Sodium Phenylbutyrate (Buphenyl) – Side Effects, Interactions, Uses, Dosage, Warnings

Reviewed: June 23, 2020

Sodium phenylbutyrate is converted to a substance in the body that helps the kidneys eliminate waste substances from the body. These waste substances can produce ammonia, which is toxic if it builds up in your blood.

Sodium phenylbutyrate is used to treat urea cycle disorders in people who lack certain liver enzymes needed to properly eliminate waste substances from the body. This medicine helps prevent a build-up of ammonia in the blood.

Sodium phenylbutyrate may also be used for other purposes not listed in this medication guide.

uses

What is Sodium Phenylbutyrate (Buphenyl) used for?

• Urea Cycle Disorders

warnings

What is the most important information I should know about Sodium Phenylbutyrate (Buphenyl)?

You should not use sodium phenylbutyrate if you are allergic to it. This medicine will not treat immediate symptoms of an ammonia build-up.

To make sure sodium phenylbutyrate is safe for you, tell your doctor if you have:

• congestive heart failure;
• high blood pressure;
• severe kidney disease;
• swelling or fluid retention; or
• epilepsy or other seizure disorder.

FDA pregnancy category C. It is not known whether sodium phenylbutyrate will harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant while using this medicine.

It is not known whether sodium phenylbutyrate passes into breast milk or if it could harm a nursing baby. Tell your doctor if you are breast-feeding a baby.

Do not give this medicine to a child who weighs less than 44 pounds unless your doctor tells you to.

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Side Effects

What are the side effects of Sodium Phenylbutyrate (Buphenyl)?

Get emergency medical help if you have signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Even with treatment, urea cycle disorders can cause a build-up of ammonia in the blood which can result in life-threatening side effects. Seek emergency medical attention if you notice any signs of ammonia build-up, including mood or behavior changes, memory loss, thinking problems, muscle twitching, back and forth movement of the eyes, vomiting, increasing weakness, slurred speech, seizure (convulsions), and fainting.

Call your doctor at once if you have:

• severe drowsiness, confusion;
• swelling, rapid weight gain;
• a light-headed feeling, like you might pass out; or
• pale skin, feeling short of breath, rapid heart rate.

Common side effects may include:

• missed menstrual periods, changes in your regular cycle;
• loss of appetite;
• headache; or
• unusual or unpleasant taste in your mouth.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Pregnancy & Breastfeeding

Can I take Sodium Phenylbutyrate (Buphenyl) if I’m pregnant or breastfeeding?

FDA pregnancy category C. It is not known whether sodium phenylbutyrate will harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant while using this medicine.

It is not known whether sodium phenylbutyrate passes into breast milk or if it could harm a nursing baby. Tell your doctor if you are breast-feeding a baby.

Interactions

What drugs and food should I avoid while taking Sodium Phenylbutyrate (Buphenyl)?

Follow your doctor’s instructions about any restrictions on food, beverages, or activity.

Dosage Guidelines & Tips

How to take Sodium Phenylbutyrate (Buphenyl)?

Use Sodium Phenylbutyrate (Buphenyl) exactly as directed on the label, or as prescribed by your doctor. Do not use in larger or smaller amounts or for longer than recommended.

What should I do if I missed a dose of Sodium Phenylbutyrate (Buphenyl)?

Take the missed dose as soon as you remember. Skip the missed dose if it is almost time for your next scheduled dose. Do not take extra medicine to make up the missed dose.

Be sure to take this medicine with food.

Overdose Signs

What happens if I overdose on Sodium Phenylbutyrate (Buphenyl)?

If you think you or someone else may have overdosed on: Sodium Phenylbutyrate (Buphenyl),  call your doctor or the Poison Control center

(800) 222-1222

If someone collapses or isn’t breathing after taking Sodium Phenylbutyrate (Buphenyl), call 911

911

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Medical Disclaimer

Drugs A-Z provides drug information from Everyday Health and our partners, as well as ratings from our members, all in one place. Cerner Multum™ provides the data within some of the Overview, Uses, Warnings, Side Effects, Pregnancy, Interactions, Dosage, Overdose, and Images sections. The information within all other sections is proprietary to Everyday Health. 

