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For full information on a Roche medicine, please see the relevant Summary of Product Characteristics.
Hemlibra is indicated for routine prophylaxis of bleeding episodes in patients with haemophilia A (congenital factor VIII deficiency):
- with factor VIII inhibitors
- without factor VIII inhibitors who have:
- severe disease (FVIII < 1%)
- moderate disease (FVIII ≥ 1% and ≤ 5%) with severe bleeding phenotype.
Hemlibra can be used in all age groups.
Clinical use:
The indication is granted market authorization based on objective response rate (ORR) demonstrated in a single-arm Phase II trial. Overall survival (OS) benefit in a single-arm trial cannot be confirmed.
In the pivotal trial, the majority of the clinical responses occurred within 16 weeks. Benefit of continued treatment should be regularly assessed, with the optimal duration of therapy varying for each individual patient.
Distribution Restrictions: ERIVEDGE is only available through a controlled distribution program called the ERIVEDGE Pregnancy Prevention Program (EPPP). For more information please contact the EPPP at 1-888-748-8926 or log onto rocheproplus.ca/en/products-resources/erivedge.html.
Geriatrics (≥65 years of age): Elderly patients should be treated with caution and monitored for adverse events.
Pediatrics (<18 years of age): The safety and efficacy of ERIVEDGE in pediatric patients has not been established. Irreversible premature fusion of the epiphyses and precocious puberty have been reported in pediatric patients exposed to ERIVEDGE. Premature fusion can progress after discontinuation of treatment. Due to safety concerns ERIVEDGE is contraindicated in children and adolescents aged below 18 years.
Contraindications:
Female patients who are pregnant or at risk of becoming pregnant
Breastfeeding female patients
Female patients of childbearing potential (FCBP) and male patients who do not comply with the ERIVEDGE Pregnancy Prevention Program
Children and adolescents aged less than 18 years of age
Patients who are hypersensitive to vismodegib or to any ingredient in the formulation
Most serious warnings and precautions:
Embryo-fetal death or severe birth defects: Can cause severe malformations, including craniofacial anomalies, midline defects and limb defects when administered to a female who is pregnant. Must not be used during pregnancy.
Effects on post-natal development: Irreversible premature fusion of the epiphyses (EPF) and precocious puberty have been reported in pediatric patients exposed to ERIVEDGE. In some cases of EPF, fusion progressed after drug discontinuation.
Renal impairment: The safety and efficacy of ERIVEDGE in patients with severe renal impairment have not been studied. No dedicated clinical studies have been conducted to evaluate the effect of mild, moderate and severe renal impairment on the pharmacokinetics of vismodegib.
Hepatic impairment: ERIVEDGE is not recommended for use in patients with severe hepatic impairment since limited data are available in these patients. ERIVEDGE should be used with caution in patients with mild and moderate hepatic impairment.
Other relevant warnings and precautions:
Effects on post-natal development
Blood donation: Patients must not donate blood while on treatment and for 24 months after discontinuation
Patients with advanced BCC (aBCC) have an increased risk of developing cutaneous squamous cell carcinoma (cuSCC). Cases of cuSCC have been reported in aBCC patients treated with ERIVEDGE. All patients should be monitored routinely while taking ERIVEDGE
Cardiovascular-related events
Decreased appetite, decreased weight and dehydration
Electrolyte abnormalities
Hepatotoxicity
Patients with a history of pancreatitis or gallbladder disease
Gastrointestinal-related events
Anaemia and lymphopenia
Grade 1 hypersensitivity
Arthralgia, back pain, muscle spasms, fractures, and elevated creatine phospohokinase (CPK) measurements reported
Syncope, dysgeusia and ageusia
Psychiatric disorders
Renal disorders and cases of renal failure have been observed in patients treated with ERIVEDGE
Amenorrhea has been observed in clinical trials in 30% of FCBP (Female of Childbearing Potential). Potential to impair fertility in patients
Blood work monitoring
For more information:
Please consult the Product Monograph for important information relating to warnings and precautions, adverse reactions, drug interactions, and dosing information that has not been discussed in this piece.
The Product Monograph is also available by calling us at 1-888-762-4388.
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Professor Steven Pipe discusses clinical trial data informing Hemlibra's safety and efficacy. The comprehensive HAVEN programme covered various haemophilia types, ages, and dosing, with prospective and long-term outcomes. This robust development, supported by real-world data, increases confidence in Hemlibra's clinical use.
Professor Steven Pipe describes their centre's shift to early Hemlibra prophylaxis due to IV access challenges with factor VIII. Families readily adopted it for its low burden and expected haemostatic benefits, supported by clinical trial data, including HAVEN 75, reinforcing their confidence in this approach for infants.
Professor Steven Pipe highlights that early Hemlibra prophylaxis fills a treatment gap, enabling haemostatic protection from birth. HAVEN 75 data showed no treated spontaneous bleeds in infants over the course of the study. Their clinic's experience over five years indicates reduced factor VIII exposure and no new inhibitors in early-treated patients, potentially altering inhibitor development history.
Professor Steven Pipe summarises the HAVEN 75 study, enrolling 55 infants treated with Hemlibra. Results showed a significant reduction in bleed rates, high efficacy levels maintained, no anti-drug antibodies, and a safety profile consistent with what has been seen in the Hemlibra clinical trials. The study provides strong evidence supporting Hemlibra's effectiveness and safety in treating young infants with haemophilia A.
Professor Steven Pipe introduces the HAVEN 75 study, evaluating Hemlibra prophylaxis efficacy and safety in infants with severe haemophilia A without inhibitors. He highlights the challenge of early factor VIII prophylaxis due to IV access issues, often delaying treatment until around one year, leaving a critical period with bleeding risks. This study addresses the data gap for this age group.
References:
- Mahlangu J et al. N Engl J Med 2018;379:811–22.
- Oldenburg J et al. N Engl J Med 2017;377:809–18.
- Callaghan M et al. Blood 2021;137:2231–42.
- Kempton C et al. Haemophilia 2021;27:221–28.
- Pipe SW et al. Blood 2024;143:1355–64.
Reporting suspected adverse reactions after authorisation of the medicinal product is important. It allows continued monitoring of the benefit/risk balance of the medicinal product. Adverse events should be reported. Reporting forms and information can be found at www.mhra.gov.uk/yellowcard. You can also report via the free Yellow Card app available from the Apple App Store or Google Play Store. Adverse events should also be reported to Roche Products Ltd. Please contact Roche Drug Safety Centre by emailing welwyn.uk_dsc@roche.com or calling +44 (0)1707 367554.
Adverse reactions should be reported by brand name and batch number.