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To determine whether GENGLYCOS is right for your patient, the following tests and assessments should be performed prior to treatment1
Testing for pre-existing antibodies to AAV8. Do not administer GENGLYCOS to patients who test positive for AAV8 antibodies
Liver function tests (ie, ALT, AST) and evaluation of liver-related medical history
Patient’s ability to receive corticosteroids and other immunomodulatory therapy
Timing of other medications before and after GENGLYCOS infusion1
1 month before infusion:
Finish administering any vaccinations
Vaccines should be avoided 1 month prior to GENGLYCOS treatment.
30 to 60 minutes before infusion:
Administer premedication
To help reduce the risk of infusion-related reactions, premedicate with acetaminophen and non-sedating antihistamines 30 to 60 minutes prior to GENGLYCOS infusion.
2 weeks post-infusion:
Start oral corticosteroids
To mitigate hepatic reactions, administer a course of prophylactic oral corticosteroids (prednisone or equivalent) starting 2 weeks after GENGLYCOS treatment, and continue for a minimum of 8 weeks.
The use or addition of other immunomodulatory therapy may be considered.
Patient Monitoring1
Infusion-related reactions
- Monitor patients during and after completion of GENGLYCOS infusion as clinically indicated
- In the event of an infusion-related reaction, pause the infusion and provide supportive care as needed. If the infusion is paused, restart at half the prior rate after the infusion-related reaction has resolved
- If the infusion needs to be paused and restarted, GENGLYCOS infusion should be completed within 24 hours of dose preparation
Liver function tests and imaging
-
Perform regular liver function tests to monitor for transaminase elevations, which may
indicate immune-mediated hepatic reactions
- Monitor ALT and AST levels after GENGLYCOS administration weekly until 2 weeks after completion of the corticosteroid taper period, then every 2 to 4 weeks until month 6, and as clinically indicated thereafter
- If clinically significant transaminase elevations occur, the corticosteroid regimen may need to be adjusted, as clinically indicated
- Perform annual liver imaging in patients, in particular patients with additional risk factors for hepatocellular carcinoma (eg, hepatitis B or C, non-alcoholic fatty liver disease, chronic alcohol consumption, non-alcoholic steatohepatitis, advanced age)
Nutritional intake, glycemic control, and metabolic control
-
Following GENGLYCOS administration, monitor glycemic and metabolic control to assess
the need for nutritional modifications, as endogenous glucose production is increased
- Monitor glucose levels regularly (eg, self-monitored blood glucose or continuous glucose monitoring) and maintain normal blood glucose levels, avoiding hypoglycemia, hyperglycemia, and hyperglycemia-associated insulin spikes that may precipitate hypoglycemic events
- Monitor available parameters of metabolic control as clinically indicated, such as lactate, triglycerides, and uric acid
-
Modify cornstarch and diet based on individual evaluation of clinical status,
including nutritional assessment, glycemic control, and metabolic control
- As cornstarch is reduced, adjustments to diet are required to meet individual energy and nutrient goals
- Monitor the amounts and spacing of cornstarch and dietary carbohydrates to achieve glycemic, metabolic, and nutritional stability
- Continue close monitoring of glucose and metabolic control to adequately modify cornstarch and diet as needed
GENGLYCOS treatment brochure
Download this resource to learn more about GENGLYCOS dosing, administration, and monitoring.
Download
Enrollment Form
To initiate treatment with GENGLYCOS, complete the Enrollment Form with your patient.
Patient Enrollment FormINDICATION AND IMPORTANT SAFETY INFORMATION
Indication
GENGLYCOS (pariglasgene brecaparvovec-xxxx) is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of adult and pediatric patients 8 years and older with glycogen storage disease type Ia (GSDIa) without detectable antibodies to adeno-associated virus serotype 8 (AAV8).
Important Safety Information
GENGLYCOS is contraindicated in patients with known severe hepatic fibrosis or cirrhosis.