Sodium Phenylbutyrate Oral: Uses, Side Effects, Interactions, Pictures, Warnings & Dosing

Uses

This medication is used along with diet changes for long-term treatment of a certain inherited disorder (urea cycle disorder). It helps remove a certain chemical (ammonia) from the body. Too much ammonia in the body can cause brain damage and sometimes death.This medication should not be used to treat sudden, severely high levels of ammonia in the body. Get medical help right away if you have sudden symptoms of too much ammonia, such as trouble thinking, nausea, vomiting, irritability, trouble speaking, trouble walking, fainting.

How to use Sodium Phenylbutyrate 483 Mg/Gram Oral Granules

Read the Patient Information Leaflet if available from your pharmacist before you start taking this medication and each time you get a refill. If you have any questions, ask your doctor or pharmacist.

Take this medication by mouth exactly as directed by your doctor. The dosage is based on your weight or body size, medical condition, and response to treatment. The total daily dose should be divided into equal amounts and taken with each meal or feeding (such as 3 to 6 times daily). Do not increase or decrease your dose without your doctor’s approval.

Carefully measure the dose using a special measuring device/spoon. Take this medication with water, fruit juice, or protein-free infant formula. You may also sprinkle the dose onto a spoonful of solid food (such as apple sauce) and eat right away to prevent salty taste. Do not chew the medication. Do not prepare a supply in advance.

Some brands may be given by a feeding tube (nasogastric/gastric). For specific directions, ask your health care professional.

Use this medication regularly to get the most benefit from it. To help you remember, take it at the same times each day.

Side Effects

Loss of appetite or menstrual changes (delayed/irregular/absent periods) may occur. If any of these effects last or get worse, tell your doctor or pharmacist promptly.

Remember that this medication has been prescribed because your doctor has judged that the benefit to you is greater than the risk of side effects. Many people using this medication do not have serious side effects.

Tell your doctor right away if you have any serious side effects, including: drowsiness/lightheadedness, easy bruising/bleeding, fast/pounding heartbeat, mental/mood changes, signs of infection (such as sore throat that doesn’t go away, fever), tiredness, signs of metabolic imbalance (such as rapid breathing, muscle twitching/spasms).

A very serious allergic reaction to this drug is rare. However, get medical help right away if you notice any symptoms of a serious allergic reaction, including: rash, itching/swelling (especially of the face/tongue/throat), severe dizziness, trouble breathing.

This is not a complete list of possible side effects. If you notice other effects not listed above, contact your doctor or pharmacist.

In the US – Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088 or at www.fda.gov/medwatch.

In Canada – Call your doctor for medical advice about side effects. You may report side effects to Health Canada at 1-866-234-2345.

Precautions

Before taking this medication, tell your doctor or pharmacist if you are allergic to it; or if you have any other allergies. This product may contain inactive ingredients, which can cause allergic reactions or other problems. Talk to your pharmacist for more details.

Before using this medication, tell your doctor or pharmacist your medical history, especially of: a certain heart problem (heart failure), kidney disease, liver disease, swelling (edema).

This medication contains salt (sodium). Before taking this medication, tell your doctor or pharmacist if you are on a low-salt diet.

Some brands of this medication may contain sugar. Caution is advised if you have diabetes, fructose intolerance, or any other condition that requires you to limit/avoid sugar in your diet. Ask your doctor or pharmacist about using this medication safely.

This drug may make you drowsy. Alcohol or marijuana (cannabis) can make you more drowsy. Do not drive, use machinery, or do anything that needs alertness until you can do it safely. Limit alcoholic beverages. Talk to your doctor if you are using marijuana (cannabis).

Before having surgery, tell your doctor or dentist about all the products you use (including prescription drugs, nonprescription drugs, and herbal products).

During pregnancy, this medication should be used only when clearly needed. Discuss the risks and benefits with your doctor.

It is unknown if this medication passes into breast milk. Consult your doctor before breast-feeding.

