Peyona

Summary

EUROPEAN MEDICINES AGENCY

SCIENCE MEDICINES HEALTH

EMA/239231/2015

EMEA/H/C/001014

EPAR summary for the public

Peyona¹

caffeine citrate

This is a summary of the European public assessment report (EPAR) for Peyona. It explains how the Committee for Medicinal Products for Human Use (CHMP) assessed the medicine to reach its opinion in favour of granting a marketing authorisation and its recommendations on the conditions of use for Peyona.

What is Peyona?

Peyona is a solution for infusion (drip) into a vein that contains the active substance caffeine citrate (20 mg/ml). The solution can also be given by mouth.

What is Peyona used for?

Peyona is used to treat primary apnoea in premature newborns. Apnoea in newborns is the cessation of breathing for more than 20 seconds. Primary means that the apnoea has no obvious cause.

Because the number of patients with primary apnoea of premature newborns is low, the disease is considered 'rare', and Peyona was designated an 'orphan medicine' (a medicine used in rare diseases) on 17 February 2003.

The medicine can only be obtained with a prescription.

How is Peyona used?

Treatment with Peyona should only be started under the supervision of a doctor experienced in treating newborn babies requiring intensive care. Treatment with the medicine should only take place in an intensive care unit for newborns that is well equipped for monitoring the baby.

Treatment is started with a dose of 20 mg per kilogram bodyweight given once by slow infusion over 30 minutes. After 24 hours, a 'maintenance dose' of 5 mg per kg bodyweight may be given everyday ¹ Previously known as Nymusa.

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by slow infusion over 10 minutes or by mouth using a nasogastric tube (a tube through the nose to the stomach).

When judged necessary, the doctor may monitor the level of caffeine in the baby's blood and adjust the dose accordingly. The doctor will stop treatment with Peyona when the baby has five to seven consecutive days without apnoea attacks.

How does Peyona work?

Apnoea in premature babies is due to the baby's breathing centres in the brain not being fully developed. The active substance in Peyona, caffeine citrate, is a stimulant of the nervous system. Caffeine citrate is an 'antagonist' of adenosine, a substance that blocks the activity of some parts of the brain, including the part responsible for controlling breathing. Caffeine citrate works in apnoea by blocking the receptors that adenosine normally attaches to. This reduces the effect of adenosine, stimulating the brain to resume breathing.

How has Peyona been studied?

Because caffeine citrate has been used in premature babies for a long time, the company presented information from the scientific literature. In a published study involving 85 premature babies who had had several episodes of apnoea, caffeine citrate was compared with placebo (a dummy treatment). The main measure of effectiveness was based on the reduction by at least a half in the daily number of apnoea episodes over ten days.

A large published study compared caffeine citrate with placebo in treating 2,006 premature babies with apnoea. This study looked at how long the babies survived and whether or not they had neurological disabilities after 18 months.

In a published review of five studies, caffeine and theophylline (another stimulant) were compared with placebo in 192 premature babies with apnoea. The main measure of effectiveness in this review was the number of patients who did not have 'treatment failure'. A baby was considered to be in treatment failure if there was no halving of the number of apnoea episodes, if the baby required a machine to assist in breathing or if the baby did not survive.

What benefit has Peyona shown during the studies?

Caffeine citrate was more effective than placebo at treating apnoea in premature babies. In six out of ten days caffeine citrate was more effective than placebo in reducing the number of apnoea episodes by at least a half. In addition, more babies who were given caffeine citrate had at least eight days with no apnoea: 22% of babies given caffeine citrate compared with none of babies who were given placebo.

In the large published study, 46% of babies given placebo (431 out of 932) died or had neurological disabilities compared with 40% of babies given caffeine citrate (377 out of 937).

In the review of five studies, fewer babies treated with caffeine or theophylline had treatment failure compared with placebo.

What is the risk associated with Peyona?

The most common side effects with caffeine citrate (seen in between 1 and 10 patients in 100) are hyperglycaemia (high blood glucose levels), tachycardia (rapid heartbeat) and phlebitis (inflammation

PeyonaOF

EMA/239231/2015 of a vein) and inflammation at the site of infusion. For the full list of all side effects and restrictions with caffeine citrate, see the package leaflet.

Why has Peyona been approved?

The CHMP decided that Peyona's benefits are greater than its risks and recommended that it be given marketing authorisation.

What measures are being taken to ensure the safe and effective use of Peyona?

A risk management plan has been developed to ensure that Peyona is used as safely as possible.

Based on this plan, safety information has been included in the summary of product characteristics and the package leaflet for Peyona, including the appropriate precautions to be followed by healthcare professionals and patients.

In addition, the company that makes Peyona will agree with Member States on a card that will be displayed in intensive care units where the medicine will be used. The card will contain information on how Peyona should be administered, including doses, the monitoring of plasma caffeine levels and the side effects that may occur during treatment.

Other information about Peyona

The European Commission granted a marketing authorisation valid throughout the European Union for Nymusa on 02 July 2009. The name of the medicine was changed to Peyona on 24 November 2010.

The summary of opinion of the Committee for Orphan Medicinal Products for Peyona can be found on the Agency's website ema.europa.eu/Find medicine/Human medicines/Rare disease designation.

The full EPAR for Peyona can be found on the Agency's website ema.europa.eu/Find medicine/Human medicines/European Public Assessment Reports. For more information about treatment with Peyona, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.

This summary was last updated in 03-2015.

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