Adverse reactions : תופעות לוואי

4.8    Undesirable effects
Summary of the safety profile

The most serious adverse reactions in patients in clinical studies were hypersensitivity reactions (2.1%).

The most common adverse reactions were infusion-related reactions (39.4%). The most commonly observed symptoms of infusion-related reactions were: headache, dizziness, hypotension, hypertension, nausea, fatigue/asthenia, and pyrexia/body temperature increased (see section 4.4 for further information). The only adverse reaction leading to discontinuation of treatment was an infusion-related reaction.

Tabulated list of adverse reactions

Adverse reactions reported in patients with type 1 Gaucher disease are listed in Table 1. Information is presented by system organ class and frequency according to MedDRA convention. Frequency is defined as very common (1/10), common (1/100 to <1/10), and uncommon (≥1/1,000 to <1/100).
Within each frequency grouping, adverse reactions are presented in order of decreasing seriousness.

Table 1: Adverse reactions reported with VPRIV in patients with type 1 Gaucher disease 
System organ
Adverse reaction class

Very common                     Common                          Uncommon Immune system                                 hypersensitivity reactions disorders                                     (includes dermatitis allergic and anaphylactic*/anaphylactoid reactions)
Nervous system       headache, dizziness disorders
Eye disorders                                                                vision blurred* Cardiac disorders                              tachycardia
Respiratory,                                   dyspnoea* thoracic and mediastinal disorders
Vascular                                       hypertension, hypotension, disorders                                      flushing
Gastrointestinal      abdominal                nausea                        vomiting* disorders             pain/abdominal pain upper
Skin and                                       rash, urticaria, pruritus* subcutaneous tissue disorders
Musculoskeletal       bone pain, arthralgia,
and connective        back pain tissue disorders
General disorders     infusion-related         chest discomfort* and                   reaction,
administration        asthenia/fatigue,
site conditions       pyrexia/body temperature increased
Investigations                             activated partial thromboplastin time prolonged, neutralizing antibody positive
*Adverse reactions derived from post-marketing reports

Description of selected adverse reactions

Vomiting
In some cases vomiting can be serious and severe. Vomiting most often occurs during the infusion and up to 24 hours after the infusion.

Other special populations

Elderly population (≥65 years)
The safety profile of VPRIV in clinical studies involving patients aged 65 years and above was similar to that observed in other adult patients.

Paediatric population

The safety profile of VPRIV in clinical studies involving children and adolescents aged 4 to 17 years was similar to that observed in adult patients.

Reporting of suspected adverse reactions

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.
Any suspected adverse events should be reported to the Ministry of Health according to the National Regulation by using an online form https://sideeffects.health.gov.il/