Interactions

Drug interactions may change how your medications work or increase your risk for serious side effects. This document does not contain all possible drug interactions. Keep a list of all the products you use (including prescription/nonprescription drugs and herbal products) and share it with your doctor and pharmacist. Do not start, stop, or change the dosage of any medicines without your doctor’s approval.

Does Sodium Phenylbutyrate 483 Mg/Gram Oral Granules interact with other drugs you are taking?

Enter your medication into the WebMD interaction checker

Overdose

If someone has overdosed and has serious symptoms such as passing out or trouble breathing, call 911. Otherwise, call a poison control center right away. US residents can call their local poison control center at 1-800-222-1222. Canada residents can call a provincial poison control center.

Do not share this medication with others.

Lab and/or medical tests (such as plasma ammonia/amino acid levels, complete blood count, blood chemistries, urine tests, drug levels) should be done while you are taking this medication. Keep all medical and lab appointments. Consult your doctor for more details.

If you miss a dose, take it as soon as you remember. If it is near the time of the next dose, skip the missed dose. Take your next dose at the regular time. Do not double the dose to catch up. It is very important to take the full amount prescribed for each 24-hour period.

Store the granules at room temperature away from light and moisture. Do not store in the bathroom. Use within 45 days of opening. Keep all medications away from children and pets.

Do not flush medications down the toilet or pour them into a drain unless instructed to do so. Properly discard this product when it is expired or no longer needed. Consult your pharmacist or local waste disposal company.

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Selected from data included with permission and copyrighted by First Databank, Inc. This copyrighted material has been downloaded from a licensed data provider and is not for distribution, except as may be authorized by the applicable terms of use.

CONDITIONS OF USE: The information in this database is intended to supplement, not substitute for, the expertise and judgment of healthcare professionals. The information is not intended to cover all possible uses, directions, precautions, drug interactions or adverse effects, nor should it be construed to indicate that use of a particular drug is safe, appropriate or effective for you or anyone else. A healthcare professional should be consulted before taking any drug, changing any diet or commencing or discontinuing any course of treatment.

instructions for use, dosage, composition, analogs, side effects / Pillintrip

1. What is AMMONAPS and what is it used for

AMMONAPS is indicated for patients with urea cycle disorders. Patients with these rare disorders are deficient in certain liver enzymes so they cannot remove residual nitrogen. Since nitrogen is a constituent of proteins, it accumulates in the body after protein intake. Residues of nitrogen in the form of ammonia are particularly toxic to the brain and, in severe cases, can lead to loss of consciousness and coma.
AMMONAPS helps the body remove remaining nitrogen and reduces the amount of ammonia in the body.

2. Que necesita saber antes de empezar a tomar AMMONAPS No tome AMMONAPS:

– If she is pregnant.
– If he is breastfeeding.
– If you are allergic (hypersensitive) to sodium phenylbutyrate or any of the other ingredients of this medicine (listed in section 6).

Advertencias y precauciones

Talk to your doctor or pharmacist before taking AMMONAPS
– If you have difficulty swallowing. AMMONAPS tablets can get stuck in the esophagus and cause ulcers. If you find it difficult to swallow them, it is recommended to use AMMONAP granules as an alternative.
– If you suffer from heart failure, kidney failure or other disease that may be aggravated by water retention due to the sodium salt content of this preparation.
– If you suffer from liver or kidney failure, since AMMONAPS is eliminated from the body through the kidney and liver.
– When administered to young children, as they may be unable to swallow tablets and choke. As an alternative, it is recommended to administer granulated AMMONAPS.
AMMONAPS should be combined with a low protein diet provided by a physician or
nutritionist specifically for your case. You will need to follow this diet carefully.
AMMONAPS does not completely prevent the formation of excess ammonia in the blood and is not suitable for the treatment of this disorder, which is considered a medical emergency.
If you need to have tests done, it is important to remind your doctor that you are taking AMMONAPS, as sodium phenylbutyrate may change the results of some tests.

