOR US HEALTHCARE PROFESSIONALS

INDICATION

DOJOLVI is a medium-chain triglyceride indicated as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD).

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Feeding Tube Dysfunction

  • Feeding tube performance and functionality can degrade over time depending on usage and environmental conditions. In clinical trials, feeding tube feeding dysfunction was reported in patients receiving triheptanoin. The contribution of DOJOLVI cannot be ruled out. Do not administer DOJOLVI in feeding tubes manufactured of polyvinyl chloride (PVC). Regularly monitor the feeding tube to ensure proper functioning and integrity.

Intestinal Malabsorption in Patients with Pancreatic Insufficiency

  • Pancreatic enzymes hydrolyze triheptanoin and release heptanoate as medium-chain fatty acids in the small intestine. Low or absent pancreatic enzymes may result in reduced absorption of heptanoate subsequently leading to insufficient supplementation of medium-chain fatty acids. Avoid administration of DOJOLVI in patients with pancreatic insufficiency.

ADVERSE REACTIONS

Gastrointestinal (GI)

  • The most common GI-related adverse reactions reported in the pooled safety population of Studies 1 and 2 were abdominal pain (abdominal discomfort, abdominal pain, abdominal distension, abdominal pain upper, GI pain) [60%], diarrhea [44%], vomiting [44%], and nausea [14%].

DRUG INTERACTIONS

Pancreatic Lipase Inhibitors

  • Co-administration of triheptanoin with a pancreatic lipase inhibitor (e.g., orlistat) may reduce exposure to the triheptanoin metabolite, heptanoate, and reduce the clinical effect of triheptanoin. Avoid co-administration of DOJOLVI with pancreatic lipase inhibitors.

USE IN SPECIFIC POPULATIONS

Pregnancy and Lactation

  • There are no available data on triheptanoin use in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. Advise women to report pregnancies to Ultragenyx Pharmaceutical Inc. at 1-888-756-8657.
  • There are no data on the presence of triheptanoin or its metabolites in human or animal milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the clinical need for DOJOLVI and any potential adverse effect on the breastfed infant from DOJOLVI or from the underlying condition.

PATIENT COUNSELING INFORMATION

  • Advise the patient or caregiver to read the FDA-approved patient labeling, which includes information on the appropriate oral or feeding tube preparation, administration, and storage.
  • Advise the patient or caregiver to regularly inspect the feeding tube for proper functioning and integrity and report to the healthcare provider if any issues are identified.
  • Inform the patient or caregiver that pancreatic insufficiency may reduce clinical effect of DOJOLVI. Any known pancreatic insufficiency should be reported to the healthcare provider.

You may report side effects to Ultragenyx Pharmaceutical Inc. at 1-888-756-8657 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.


Please see full Prescribing Information, including the Patient Information leaflet, for a complete discussion of the risks associated with DOJOLVI.

INDICATION

DOJOLVI is a medium-chain triglyceride indicated as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD).

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Feeding Tube Dysfunction

  • Feeding tube performance and functionality can degrade over time depending on usage and environmental conditions. In clinical trials, feeding tube feeding dysfunction was reported in patients receiving triheptanoin. The contribution of DOJOLVI cannot be ruled out. Do not administer DOJOLVI in feeding tubes manufactured of polyvinyl chloride (PVC). Regularly monitor the feeding tube to ensure proper functioning and integrity.

Intestinal Malabsorption in Patients with Pancreatic Insufficiency

  • Pancreatic enzymes hydrolyze triheptanoin and release heptanoate as medium-chain fatty acids in the small intestine. Low or absent pancreatic enzymes may result in reduced absorption of heptanoate subsequently leading to insufficient supplementation of medium-chain fatty acids. Avoid administration of DOJOLVI in patients with pancreatic insufficiency.

ADVERSE REACTIONS

Gastrointestinal (GI)

  • The most common GI-related adverse reactions reported in the pooled safety population of Studies 1 and 2 were abdominal pain (abdominal discomfort, abdominal pain, abdominal distension, abdominal pain upper, GI pain) [60%], diarrhea [44%], vomiting [44%], and nausea [14%].

DRUG INTERACTIONS

Pancreatic Lipase Inhibitors

  • Co-administration of triheptanoin with a pancreatic lipase inhibitor (e.g., orlistat) may reduce exposure to the triheptanoin metabolite, heptanoate, and reduce the clinical effect of triheptanoin. Avoid co-administration of DOJOLVI with pancreatic lipase inhibitors.

USE IN SPECIFIC POPULATIONS

Pregnancy and Lactation

  • There are no available data on triheptanoin use in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. Advise women to report pregnancies to Ultragenyx Pharmaceutical Inc. at 1-888-756-8657.
  • There are no data on the presence of triheptanoin or its metabolites in human or animal milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the clinical need for DOJOLVI and any potential adverse effect on the breastfed infant from DOJOLVI or from the underlying condition.

PATIENT COUNSELING INFORMATION

  • Advise the patient or caregiver to read the FDA-approved patient labeling, which includes information on the appropriate oral or feeding tube preparation, administration, and storage.
  • Advise the patient or caregiver to regularly inspect the feeding tube for proper functioning and integrity and report to the healthcare provider if any issues are identified.
  • Inform the patient or caregiver that pancreatic insufficiency may reduce clinical effect of DOJOLVI. Any known pancreatic insufficiency should be reported to the healthcare provider.

You may report side effects to Ultragenyx Pharmaceutical Inc. at 1-888-756-8657 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.


Please see full Prescribing Information, including the Patient Information leaflet, for a complete discussion of the risks associated with DOJOLVI.