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Patient Information & Instructions For Use

Patient Information

PURIXAN® (pure-ee-zan)
(mercaptopurine) oral suspension 20 mg/mL

What is PURIXAN?

PURIXAN is a prescription medicine used along with other medicines to treat people with acute lymphoblastic leukemia (ALL).

What should I tell my healthcare provider before taking PURIXAN?

Before you take PURIXAN, tell your healthcare provider about all of your medical conditions, including if you:

  • have kidney or liver problems.
  • have a condition where your body produces too little of the enzyme thiopurine methyltransferase (TPMT) or the enzyme nucleotide diphosphatase (NUDT15).
  • have recently received or plan to receive a vaccine.
  • are pregnant or plan to become pregnant. PURIXAN can harm your unborn baby.
    If you are a female who is able to become pregnant:

    • Your healthcare provider will do a pregnancy test before you start treatment with PURIXAN.
    • Use an effective method of birth control (contraception) during treatment with PURIXAN and for 6 months after your last dose. Talk with your healthcare provider about birth control methods you can use during this time.
    • Tell your healthcare provider right away if you become pregnant or think you are pregnant during treatment with PURIXAN.

    If you are a male with a female partner who is able to become pregnant:

    • Use effective birth control (contraception) during treatment with PURIXAN and for 3 months after your last dose.
    • Tell your healthcare provider right away if your female partner becomes pregnant or thinks she is pregnant during your treatment with PURIXAN.
  • are breastfeeding or plan to breastfeed. Do not breastfeed during treatment with PURIXAN and for at least 1 week after your last dose.
  • Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

How should I take PURIXAN?

  • See the detailed “Instructions for Use” that comes with PURIXAN for information about the right way to measure and take a dose of PURIXAN.
  • Take PURIXAN exactly as your healthcare provider tells you. Do not stop taking PURIXAN or change your dose without talking to your healthcare provider.
  • Take PURIXAN by mouth 1 time each day.
  • If PURIXAN comes into contact with skin, eyes, or clothes?
    • Remove contaminated clothing.
    • Wash skin or eyes immediately with water.
    • Contact with skin or eyes can cause hypersensitive reactions resulting in rash, redness, itching and inflammation. If symptoms appear seek medical attention.
  • During treatment with PURIXAN, your healthcare provider will do blood tests regularly to check your blood cell counts and liver function. Your healthcare provider may change your dose if you have side effects.
  • If you miss a dose of PURIXAN, call your healthcare provider for advice.
  • If you take too much PURIXAN, call your healthcare provider or go to the nearest emergency room right away.

What should I avoid while taking PURIXAN?

PURIXAN can make your skin more sensitive to sunlight. Protect yourself from sunlight during treatment with PURIXAN.

What are the possible side effects of PURIXAN?

PURIXAN can cause serious side effects, including:

  • Decreased blood cell counts are common with PURIXAN, but can also be severe. PURIXAN affects your bone marrow and can cause decreased white blood cells, red blood cells, and platelets. Decreased blood cell counts can make you more likely to develop infections, bleeding, or anemia. If you take certain medicines during treatment with PURIXAN, it could make the effects on your bone marrow worse. Tell your healthcare provider if you develop any of the following symptoms during treatment with PURIXAN:
    • fever
    • sore throat
    • cuts or wounds that are red, or swollen, or are draining
    • any bleeding
    • tiredness or weakness
    • shortness of breath
  • Liver problems. Increases in liver function test results are common with PURIXAN, but you can also develop severe liver problems with PURIXAN that can lead to death. Your healthcare provider may tell you to stop taking PURIXAN if you develop liver problems. Tell your healthcare provider right away if you develop any of the following symptoms of a liver problem during treatment with PURIXAN:
    • decreased appetite
    • diarrhea
    • nausea or vomiting
    • yellowing of your skin or the whites of your eyes
    • a build-up of fluid in your stomach-area (ascites)
  • Possible increased risk of other cancers. Talk with your healthcare provider about your risk of other cancers if you take PURIXAN.

Less common side effects of PURIXAN include: loss of appetite, nausea, vomiting, diarrhea, generally do not feel well, and rash.

Low blood sugar (hypoglycemia) can happen, especially in children under six years of age.

Tell your healthcare provider if you have any side effect that bothers you or that does not go away. These are not all the possible side effects of PURIXAN.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

How should I store PURIXAN?

  • PURIXAN comes in a bottle with a child-resistant cap.
  • Store PURIXAN between 59ºF to 77ºF (15ºC to 25ºC), in a dry place. Do not store above 25°C.
  • Store the oral dispensing syringe in a clean place, with the medicine.
  • PURIXAN oral suspension should be used within 8 weeks after opening the bottle. Dispose of (throw away) any unused medicine after 8 weeks.
  • Do not use after the expiry date which is stated on the carton and the bottle after ‘EXP’.
  • Keep the bottle tightly closed to prevent spoilage of the medicine and reduce the risk of accidental spillage.
  • Keep PURIXAN out of the reach of children, preferably in a locked cupboard. If a child accidentally takes PURIXAN, it could cause death.

