What is Nymusa?
Nymusa is a solution for infusion (drip into a vein) containing the active ingredient caffeine citrate (20 mg / ml). The solution can also be taken orally.
What is Nymusa used for?
Nymusa is used in the treatment of primary apnea in premature infants. In the newborn, apnea is the cessation of breathing for over 20 seconds; "primary" means the fact that it has no apparent cause. The medicine can only be obtained with a prescription.
Because the number of premature infants with primary apnea is low, the disease is considered "rare" and Nymusa was designated an "orphan medicine" (a medicine used in rare diseases) on 17 February 2003.
How is Nymusa used?
Treatment with Nymusa should only be started under the supervision of a doctor experienced in treating newborns who need intensive care. Treatment with the medicine should only be conducted in a neonatal intensive care unit appropriately equipped for monitoring the baby.
The treatment starts at a dose of 20 mg per kilogram of body weight, to be administered at once by slow infusion lasting 30 minutes. After 24 hours it is possible to start the daily administration of a maintenance dose of 5 mg per kg of body weight, by slow infusion lasting 10 minutes or by mouth through a nasogastric tube (a tube that is inserted into the nose and reaches the stomach) .
If necessary, the doctor can monitor the blood level of caffeine in the small, possibly correcting the dose. The doctor stops treatment with Nymusa after five to seven consecutive days without apnea.
How does Nymusa work?
In premature babies apnea is due to incomplete development of the brain centers responsible for breathing. The active ingredient in Nymusa, 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 one that controls breathing. In the case of apnea, caffeine citrate works by blocking the receptors to which adenosine normally binds, thus reducing the effect of adenosine and stimulating the brain to reactivate breathing.
What studies have been carried out on Nymusa?
Since caffeine citrate has been used for a long time in premature infants, the company presented data from the scientific literature. In a published study of 85 premature babies with multiple apnea episodes, caffeine citrate was compared with a placebo (a dummy treatment). The main measure of effectiveness was based on the reduction to at least half of the daily number of apnea episodes during ten days.
A large-scale study published comparing caffeine citrate with a placebo in the treatment of 2006 apnea-born premature babies. The study examined the duration of child survival and the presence or absence of neurological handicaps after 18 months.
In a published review of five studies, caffeine and theophylline (another stimulant) were compared with placebo in 192 premature babies with apnea. The main efficacy parameter was the number of patients without "therapeutic failure", understood as failure to halve the apneic episodes, or need to resort to assisted breathing or the death of the child.
What benefit has Nymusa shown during the studies?
Caffeine citrate was more effective than placebo in the treatment of apnea in premature infants. In 6 days out of 10, caffeine citrate was more effective than placebo in reducing the number of apneic episodes by at least half. In addition, the number of newborns treated with caffeine citrate was greater and they spent at least eight days without apneic episodes, 22% of babies treated with caffeine citrate compared to no newborn treated with placebo.
In a large published study, 46% of placebo-treated infants (431 out of 932) died or suffered neurological handicaps, compared with 40% of babies treated with caffeine citrate (377 out of 937).
In the review of the five studies, fewer treatment failures emerged in children treated with caffeine or theophylline than in those treated with placebo.
What is the risk associated with Nymusa?
The most common side effects associated with caffeine citrate (ie seen in between 1 and 10 patients in 100) are phlebitis (inflammation of a vein) at the site of infusion and inflammation of the site. For the full list of all side effects reported with caffeine citrate, see the Package Leaflet.
Nymusa should not be used in infants who may be hypersensitive (allergic) to caffeine citrate or any of the other ingredients.
Why was Nymusa approved?
The Committee for Medicinal Products for Human Use (CHMP) decided that the benefits of Nymusa outweigh the risks in the treatment of primary apnea in premature infants, and recommended the granting of a marketing authorization for Nymusa.
What measures are being taken to ensure the safe use of Nymusa?
The company that produces Nymusa is committed to preparing, in agreement with the Member States, a card to be posted in the intensive care units where the drug will be used; this card will contain information on how Nymusa is administered, dosages, the need to monitor the levels of caffeine in the plasma and the side effects that may occur during
treatment.
More information on Nymusa:
On 02 July 2009, the European Commission issued a marketing authorization to Chiesi Farmaceutical SpA for Nymusa, valid throughout the European Union.
For a summary of the opinion of the Committee for Orphan Medicinal Products on Nymusa, click here.
For the full EPAR for Nymusa, click here.
Last update of this summary: 05-2009.