Uso de AMMONAPS con otros medicamentos

Tell your doctor or pharmacist if you are using, have recently used or may need to use any other medicines.
It is especially important to tell your doctor if you are taking medicines containing:
– valproic acid (an antiepileptic drug)
– haloperidol (used to treat certain psychotic disorders)
– corticoids (cortisone-like drugs used to relieve inflamed areas of the body)
– probenecid (to treat hyperuricemia associated with gout)
These drugs can change the effect of AMMONAPS and you need to more frequent blood tests. If you are not sure if the medicines you are using contain these active substances, ask your doctor or pharmacist.

Embarazo and lactancia

Do not take AMMONAPS if you are pregnant as this medicine may harm the fetus. If you can become pregnant, you must use an effective method of contraception during treatment with AMMONAP.
Do not use AMMONAPS if you are breast-feeding as this medicine can pass into your breast milk and harm your baby.

Conducción y uso de máquinas

No studies have been conducted on the effect on the ability to drive and use machines.

AMMONAPS contiene sodio

Each AMMONAPS tablet contains 62 mg of sodium, which should be taken into account in patients on low sodium diets.

3. Como tomar AMMONAPS

Follow your doctor’s directions for administering this medicine exactly. If in doubt, ask your doctor or pharmacist again.

Dosificación

The daily dose of AMMONAPS is calculated based on your protein tolerance, diet and body weight or body surface area. You will need to have regular blood tests to adjust the correct daily dose. Your doctor will tell you how many tablets you should take.

Forma de administración

AMMONAPS should be taken orally in equal doses with each meal (eg three times a day). You must take AMMONAPS with plenty of water.
AMMONAPS should be taken with a special low protein diet. AMMONAPS tablets should not be given to children who are unable to swallow
squeezing. As an alternative, it is recommended to administer granulated AMMONAPS.
You will need to stay on diet and medication for life if you do not successfully have a liver transplant.

Si toma más AMMONAPS del que debiera

Patients who have taken very high doses of AMMONAPS experience:
– drowsiness, fatigue, dizziness and, rarely, confusion
– headache
– changes in taste (taste disorders)
– decreased hearing loss
– confusion
– memory problems,
– worsening of existing neurological conditions
If you experience any of these symptoms, you should immediately contact your doctor or the nearest hospital emergency department for symptomatic treatment.

Si olvidó tomar AMMONAPS

You should take your dose as soon as possible with your next meal. Make sure they go at least 3 hours between two doses. Do not take a double dose to make up for missed doses.
If you have any other doubts about the use of this medicine, ask your doctor or pharmacist.

4. Posibles efectos adversos

Like all medicines, this medicine can cause side effects, even if not everyone gets them.
The frequencies of possible adverse effects are listed below. Very common: Affects more than 1 in 10 people
Common: Affects 1 to 10 in 100 rare people: Affects 1 to 10 in 1,000 rare people: Affects 1 to 10 in 10,000 very rare people: Affects less than 1 in 10 000 people
Not known: frequency cannot be calculated from the available data

Very common side effects: irregular menstruation and cessation of menstruation.

It is possible that the absence of menstruation in sexually active women is not caused by the use of AMMONAPS. If this happens, check with your doctor as you may be pregnant (see “pregnancy and breastfeeding” in the previous section).

Common side effects: changes in the number of blood cells (erythrocytes, leukocytes and platelets), decreased appetite, depression, irritability, headache, fainting, fluid retention (bloating), changes in taste (taste disorders), abdominal pain, vomiting, nausea, constipation, body odor, rash, impaired kidney function, weight gain, change in test results.

Rare side effects: low red blood cell count (anemia) due to bone marrow depression, abnormal heart rhythm, rectal bleeding, stomach irritation, gastroduodenal ulcer, inflammation of the pancreas.

If you have persistent vomiting, you should consult your doctor immediately.

Comunicación de efectos adversos

If you experience any side effects, contact your doctor or pharmacist, even if they are possible side effects that are not listed in this leaflet. You can also contact them

directly through the National Notification System included in Appendix V. By reporting side effects, you can contribute to providing more information about the safety of this drug.