How should I dispose of PURIXAN?

  • This medicine should not be disposed of in wastewater or household waste. Ask your pharmacist how to dispose of (throw away) PURIXAN that is no longer needed.

General information about the safe and effective use of PURIXAN.

Medicines are sometimes prescribed for purposes other than those listed in a Patient Information leaflet. Do not use PURIXAN for a condition for which it was not prescribed. Do not give PURIXAN to other people, even if they have the same symptoms you have. It could harm them. You can ask your healthcare provider or pharmacist for information about PURIXAN that is written for health professionals.

What are the ingredients in PURIXAN?

Active ingredient: mercaptopurine
Inactive ingredients: xanthan gum, aspartame, concentrated raspberry juice, sucrose, ethyl parahydroxybenzoate sodium, methyl parahydroxybenzoate sodium, potassium sorbate, sodium hydroxide and purified water.

Manufactured by: Nova Laboratories Ltd, Leicester, LE18 4YL, United Kingdom
Manufactured for: Rare Disease Therapeutics, Inc., 2550 Meridian Blvd. Suite 150, Franklin, TN 37067
For more information, go to www.purixan-us.com.
This Patient Information has been approved by the U.S. Food and Drug Administration
Revised: April 2020
Part Number: D001113/1

Instructions for Use

PURIXAN® (pure-ee-zan)
(mercaptopurine) oral suspension 20 mg/mL

Read these Instructions for Use before you start taking PURIXAN, and each time you get a refill. There may be new information. This information does not take the place of talking to your healthcare provider about your medical condition or your treatment.

Important information about measuring PURIXAN oral suspension

  • Always use the oral dispensing syringe provided with your PURIXAN oral suspension to make sure you measure the right amount.
  • You will be provided:
    • 1 bottle of PURIXAN oral suspension
    • 1 bottle adapter
    • 2 oral dispensing syringes (one 1 mL and one 5 mL)

If you did not receive an oral dispensing syringe with your PURIXAN oral suspension, ask your pharmacist to give you one.

You will also need disposable gloves.

Important Information You Need to Know Before Administering PURIXAN:

  1. Wash your hands well with soap and water before and after administering a dose.
  2. Put on disposable gloves before handling PURIXAN.
  3. Shake the bottle vigorously for at least 30 seconds to make sure that the medicine is well mixed (See Figure A).
  4. Remove the child-resistant bottle cap (see Figure B).
  5. Push the ribbed end of the bottle adapter into the neck of the bottle until it is firmly in place. The bottom edge of the adapter should fully contact the top rim of the bottle (See Figure C). Do not remove the adapter from the bottle after it is inserted.

Preparing a dose of PURIXAN:

  1. Hold the bottle upright. Remove the bottle cap by turning in the direction of the arrow (See Figure B).
  2. Push the tip of the oral dispensing syringe into the hole in the bottle adapter (See Figure D and Figure E).
  3. Turn the bottle upside down (See Figure F).
  4. Pull back slowly on the plunger of the oral dispensing syringe to withdraw the prescribed dose of PURIXAN. Pull the plunger back to the mL mark on the syringe that corresponds to the dose prescribed (Figure F). If you are not sure about how much medicine to draw into the oral dispensing syringe, always ask your doctor, pharmacist or nurse for advice.
  5. Leave the oral dispensing syringe in the bottle adapter and turn the bottle right-side up. Place the bottle onto a flat surface. Hold the oral dispensing syringe by the barrel and carefully remove it from the adapter. Do not hold the oral dispensing syringe by the plunger, because the plunger may come out.
  6. Place the tip of the oral dispensing syringe in your mouth and aim the tip toward the inside of your cheek.
  7. Gently squirt the PURIXAN oral suspension into your mouth by pushing on the plunger until the oral dispensing syringe is empty. Swallow the medicine.
    • Do not forcefully push on the plunger.
    • Do not squirt the medicine to the back of your mouth or throat. This may cause you to choke.
  8. Remove the oral dispensing syringe from your mouth.
  9. Swallow the dose of oral suspension then drink some water, making sure no medicine is left in your mouth.
  10. Put the cap back on the bottle with the adapter left in place. Close the cap tightly.
  11. Wash the oral dispensing syringe with warm soapy water and rinse well. Hold the oral dispensing syringe under water and move the plunger up and down several times to make sure the inside of the oral dispensing syringe is clean. Let the oral dispensing syringe dry completely before you use it again for
    dosing. Do not throw away the oral dispensing syringe after use.

Dispensing Instructions

Ingredients in PURIXAN

Active ingredient: mercaptopurine
Inactive ingredients: xanthan gum, aspartame, concentrated raspberry juice, sucrose, ethyl
parahydroxybenzoate sodium, methyl parahydroxybenzoate sodium, potassium sorbate, sodium hydroxide, and purified water.

