Adverse reactions : תופעות לוואי

4.8    Undesirable effects

Summary of the safety profile
Approximately 35% of the 579 patients aged 4 years and over who received treatment with sapropterin dihydrochloride (5 to 20 mg/kg/day) in the clinical trials for Kuvan experienced adverse reactions. The most commonly reported adverse reactions are headache and rhinorrhoea.
In a further clinical trial, approximately 30% of the 27 children aged below 4 years who received treatment with sapropterin dihydrochloride (10 or 20 mg/kg/day) experienced adverse reactions. The most commonly reported adverse reactions are “amino acid level decreased” (hypophenylalaninaemia), vomiting and rhinitis.

Tabulated list of adverse reactions

In the pivotal clinical trials and in the post-marketing experience for Kuvan, the following adverse reactions have been identified.

The following definitions apply to the frequency terminology used hereafter: 
Very common (1/10)
Common (1/100 to <1/10)
Uncommon (1/1,000 to <1/100)
Rare (1/10,000 to <1/1,000)
Very rare (<1/10,000)
Frequency not known (cannot be estimated from available data)

Within each frequency grouping, adverse reactions are presented in order of decreasing seriousness.

Immune system disorders
Not known:     Hypersensitivity reactions (including serious allergic reactions) and rash 
Metabolism and nutrition disorders:
Common:         Hypophenylalaninaemia

Nervous system disorders:
Very common:     Headache
Respiratory, thoracic and mediastinal disorders:
Very common: Rhinorrhoea
Common:           Pharyngolaryngeal pain, nasal congestion, cough

Gastrointestinal disorders:
Common:           Diarrhoea, vomiting, abdominal pain, dyspepsia, nausea Unknown:          Gastritis, oesophagitis

Paediatric population
Frequency, type, and severity of adverse reactions in children were essentially similar to those in adults.

Reporting of suspected adverse reactions
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/