5. Conservation de AMMONAPS

Keep this medicine out of sight and out of the reach of children.
Do not use this medicine after the expiry date which is stated on the packaging and vial label after “CAD”” Expiry date is the last day of the month indicated.
Do not store above 30°C. Ask your pharmacist how to dispose of packaging and medicines that you don’t need, this way it will help protect the environment.0003
6. Contenido del envase e información adicional
Composición de AMMONAPS

– The active substance is sodium phenylbutyrate
Each AMMONAPS tablet contains 500 mg of sodium phenylbutyrate.
– The other components are microcrystalline cellulose, magnesium stearate and anhydrous colloidal silica.

Aspecto de producto y contenido del envase

AMMONAPS tablets are whitish, oval and engraved with “UCY 500”.
Tablets are packaged in plastic jars with baby stoppers. Each jar contains 250 or 500 tablets.

Titular de la autorización de comercialización

Horizon Pharma Ireland Limited
Connaught House, 1st Floor,
1 Burlington Road, Dublin 4, D04C5Y6
Ireland

Responsable de la fabricación

Bioglan AB
PO Box 50310
SE-202 13 Malmö
Sweden

Fecha de la última revisión de este prospecto:

Detailed information about this medicine is available on the website of the European Agency
Medicines http://www.ema.europa.eu/

Prospecto: información para el usuario AMMONAPS 940 mg/g granulado

Sodium phenylbutyrate

– Conserve este prospecto, ya que puede tener que volver a leerlo.

– If in any doubt, ask your doctor or pharmacist.
– This medicine has only been prescribed for you and you should not give it to other people, even if they have the same symptoms as you, because it could harm them.
– If you experience side effects, contact your doctor or pharmacist, even if they are side effects that do not appear in this leaflet. section 4.

Contenido del prospecto:

1. What AMMONAPS is and what it is used for
2. What you need to know before you start taking AMMONAPS
3. How to take AMMONAPS
4. Possible side effects of
5. Preservation AMMONAPS
6. contents of the container and additional information

1. Qué es AMMONAPS y para qué se utiliza

AMMONAPS is indicated for patients with urea cycle disorders. Patients with these rare disorders do not have certain liver enzymes. Since nitrogen is a constituent of proteins, it accumulates in the body after protein intake. The remaining nitrogen, in the form of ammonia, is especially toxic to the brain and, in severe cases, can lead to decreased levels of consciousness and coma.
AMMONAPS helps the body remove remaining nitrogen and reduces the amount of ammonia in the body.

2. Antes de tomar AMMONAPS No tome AMMONAPS:

– If she is pregnant.
– If he is breastfeeding.
– If you are allergic (hypersensitive) to sodium phenylbutyrate or any of the other ingredients of this medicine (listed in section 6).

Advertencias y precauciones

Talk to your doctor or pharmacist before taking AMMONAPS
– If you suffer from heart failure, decreased kidney function or any other condition in which the retention of the sodium salt in this medicine may make your condition worse.

– If you suffer from decreased liver or kidney function, since AMMONAPS is removed from the body

via the kidneys and liver.
AMMONAPS should be combined with a low protein diet that your doctor or nutritionist has developed specifically for you. You will need to follow this diet carefully.
AMMONAPS does not completely prevent the formation of excess ammonia in the blood and is not suitable for the treatment of such a disorder, which is a medical emergency.
If you need to have tests, it is important to remind your doctor that you have been taking AMMONAPS for
that sodium phenylbutyrate may change the results of some tests.

Toma de AMMONAPS con otros medicamentos

Tell your doctor or pharmacist if you are using, have recently used or may need to use any other medicines.
It is especially important to tell your doctor if you are taking medicines containing:
– valproic acid (an antiepileptic drug)
– haloperidol (used to treat certain psychotic disorders)
– corticoids (cortisone-like drugs used to relieve inflamed areas of the body)
– probenecid (to treat hyperuricemia associated with gout)
These drugs may change the effect of AMMONAPS and you may need to have more frequent blood tests. If you are not sure if your medicines contain these substances, ask your doctor or pharmacist.

