Document Details

Generated Narrative: Bundle TEST PURPOSES ONLY - upstaza

Document Subject

Generated Narrative: MedicinalProductDefinition mp7303c8e036c7a4b4794a60ab51432595

identifier: http://ema.europa.eu/identifier/EU/1/22/1653/001

type: Medicinal Product

domain: Human use

status: active

legalStatusOfSupply: Medicinal product subject to medical prescription

name

productName: Upstaza 2.8 × 1011 vector genomes (vg)/0.5 mL solution for infusion

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-7303c8e036c7a4b4794a60ab51432595

identifier: http://ema.europa.eu/identifier/EU/1/22/1653/001

status: Final

type: Package Leaflet

category: Raw

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

author: Organization ACME industry

title: TEST PURPOSES ONLY - upstaza

Attesters

What is in this leaflet

1. What Upstaza is and what it is used for
2. What you need to know before you or your child is given Upstaza
3. How Upstaza is given to you or your child
4. Possible side effects
5. How Upstaza is stored
6. Contents of the pack and other information

What Upstaza is Upstaza is a gene therapy medicine that contains the active substance eladocagene exuparvovec.

What Upstaza is used for Upstaza is used for the treatment of patients aged 18 months and older, with a deficiency of the protein called aromatic L-amino acid decarboxylase (AADC). This protein is essential to make certain substances that the body s nervous system needs to work properly.

AADC deficiency is an inherited condition caused by a mutation (change) in the gene that controls the production of AADC (also called dopa decarboxylase or DDC gene). The condition prevents development of the child s nervous system, which means that many of the body s functions do not develop correctly during childhood, including movement, eating, breathing, speech and mental ability.

How Upstaza works The active substance in Upstaza, eladocagene exuparvovec, is a type of virus called adeno-associated virus that has been modified to include a copy of the DDC gene that works correctly. Upstaza is given by infusion (drip) into an area of the brain called the putamen, where AADC is made. The adeno-associated virus allows the DDC gene to pass into brain cells. In this way, Upstaza enables the cells to produce AADC so that the body can then make the substances that the nervous system needs.

The adeno-associated virus used to deliver the gene does not cause disease in humans.

You or your child will not be given Upstaza:

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

Warnings and precautions

• Mild or moderate uncontrollable jerky movements (also called dyskinesia) or sleep disorders (insomnia) may occur or worsen 1 month after treatment with Upstaza and last for several months after. Your doctor will decide if you or your child needs treatment for these effects.
• The doctor will monitor you or your child for complications of Upstaza treatment, such as leakage of the fluid surrounding the brain, meningitis, or encephalitis.
• Within the next days following the surgery, the doctor will monitor you or your child for any complications secondary to the surgery, the disease, and to the general anaesthesia. Some of the disease symptoms may be amplified during that period.
• Some specific symptoms of AADC deficiency may persist after treatment, examples of such symptoms may include impact on mood, sweating, and body temperature.
• After treatment, some medicine may enter your or your child s body fluids (eg, tears, blood, nasal secretions, and cerebrospinal fluid); this is known as shedding . You or your child and the child s caregiver (especially if pregnant, breast-feeding, or with a suppressed immune system) should wear gloves and place any used dressings and other waste material with tears and nasal secretions in sealed bags before throwing them away. You should follow these precautions for 14 days.
• You or your child must not donate blood, organs, tissues, and cells for transplantation after treatment with Upstaza. This is because Upstaza is a gene therapy product.

Children and adolescents Upstaza has not been studied in children under 18 months of age. Limited experience is available in children above 12 years.

Other medicines and Upstaza Tell your doctor if you or your child are taking, have recently taken, or might take any other medicines.

You or your child can receive routine childhood vaccinations as normal.

Pregnancy and breast-feeding and fertility

The effects of this medicine on pregnancy and the unborn child are not known.

Upstaza has not been studied in breast-feeding women.

There is no information on the effect of Upstaza on male or female fertility.

Upstaza contains sodium and potassium This medicine contains less than 1 mmol sodium (23 mg) per dose, that is to say essentially sodium-free .

This medicine contains less than 1 mmol potassium (39 mg) per dose, that is to say essentially potassium-free .

• Upstaza will be given to you or your child in an operating room by neurosurgeons experienced in brain surgery.
• Upstaza is given under anaesthetic. The neurosurgeon will talk to you about the anaesthesia and how it will be given.
• Before giving Upstaza, the neurosurgeon will make two small holes in you or your child s skull, one on each side.
• Upstaza will then be infused through these holes into four sites in your or your child s brain, in an area called the putamen.
• After the infusion, the two holes will be closed, and you or your child will have a brain scan.
• You or your child will need to stay in or near the hospital for a few days to monitor recovery and check for any side effects from the surgery or the anaesthesia.
• The doctor will see you or your child in the hospital twice, once around 1 week after the surgery, and then 3 weeks after the surgery, to continue following up on recovery and to check for any side effects from the surgery and treatment.

If you or your child is given more Upstaza than should be As this medicine is given to you or your child by a doctor, it is unlikely that you or your child will be given too much. If it does occur, your doctor will treat the symptoms, as necessary.

If you have any further questions on the use of this medicine, ask your doctor or nurse.

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

The following side effects may happen with Upstaza:

Very common (may affect more than 1 in 10 people)

• Dyskinesia (Uncontrollable jerky movements)
• Insomnia (difficulty sleeping), irritability

Common (may affect up to 1 in 10 people)

• Increased saliva production

The following side effects may happen with the surgery to administer Upstaza:

Very common (may affect more than 1 in 10 people)

• Low levels of red blood cells (anaemia)
• Leakage of the fluid surrounding the brain (called cerebrospinal fluid) (possible symptoms include headache, nausea and vomiting, neck pain or stiffness, change in hearing, sense of imbalance, dizziness or vertigo)

The following side effects may happen within the next 2 weeks following the surgery to administer Upstaza, due to either anaesthesia or to post-surgery effects:

Very common (may affect more than 1 in 10 people)

• Gastrointestinal bleeding, diarrhoea
• Fever, abnormal breath sounds
• Pneumonia
• Low level of blood potassium
• Irritability
• Hypotension (low blood pressure)

Common (may affect up to 1 in 10 people)

• Cyanosis (bluish discoloration of the skin caused by lack of oxygen in the blood)
• Mouth ulceration
• Hypothermia (low body temperature)
• Gastroenteritis
• Dyskinesia (Uncontrollable jerky movements)
• Respiratory failure
• Pressure sore, diaper rash, rash
• Tooth extraction
• Hypovolemic shock (severe loss of blood or body fluids)

Reporting of side effects If you or your child gets 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 via the national reporting system listed in Appendix V. By reporting side effects, you can help provide more information on the safety of this medicine.

The following information is intended for doctors only.

Upstaza will be stored at the hospital. It has to be stored and transported frozen at -65 oC. It is thawed before use and, once thawed, has to be used within 6 hours. It should not be re-frozen. Do not use this medicine after the expiry date, which is stated on the carton after EXP.

What Upstaza contains

• The active substance is eladocagene exuparvovec. Each 0.5 ml of solution contains 2.8 1vector genomes of eladocagene exuparvovec. The other ingredients are potassium chloride, sodium chloride, potassium dihydrogen phosphate, disodium hydrogen phosphate, poloxamer 188, water for injections (see section 2 Upstaza contains sodium and potassium ).

What Upstaza looks like and contents of the pack

Upstaza is a clear to slightly opaque, colourless to faint-white solution for infusion, supplied in a clear glass vial.

Each carton contains 1 vial.

Marketing Authorisation Holder
PTC Therapeutics International Limited
70 Sir John Rogerson's Quay Dublin 2 Ireland

Manufacturer Almac Pharma Services (Ireland) Limited
Finnabair Industrial Estate Dundalk, Co. Louth, A91 P9KD Ireland

For any information about this medicine, please contact the local representative of the Marketing Authorisation Holder:

AT, BE, BG, CY, CZ, DK, DE, EE, EL, ES, HR, HU, IE, IS, IT, LT, LU, LV, MT, NL, NO, PL, PT, RO, SI, SK, FI, SE, UK (NI) PTC Therapeutics International Ltd. (Ireland) +353 (0)1 447 5medinfo@ptcbio.com

FR PTC Therapeutics France Tel: +33(0)1 76 70 10 medinfo@ptcbio.com

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: