Sapropterin Amarox (sapropterin dihydrochloride) is an oraly administered Phenylalanine  Hydroxylase   activator (or PAH activator). Sapropterin dihydrochloride, the active pharmaceutical ingredient in Sapropterin Amarox, is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4). Sapropterin dihydrochloride is an off-white light yellow crystals or crystalline powder.

Sapropterin Amarox is a prescription medicine used to lower blood levels of  phenylalanine  (Phe),  in  people  with a certain type of Phenylketonuria (PKU). Sapropterin Amarox is used along with a Phe-restricted diet.

If you are alergic to sapropterin or any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions 

Talk to your doctor or pharmacist before taking Sapropterin Amarox, particularly:

·           If you have a fever

·           If you have liver or kidney problems

·         If you are alergic to sapropterin dihydrochloride or any of the ingredients  in  Sapropterin Amarox.  See the  list of ingredients in Sapropterin Amarox at the end of this leaflet.

·         If you have poor nutrition or have loss of appetite

·         If you are pregnant or plan to become pregnant.

Pregnancy Registry: There is a pregnancy registry for women who take Sapropterin Amarox during pregnancy. The purpose of this registry is to collect information about the health of you and your baby. Talk to your doctor about how you can take part in this registry.

·         If you are breastfeeding or plan to breastfeed. It is  not  known  if  Sapropterin Amarox passes  into  your  breast milk. Talk to your doctor about the best way to feed your baby if you take Sapropterin Amarox.

Other medicines and Sapropterin Amarox 

Tel your doctor or pharmacist about all the medicines  you  take,  including  prescription  and  over-the- counter  medicines,  vitamins,  herbal,  and  dietary  supplements.  Sapropterin Amarox and other  medicines   may interact with each other.

Especially tel your doctor if you take:

•                                            a medicine that contains levodopa

•                                            an antifolate medicine

•                                            avanafil (Stendra), sildenafil (Revatio, Viagra), tadalafil (Adcirca, Cialis), vardenafil (Staxyn, Levitra)

Tel your doctor if you are not sure if your medicine is one that is listed above.

Know the medicines you take. Keep a list of them to  show  your  doctor  and  pharmacist  when you get a new medicine.

Driving and using machines

Sapropterin Amarox is not expected to affect the ability to drive and use machines.

•                        Take Sapropterin Amarox exactly as your doctor tells you. Your doctor should tell you how much Sapropterin Amarox to take and when to take it.

•                        Your doctor may change your dose of Sapropterin Amarox depending on how you respond to treatment.

•                        Take  Sapropterin Amarox 1  time  each day with  a  meal.  It  is  best  to  take  Sapropterin Amarox  at  the same time each day.                                                                                                                    

•                      Sapropterin Amarox comes as a powder for oral solution.

o   Sapropterin Amarox powder for  oral solution  should  be dissolved  in  water  or apple  juice.    You may   also mix the powder for oral solution in a small amount of soft food, such as apple sauce or pudding before taking.

o           See the detailed “Instructions for Use” that comes with Sapropterin Amarox for information about the correct way to dissolve and take a dose of Sapropterin Amarox tablets or Sapropterin Amarox powder for oral solution.

•                        It is not possible to know if Sapropterin Amarox will work  for  you  until  you  start  taking  Sapropterin Amarox.  Your doctor wil check your blood Phe levels when you start taking Sapropterin Amarox to see  if  the  medicine  is working.

•                      During treatment with Sapropterin Amarox:

o  Any change you make to your diet may affect your blood Phe level. Follow your doctor’s  instructions carefully and do not make any changes  to  your  dietary  Phe  intake  without  first  talking with your doctor. Even if you take Sapropterin Amarox, if  your  Phe  blood  levels  are  not  well control ed, you can develop severe neurologic problems.

o    Your doctor should continue  to  monitor  your  blood  Phe  levels  often  during  your  treatment with Sapropterin Amarox, to make sure that your blood Phe levels are not too high or too low.

o  If you have a fever, or if you are sick, your blood  Phe  level may  go  up.  Tell  your  doctor as  soon as possible so they can change your dose  of  Sapropterin Amarox to  help  keep  your  blood  Phe levels in the desired range.

•                                  If you forget to take your dose of Sapropterin Amarox,  take  it  as soon as you remember  that  day.  Do  not  take 2 doses in a day.

•                                            If you take too much Sapropterin Amarox, call your doctor for advice.

Like all medicines, this medicine can cause side effects, although not everybody gets them.

Sapropterin Amarox can cause serious side effects, including: 

•         Severe allergic reactions. Stop taking  Sapropterin Amarox and  get  medical  help  right  away if you develop  any of these symptoms of a severe allergic reaction:

•                      wheezing or trouble

•                      feeling lightheaded or you breathing faint

•                      coughing

•                      flushing

•                      nausea

•                      rash

•                                           Inflammation of the lining of the stomach (gastritis). Gastritis  can happen with Sapropterin Amarox and may be severe. Call your doctor right away if you have any of these signs or symptoms:

•                  severe upper stomach-area (abdominal) discomfort or pain, nausea and vomiting

•                  blood in your vomit or stool

•                  black, tarry stools

•                                           Phe levels that are too low. Some children under the age of 7 who take high doses of Sapropterin Amarox each day may experience low Phe levels.

•                                            Too much or constant activity (hyperactivity) can happen with Sapropterin Amarox.

Tel your doctor if you have any signs of hyperactivity, including:

•  fidgeting or moving around too much

•  talking too much

The most common side effects of Sapropterin Amarox are:

•  headache

•  runny nose and nasal congestion

•  sore throat

•  diarrhoea

•   vomiting

•   cough

Tel your doctor if you have any side effect that bothers you or that does not go away.

These are not all the possible side effects of Sapropterin Amarox. For more information,  ask  your  doctor  or  pharmacist. Call your doctor for medical advice about side effects.

Reporting of side effects 

If any of the side effects gets serious, or if you notice any side effects not listed in this leaflet, please tell your doctor or pharmacist.

• Store below 30°C.

• Keep out of reach of children.

• Do not use this medicine after the expiry date which is stated on the  pack after EXP. The expiry date refers to the last day of the month.

• Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how

to throw away medicines you no longer use. These measures will help protect the environment.