Embarazo y lactancia

Do not take AMMONAP if you are pregnant as this medicine may harm the fetus. If you can become pregnant, you must use an effective method of contraception during treatment with AMMONAP.
Do not use AMMONAPS if you are breast-feeding as this medicine can pass through your breast milk and harm your baby.

Conducción y uso de máquinas

No studies have been conducted on the ability to drive and use machines.

AMMONAPS contiene sodio

A small white tablespoon of granulated Ammonaps contains 149 mg of sodium. An average yellow tablespoon of granulated Ammonaps contains 408 mg of sodium. A large blue tablespoon of granulated Ammonaps contains 1200 mg of sodium.
What to consider in patients on low sodium diets.

3. Cómo tomar AMMONAPS

Follow exactly the instructions given by your doctor for the administration of this medicine. If in doubt, ask your doctor or pharmacist again.

Dosificación

The daily dose of AMMONAPS will be calculated based on your protein tolerance, diet and body weight or body surface area. You will need to have regular blood tests to adjust the correct daily dose. Your doctor will tell you how many tablets you should take

Forma de administración

AMMONAPS should be taken in equal doses by mouth, by gastrostomy (a tube that goes down the abdomen into the stomach) or by a nasogastric tube (a tube that goes from the nose to the stomach).
AMMONAPS should be taken with a special low protein diet.
AMMONAPS should be taken at every meal or feeding. In young children, this can be from 4 to
6 times a day.
For dose measurement:

* Shake the jar lightly before opening it

* Use the correct spoon depending on how many Ammonaps you need: 1.2 g =

white small spoon, 3.3 g = yellow medium spoon and 9.7 d = large blue spoon

* Remove a heap of a tablespoon of granulate from the jar

* Pass a flat surface, such as the back of a knife blade, over the spoon to remove excess granules

* The granulate remaining in the spoon corresponds to the measurement of a tablespoon

* Remove the desired number of tablespoons of granulate from the jar

If administered orally:

Mix the measured dose with solid foods (such as mashed potatoes or applesauce) or liquids (such as water, apple juice, orange juice or seed milk without protein) and swallow it immediately after preparing the formula.

Patients with a gastrostomy or nasogastric tube:

Mix the granulate with water until the granulate is dry (stirring the solution helps dissolve the granulate). By dissolving the granulate in water, you will get a milky white liquid. Make a decision immediately after the mixture is done.
You will need to stay on diet and medication for life if you do not successfully have a liver transplant.

Si toma más AMMONAPS del que debiera

Patients who have taken very high doses of AMMONAPS experience:
– drowsiness, fatigue, dizziness and, rarely, confusion
– headache
– changes in taste (taste disorders)
– decreased hearing
– disorientation
– memory problems,
– worsening of existing neurological conditions
If you experience any of these symptoms, you should immediately contact your doctor or the nearest hospital emergency room for symptomatic treatment.

Si olvidó tomar AMMONAPS

You should take the dose as soon as possible with the next meal. Make sure they go at least 3 hours between two doses. Do not take a double dose to make up for missed doses.
If you have any other doubts about the use of this medicine, ask your doctor or pharmacist.

4. Posibles efectos adversos

Like all medicines, this medicine can cause side effects, even if not everyone gets them.
The frequencies of possible adverse effects are listed below. Very common: Affects more than 1 in 10 people
Common: Affects 1 to 10 people in 100
Rare: Affects 1 to 10 people in 1000 10,000 people
Unknown: frequency cannot be calculated from available data

Very common side effects: Irregular menstruation and cessation of menstruation.

It is possible that the absence of menstruation in sexually active women is not caused by the use of AMMONAPS. If this happens, check with your doctor as you may be pregnant (see “pregnancy and breastfeeding” in the previous section).

Common side effects: changes in the number of blood cells (erythrocytes, leukocytes and platelets), decreased appetite, depression, irritability, headache, fainting, fluid retention (bloating), changes in taste (taste disorders), abdominal pain, vomiting, nausea, constipation, body odor, rash, impaired kidney function, weight gain, change in test results.