Infusion-Related Reactions
- Infusion-related reactions, including hypersensitivity reactions, occurred during GENGLYCOS administration.
- Premedicate with acetaminophen and non-sedating antihistamines and administer GENGLYCOS according to recommended infusion rates. Monitor patients during and after completion of GENGLYCOS infusion as clinically indicated. In the event of an infusion-related reaction, pause the infusion and provide supportive care as needed. If the infusion is paused, restart at half the prior rate after the infusion-related reaction has resolved.
Hepatic Reactions
- Predominantly mild to moderate, transient liver transaminase elevations were observed in clinical studies, most often in the first 15 weeks following GENGLYCOS administration.
- Prior to GENGLYCOS infusion, evaluate liver-related medical history and assess liver function by clinical examination and laboratory testing. Administer corticosteroids to all patients after GENGLYCOS infusion in order to mitigate hepatic reactions. Elevated transaminases may require adjustment of the corticosteroid treatment regimen, including increased dose or prolongation of the corticosteroid taper.
- Monitor transaminase levels for the first 6 months after GENGLYCOS administration. Continue to monitor transaminases in all patients who develop transaminase elevations, until transaminases return to baseline or as clinically indicated. If transaminase elevations are observed, consider all potential causes in the assessment of management for transaminase elevations, including infectious causes, alcohol, drugs, concomitant medication use, including herbal products and nutritional supplements, and worsening of underlying GSDIa, in addition to immune responses to GENGLYCOS.
- The effect of alcohol consumption on the magnitude and duration of the therapeutic effect has not been studied with GENGLYCOS treatment. Patients should be counseled on the potential risks of alcohol consumption following GENGLYCOS infusion.
Malignancy
- The integration of liver-targeting AAV vector DNA into the genome may carry the theoretical risk of tumorigenicity and may also insert into the DNA of other human body cells.
- GSDIa is associated with hepatic adenoma formation which carries a risk of transformation to hepatocellular carcinoma. Therefore, monitor patients with annual liver imaging, in particular patients with additional or other risk factors for hepatocellular carcinoma (e.g., hepatitis B or C, non-alcoholic fatty liver disease, chronic alcohol consumption, non-alcoholic steatohepatitis, advanced age). In the event that a malignancy occurs, contact Ultragenyx Pharmaceutical Inc. at 1-888-756-8657.
Adverse Reactions
- The most frequent adverse reactions in clinical studies were transaminases increased (85%) and nausea (29%).
Contraception
- Women of childbearing potential should use effective contraception for at least 48 weeks after administration of GENGLYCOS.
- For 6 months after administration of GENGLYCOS, men must not donate semen, and men of reproductive potential and their female partners must prevent or postpone pregnancy using an effective form of contraception.
Monitoring Nutritional Intake, Glycemic Control, and Metabolic Control
- Inform patients/caregivers of the importance of continued follow-up with their healthcare provider to monitor and adjust cornstarch and diet after receiving GENGLYCOS. At home, blood glucose should be monitored to avoid hypoglycemia and maintain normal blood glucose levels. Blood tests and dietary assessments may be needed to evaluate the appropriateness of nutritional modifications (cornstarch and diet) and to ensure glycemic and metabolic stability.
Vector Shedding
- As temporary vector shedding of GENGLYCOS occurs primarily via bodily waste, patients and caregivers should be instructed to follow appropriate hygiene precautions when handling patient waste for 3 months following GENGLYCOS infusion.
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 Ultragenyx Pharmaceutical Inc. at 1-888-756-8657.
Please see the full Prescribing Information for GENGLYCOS.
AAV8, adeno-associated virus serotype 8; ALT, alanine aminotransferase; AST, aspartate aminotransferase.
Reference
1. GENGLYCOS [Prescribing Information]. Novato, CA: Ultragenyx Pharmaceutical Inc; [Month Year].