The European Medicines Agency (EMA) has recommended that pegvaliase (Palynziq) be licensed across the European Union (EU) for the treatment of phenylketonuria (PKU) in patients aged 16 years and older.
Pegvaliase is indicated for the treatment of patients who have inadequate blood phenylalanine control (blood phenylalanine levels greater than 600 µmol/L), despite prior management.
In clinical trials, the PEGylated recombinant phenylalanine ammonia lyase enzyme was effective in lowering serum phenylalanine levels, with the majority of patients receiving pegvaliase (≤600 µmol/L) after 18 months of treatment. The data also showed that psychiatric and cognitive symptoms improved with continued long-term treatment.
The most common side effects were arthralgia and injection site reactions. These are considered to be linked to the allergic reactions seen in all patients; most were mild to moderate but acute hypersensitivity reactions occurred in a small fraction of patients (5.6%). Therefore, the EMA has instructed the company to take specific measures to minimise this risk, including making available additional educational material for prescribers and patients.
The EMA opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation.
The only medicine currently authorised in the EU for phenylketonuria is sapropterin (Kuvan).