Rare side effects: low red blood cell count (anemia) due to bone marrow depression, heart rhythm disturbances, rectal bleeding, stomach irritation, gastroduodenal ulcer, inflammation of the pancreas.

If persistent vomiting occurs, you should consult your physician immediately.

Comunicación de efectos adversos

If you experience any side effects, please contact your doctor or pharmacist, even if they are possible side effects not listed in this leaflet. You can also report them directly through the National Notification System included in Appendix V. By reporting side effects, you can contribute to providing more information about the safety of this drug.

5. Conservación de AMMONAPS

Keep this medicine out of sight and out of the reach of children.
Do not use AMMONAPS after the expiration date shown on the packaging and on the jar label after “CAD”” Expiry date is the last day of the month indicated.
Do not store above 25°C. Ask your pharmacist how to get rid of packaging and medicines you don’t need so it helps protect the environment.0003 6. Contenido del envase e información adicional Composición de AMMONAPS

– Active substance – sodium phenylbutyrate

One gram of Ammonaps granules contains 940 mg of sodium phenylbutyrate.

– Other ingredients are magnesium stearate and anhydrous colloidal silica.

Aspecto del producto y contenido del envase

AMMONAPS granulate whitish.
The granulate is packed in plastic jars with baby stoppers. Each jar contains
266 or 532 g granulate. Three spoons (small white spoon, medium yellow spoon and large blue spoon) are included to measure the daily dose.

Titular de la autorización de comercialización

Horizon Pharma Ireland Limited
Connaught House, 1st Floor,
1 Burlington Road, Dublin 4, D04C5Y6
Ireland

Responsable de la fabricación

Bioglan AB
PO Box 50310
SE-202 13 Malmö
Sweden

Fecha de la última revisión de este prospecto:

Detailed information about this drug is available on the website of the agency
European Medicamentoa http://www. ema.europa.eu

long-awaited debut in the reduced ALS market – ThePharma.Media

Amyotrophic Lateral Sclerosis (ALS) – one of the most severe diseases with unmet medical needs. A little-known company Amylyx Pharmaceuticals, which developed a combination of sodium phenylbutyrate and tauroursodeoxycholic acid, proposed its own treatment option for ALS.

Amylyx Pharmaceuticals has registered Albrioza . True, so far only in Canada – despite the fact that this is an American company, and the US market is always considered as a starting point by all developers of high-profile innovations.

Amyotrophic Lateral Sclerosis: Limited Treatment Options

Amyotrophic Lateral Sclerosis (ALS) is a relentlessly progressive, fatal neurodegenerative disease caused by the relentless death of motor neurons in the brain and spinal cord.

This still largely mysterious disease results in loss of muscle function, the ability to move and even speak, respiratory paralysis, and eventually death.

Existing treatment options for ALS are mainly symptomatic, and the specific drugs approved for this indication (riluzole and edaravone) can only moderately slow the progression of the disease.

Amylyx Pharmaceuticals has developed another option, an oral formulation in powder form for suspension, aimed at curbing motor neuron dysfunction and death. The novelty contains 3 grams of sodium phenylbutyrate and 1 gram of tauroursodeoxycholic acid – drugs already used in medicine, therefore, with proven safety.

However, the American company has been developing its candidate for a long time, and the first country in the world to approve Albrioza (conditionally and only for adults), just the other day, was Canada. There, this product will go on sale by August 2022; its price is still unknown.

Amylyx Pharmaceuticals will also register it in the US, but the US regulator is still reviewing the application; A decision is expected by the end of September 2022. At the beginning of this year, the company also submitted an application to the EMA.

See also

Mechanism of action in amyotrophic lateral sclerosis: how does a new ALS drug work?

Josh Cohen and Justin Klee, authors of Albrioza

Albrioza, first known as AMX0035, is being developed by Josh Cohen and Justin Klee

“data-file-id=””>by a pair of young American scientists since 2013 who founded Amylyx Pharmaceuticals specifically to develop this candidate.

According to the authors, the combination of phenylbutyrate and tauroursodeoxycholic acid can address the multifactorial and complex nature of neurodegenerative diseases through a multidirectional mechanism of action (the candidate is also being tested in separate trials in Wolfram syndrome and Alzheimer’s disease).

With regard to ALS, AMX0035 is hypothesized to have a synergistic neuroprotective effect and reduce neuronal death Multiple molecular mechanisms are thought to be involved in the pathogenesis of amyotrophic lateral sclerosis, detrimental to neurons. In the case of ALS, the biochemical dysfunction of the endoplasmic reticulum and mitochondria plays a key role.

“data-file-id=””>by simultaneously alleviating endoplasmic reticulum stress and reducing mitochondrial dysfunction.

Albrioza: how effective is the new drug registered for the treatment of amyotrophic lateral sclerosis

At present, the efficacy of Albrioza is most strongly supported by the CENTAUR pivotal Phase 2 CT, which included 137 adult patients with ALS randomized into two groups: the first was given twice daily AMX0035, the second was placebo. The duration of treatment was 24 weeks. (Note that patients enrolled in CENTAUR were able to continue taking riluzole and/or edaravone.)

Primary endpoint is the rate of decline in the overall score on the Revised ALS Functional Assessment Scale This diagnostic scale is often criticized. For example, it is based on a patient survey. However, this is the only informative scale for assessing ALS today.

“data-file-id=””>ALSFRS-R – Revised Amyotrophic Lateral Sclerosis Functional Rating Scale.

Changes in ALSFRS-R functionality per month in the AMX0035 group were 1.24 points, in the control group – 1.66. The difference of 0.42 points was considered statistically significant, therefore, the candidate was considered effective.

In addition, the experimental drug showed benefits in reducing the rate of muscle strength loss and lung function deterioration, as well as reduced mortality, tracheostomy, and hospitalization.

An open-ended, long-term follow-up CENTAUR-OLE arm, where all participants received the active drug, was shown to prolong overall survival by a median of 6.5 months. Additional analysis showed that the duration of drug use, rather than the severity of the disease, is more important for prolonging the survival of patients with ALS.

Post hoc analyzes of CENTAUR and its extension showed that participants treated with AMX0035 had a median survival time of 33. 6 months, compared with only 14.8 months for placebo. And when participants who switched to CENTAUR-OLE after taking a placebo started taking the experimental drug, their survival increased to 20.8 months.

Therefore, Albrioza, inhibiting the death of motor neurons, may be useful in any stage of amyotrophic lateral sclerosis. And the duration of the use of this drug is directly related to the increase in survival.

In terms of safety profile, AMX0035 was reasonably well tolerated, albeit with an increased incidence of ECG abnormalities and non-serious GI adverse reactions.

Details about the effect of the new drug on a more diverse population of ALS patients will be explored in another large-scale trial, PHOENIX, which will involve 600 people. Results are expected by spring 2024.

In the meantime, looking at the available results, one cannot cheer that ALS patients have a chance at healing.

Alas, Albrioza is far from a panacea, it is not even clear how much it changes the course of ALS, patients treated with the drug still suffered from the progression of the disease. The developers themselves acknowledge that more extensive and lengthy studies are needed, both to verify the data obtained and to resolve the remaining questions. But still, the emergence of a new remedy for amyotrophic lateral sclerosis is a major event for neurology and medicine in general.

The response of the patient and medical communities

While unknown, Endaravone, marketed under the Radicava brand by Mitsubishi Tanabe, was approved by the U.S. regulator in May 2017 based on data similar to what Amylyx Pharmaceuticals has collected so far in 2021 for its drug Amylyx Pharmaceuticals in a mid-phase CT.

“data-file-id=””>whether the FDA will approve the drug. At the end of 2020, the I AM ALS patient community and a non-profit organization that focuses on issues and concerns of ALS, as well as funding global research on this disease, The ALS Association sent to petition to the FDA asking for approval of Amylyx Pharmaceuticals as soon as possible The petition, which has received more than 50,000 signatures, urged the agency not to delay registration of a new drug, because patients who need a new drug will die if forced to wait a few more years before receiving results of additional KI.