Document Details

Generated Narrative: Bundle TEST PURPOSES ONLY - nulibry

Document Subject

Generated Narrative: MedicinalProductDefinition mpeaeff7283270f9b7e51abc26d498e673

identifier: http://ema.europa.eu/identifier/EU/1/22/1684/001

type: Medicinal Product

domain: Human use

status: active

legalStatusOfSupply: Medicinal product subject to medical prescription

name

productName: NULIBRY 9.5 mg powder for solution for injection.

type: Full name

part

part: nan

type: Invented name part

part

part: nan

type: Scientific name part

part

part: nan

type: Strength part

part

part: nan

type: Pharmaceutical dose form part

Usages

-	Country	Jurisdiction	Language
*	EU	EU	en

Document Content

Generated Narrative: Composition composition-en-eaeff7283270f9b7e51abc26d498e673

identifier: http://ema.europa.eu/identifier/EU/1/22/1684/001

status: Final

type: Package Leaflet

category: Raw

date: 2022-02-16 13:28:17+0000

author: Organization ACME industry

title: TEST PURPOSES ONLY - nulibry

Attesters

What is in this leaflet

1. What NULIBRY is and what it is used for
2. What you need to know before you use NULIBRY
3. How to use NULIBRY
4. Possible side effects
5. How to store NULIBRY
6. Contents of the pack and other information

What NULIBRY is NULIBRY contains the active substance fosdenopterin.
NULIBRY is given to people with the genetic disease molybdenum cofactor deficiency (MoCD) Type A. It is given to people when doctors suspect that they might have MoCD Type A. It needs to be continued for life if MoCD Type A is confirmed by genetic testing.

What molybdenum cofactor deficiency (MoCD) Type A is MoCD Type A is a rare inborn error of the natural chemical processes needed for your body to work (metabolism). Signs of this genetic disease usually appear shortly after birth and include difficulty feeding and seizures. Other signs are a decreased awareness or reaction to the environment, an increase in startle reactions to a sudden event, and weak or stiff muscles.

MoCD type A results from an error in the gene called MOCS1. This stops the body from making an essential substance called cyclic pyranopterin monophosphate. When this substance is missing, certain compounds (sulphites) formed in the body cannot be broken down. These compounds are toxic to the brain and can negatively affect or delay the development of a child.

How NULIBRY works NULIBRY provides the missing substance that you or your child s body needs to break down the harmful sulphite compounds.

Do not use NULIBRY

• if you or your child is allergic to fosdenopterin or any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions

Talk to your doctor or nurse before using NULIBRY.

Patients who use NULIBRY may become sensitive to direct sunlight and ultraviolet light. During treatment with fosdenopterin, patients should avoid exposure to sunlight, and wear sunscreen, protective clothing, and sunglasses when exposed to the sun. Tell your doctor immediately if you or your child develops a rash, redness or blisters on sun-exposed areas of the skin, or if you or your child experience a burning sensation of the skin.

Due to the use of sunscreens and sun protective clothing your doctor may prescribe additional vitamin D as necessary.

Other medicines and NULIBRY It is unlikely that NULIBRY has an influence on or is influenced by other medicines. However, tell your doctor if your child is taking, has recently taken, or might take any other medicines.

NULIBRY contains sodium This medicine contains less than 1 mmol sodium (23 mg) per vial, that is to say essentially sodium-free .

How NULIBRY is given NULIBRY is injected into a vein through a catheter.

A doctor who is experienced in the management of inborn errors of metabolism will start and supervise the treatment with NULIBRY.

NULIBRY can be given at home. Before you do this for the first time, your doctor or nurse will train you in how to prepare the medicine and give you or your child a dose of NULIBRY.

Always use this medicine exactly as your doctor or nurse has instructed. Check with your doctor if you or your child are not sure about how to use NULIBRY.

How much to use The dose depends on your child s age and body weight. You will need to give the dose once each day. Your doctor will work out the dose you need to give.

If you use more NULIBRY than you should If you think you or your child may have been given more NULIBRY than prescribed, tell your doctor immediately.

If you forgot to give a dose of NULIBRY. If a dose of NULIBRY is missed, give the missed dose as soon as possible. Wait at least 6 hours before you give the next dose.

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

Tell your doctor or nurse immediately if any of the side effects occur, or if you notice any side effects not listed in this leaflet.

The following side effects are very common and are related to the injection device (catheter) and not to the medicine. These may affect more than 1 in 10 people:

• Catheter-related problems, such as pain, discharge, redness, or inflammation

Complications related to catheter You or your child will have an injection device (catheter-type device). This is used to inject medicines into you or your child s blood. You or your child may develop complications related to the catheter. Please follow the instructions from your doctor or nurse on how to care for this device before and after giving a dose of NULIBRY.

Reporting of side effects If you notice any side effects, talk to your doctor or nurse. This includes any possible side effects not listed in this leaflet. You can also report side effects directly the national reporting system listed in Appendix V. By reporting side effects you can help provide more information on the safety of this medicine.

Keep this medicine out of the sight and reach of children.

Do not use this medicine after the expiry date which is stated on the carton and vial label after EXP. The expiry date refers to the last day of that month.

Unopened vial Store in a freezer at -25 C to -10 C.
Keep the vial in the outer carton in order to protect from light.

Storage of the reconstituted (mixed) NULIBRY Reconstituted NULIBRY may be stored at room temperature (15 C-25 C) or refrigerated (2 C-8 C) for up to 4 hours including the time needed to give NULIBRY.

If reconstituted NULIBRY is refrigerated, allow it to come to room temperature (by rolling each vial gently between your hands for 3 to 5 minutes (do not shake) or by leaving it at room temperature for approximately 30 minutes) before giving NULIBRY.

• Do not heat.
• Do not freeze NULIBRY after reconstitution.
• Do not shake.

The reconstituted solution must be a clear and colourless to pale yellow solution. Do not use this medicine if you notice any particles or if the solution is discoloured.

Do not throw away any medicines or waste material, including materials used for reconstitution and administration via wastewater or household waste. Ask your pharmacist how to throw away medicines you no longer use. These measures will help protect the environment.

What NULIBRY contains

• The active substance is fosdenopterin 9.5 mg. Each vial contains fosdenopterin hydrobromide dihydrate equivalent to 9.5 mg fosdenopterin.
• The other ingredients are: ascorbic acid (E300), mannitol (E421), sucrose, hydrochloric acid (E507), sodium hydroxide (E524) (see section 2 Nulibry contains sodium ).

What NULIBRY looks like and contents of the pack NULIBRY is a white to pale yellow powder for solution for injection (powder for injection).

Each pack contains one vial.

Marketing Authorisation Holder TMC Pharma (EU) Ltd 7a Durands Court, 45 Parnell Street, Waterford X91 PIreland

Manufacturer Millmount Healthcare Limited Block 7, City North Business Campus Stamullen, Co. Meath, K32 YDIreland

This leaflet was last revised in

This medicine has been authorised under exceptional circumstances . This means that because of the rarity of this disease it has been impossible to get complete information on this medicine. The European Medicines Agency will review any new information on this medicine every year and this leaflet will be updated as necessary.

Other sources of information

Detailed information on this medicine is available on the European Medicines Agency web site: