Upstaze – Eladocagen-exuparvovec uses, dose and side effects


2.8 x 10 11  vector genome/0.5 ml infusion solution

What Upstaza is and what it is used for

What Upstaza is

Upstaza is a gene therapy medicine containing the active substance eladocagen-exuparvovec.

What Upstaza is used for

Upstaza is used to treat patients aged 18 months and older with a deficiency of a protein called aromatic L-amino acid decarboxylase (AADC). This protein is necessary for certain substances that the body’s nervous system needs to function 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 the development of the child’s nervous system, meaning that many of the body’s functions do not develop correctly during childhood, including movement, eating, breathing, speech, and mental abilities.

How Upstaza works

The active substance in Upstaza, eladokagen-exuparvovec, is a type of virus called adeno-associated virus that has been modified to include a copy of the DDC gene that works properly. Upstaza is given by infusion (drip) into an area of ​​the brain called the putamen, where AADC is made. The adeno-associated virus et 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 manufacture the substances that the nervous system needs.

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

What you need to know before Upstaza is given to you or your child

You or your child will not receive Upstaza:

  • if you or your child are allergic to 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 disturbances (insomnia) may occur or worsen 1 month after treatment with Upstaza and last for several months. The doctor will decide if you or your child needs treatment for these effects.
  • The doctor will monitor you or your child for complications of treatment with Upstaza, such as leakage of fluid surrounding the brain, meningitis ( inflammation of the membranes of the brain and spinal cord), or encephalitis (disease of the brain tissue).
  • For the next few days after surgery, the doctor will monitor your child for any complications caused by the surgery, the illness, and the anesthesia. Some disease symptoms may intensify during that period.
  • Some specific symptoms of AADC deficiency may persist after treatment, for example, such symptoms may affect mood, sweating, and body temperature.
  • After treatment, a small amount of the medicine may enter your or your child’s body fluids (eg tears, blood, nasal secretions, and spinal fluid), this is called ‘excretion’. You or your child and the child’s caregiver should (especially if you are pregnant, nursing, or have a suppressed immune system) wear gloves and place all used dressings and other waste with tears and nasal secretions in sealed bags before disposal. You should follow these precautions for 14 days.
  • You or your child must not donate blood, organs, tissues, or cells for transplantation after treatment with Upstaza. This is because Upstaza is a gene therapy product.

Children and young people

Upstaza has not been studied in children under 18 months of age. There is limited experience in children over 12 years of age.

Other medicines and Upstaza

Tell the doctor if you or your child are taking, have recently taken, or might be taking other medicines.

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

Pregnancy, breastfeeding, and fertility

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

Upstaza has not been studied in nursing 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 (23 mg) of sodium per dose, i.e. it is almost “sodium-free”.

This medicine contains less than 1 mmol (39 mg) of potassium per dose, i.e. it is almost “potassium-free”.

How Upstaza is given to you or your child

  • Upstaza will be given to you or your child in an operating room by neurosurgeons experienced in brain surgery.
  • Upstaza is given under anesthesia. The neurosurgeon will talk to you about the anesthesia and how it will be given.
  • Before the neurosurgeon gives Upstaza, he makes two small holes in your or your child’s skull, one on each side.
  • Upstaza will then be infused through these holes in four places in your or your child’s brain, in an area called the putamen.
  • After infusion one, 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 anesthesia.
  • The doctor will see you or your child in the hospital twice, about 1 week after the operation and then 3 weeks after the operation, to continue to monitor recovery and to check for any side effects from the operation and treatment.

If you or your child has been given too much Upstaza

Because this medicine is given to you or your child by a doctor, it is unlikely that you or your child will get too much. If it occurs, doctors will treat the symptoms as needed.

If you have any further questions about this medicine, ask your doctor or nurse.

Possible side effects

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

The following side effects can occur 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)

  • Increase in saliva production

The following side effects may occur during the operation to give Upstaza:

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

  • Low red blood cell count ( anemia )
  • Leakage of fluid that surrounds the brain (called cerebrospinal fluid) (possible symptoms include headache, nausea, and vomiting, neck pain or stiffness, altered hearing, feeling off balance, dizziness, or vertigo)

The following side effects may occur within the next 2 weeks after the operation to give Upstaza, due to the effects of anesthesia or the operation:

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

  • Bleeding in the gastrointestinal tract, diarrhea
  • Fever, abnormal breath sounds
  • Pneumonia
  • Low level of potassium in the blood
  • Irritability
  • Hypotension (low blood pressure )

Common (may affect up to 1 in 10 people)

  • Cyanosis (bluish discoloration of the skin caused by a lack of oxygen in the blood)
  • Laryngomalacia (laxity of the tissue of the larynx causing loud breathing)
  • Mouth ulcers
  • Hypothermia (low body temperature)
  • Gastroenteritis
  • Dyskinesia (uncontrollable jerky movements)
  • Respiratory failure
  • Pressure sores, diaper rash, skin rash
  • Tooth extraction
  • Hypovolemic shock (severe loss of blood or body fluids)

How to store Upstaza

Upstaza will be stored in the hospital. It must be stored and transported frozen at ≤ -65  o C. It is thawed before use and must be used within 6 hours of thawing. It should not be refrozen.

Use before the expiry date which is stated on the carton after the Expiration date.

Contents of the packaging and other information

Contents declaration

  • The active substance is eladocagen-exuparvovec. Each 0.5 ml solution contains 2.8 × 10 11 vector genomes of eladocagen-exuparvovec .

Other ingredients are potassium chloride, sodium chloride, potassium dihydrogen phosphate, disodium hydrogen phosphate, poloxamer 188, and water for injections (see section 2 “Upstaza contains sodium and potassium”).

Appearance and package sizes of the medicine

Upstaza is a clear to slightly opaque, colorless to slightly white solution for infusion, supplied in a transparent glass vial.

Each carton contains 1 vial.

Marketing Authorisation Holder

PTC Therapeutics International Limited

70 Sir John Rogerson’s Quay

Dublin 2



Almac Pharma Services (Ireland) Limited

Finnabair Industrial Estate

Dundalk, Co. Louth, A91 P9KD


This medicine has been approved under the rules of “exceptional approval”. This means that it has not been possible to obtain complete information about this medicine.

The European Medicines Agency reviews any new information every year and updates this leaflet when necessary.

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