Storing PURIXAN

  • Store PURIXAN between 59ºF to 77ºF (15ºC to 25ºC), in a dry place. Do not store above 25°C.
  • Store the oral dispensing syringe in a clean place, with the medicine.
  • PURIXAN oral suspension should be used within 8 weeks after opening the bottle. Dispose of (throw away) any unused medicine after 8 weeks.
  • Do not use after the expiry date which is stated on the carton and the bottle after ‘EXP’.
  • Keep the bottle tightly closed to prevent spoilage of the medicine and reduce the risk of accidental spillage.
  • Keep PURIXAN oral suspension and all medicines out of the reach of children, preferably in a locked cupboard. If a child accidentally takes PURIXAN, it could cause death. Ask your pharmacist how to dispose of (throw away) PURIXAN that is no longer needed.

Disposing of PURIXAN

  • Ask your pharmacist how to dispose of PURIXAN that is expired or no longer required. Medicines should not be disposed of via wastewater or household waste.

Clean Up Spillage of PURIXAN

Use appropriate personal protective equipment (disposable gloves and eye protection). Mop up and contain spill material in a compatible container. Wash your hands thoroughly afterwards.

PURIXAN Contact with Skin, Eyes, or Clothes

  • Remove and launder contaminated clothing.
  • Wash skin or eyes immediately with water.

Contact with skin or eyes can cause hypersensitive reactions resulting in rash, redness, itching and inflammation. If symptoms appear, seek medical attention.

Manufactured by: Nova Laboratories Ltd, Leicester, LE18 4YL, United Kingdom
Manufactured for: Rare Disease Therapeutics, Inc., 2550 Meridian Blvd., Suite 150, Franklin, TN 37067
Part Number: D001112/1
This Instructions for Use has been approved by the U.S. Food and Drug Administration.
Revised: April 2020
INDICATION

PURIXAN® is indicated for the treatment of patients with acute lymphoblastic leukemia (ALL) as part of a combination chemotherapy maintenance regimen.

IMPORTANT SAFETY INFORMATION
Contraindications

None.

Warnings and Precautions

Myelosuppression

The most consistent, dose-related adverse reaction of PURIXAN is myelosuppression, manifested by anemia, leukopenia, thrombocytopenia, or any combination of these. Monitor CBC and adjust the dosage of PURIXAN for excessive myelosuppression.

Consider testing for thiopurine S-methyltransferase (TPMT) or nucleotide diphosphatase (NUDT15) deficiency in patients with severe myelosuppression or repeated episodes of myelosuppression. TPMT genotyping or phenotyping (red blood cell TPMT activity) and NUDT15 genotyping can identify patients who have reduced activity of these enzymes. Patients with homozygous TPMT or NUDT15 deficiency may require a dose reduction.

Myelosuppression can be exacerbated by coadministration with allopurinol, aminosalicylates or other products that cause myelosuppression. Reduce the dosage of PURIXAN when coadministered with allopurinol.

Hepatotoxicity

Mercaptopurine is hepatotoxic. There are reports of deaths attributed to hepatic necrosis associated with the administration of mercaptopurine. Hepatic injury can occur with any dosage but seems to occur with greater frequency when the recommended dosage is exceeded. In some patients, jaundice has cleared following withdrawal of mercaptopurine and reappeared with rechallenge.

Monitor serum transaminase levels, alkaline phosphatase, and bilirubin levels at weekly intervals when first beginning therapy and at monthly intervals thereafter. Monitor liver tests more frequently in patients who are receiving PURIXAN with other hepatotoxic drugs or with known pre-existing liver disease. Withhold PURIXAN at onset of hepatotoxicity.

Immunosuppression

Mercaptopurine is immunosuppressive and may impair the immune response to infectious agents or vaccines. Due to the immunosuppression associated with maintenance chemotherapy for ALL, response to all vaccines may be diminished and there is a risk of infection with live virus vaccines. Consult immunization guidelines for immunocompromised patients.

Treatment Related Malignancies

Patients receiving immunosuppressive therapy, including mercaptopurine, are at an increased risk of developing lymphoproliferative disorders and other malignancies, notably skin cancers (melanoma and non-melanoma), sarcomas (Kaposi's and non-Kaposi's) and uterine cervical cancer in situ. The increased risk appears to be related to the degree and duration of immunosuppression. It has been reported that discontinuation of immunosuppression may provide partial regression of the lymphoproliferative disorder.

A treatment regimen containing multiple immunosuppressants (including thiopurines) should therefore be used with caution as this could lead to lymphoproliferative disorders, some with reported fatalities. A combination of multiple immunosuppressants, given concomitantly increases the risk of Epstein-Barr virus (EBV)-associated lymphoproliferative disorders.

Macrophage Activation Syndrome

Macrophage activation syndrome (MAS) (hemophagocytic lymphohistiocytosis) is a known, life-threatening disorder that may develop in patients with autoimmune conditions, in particular with inflammatory bowel disease (IBD), and there could potentially be an increased susceptibility for developing the condition with the use of mercaptopurine (an unapproved use). If MAS occurs, or is suspected, discontinue PURIXAN. Monitor for and promptly treat infections such as EBV and cytomegalovirus (CMV), as these are known triggers for MAS.

Embryo-Fetal Toxicity

PURIXAN can cause fetal harm when administered to a pregnant woman. An increased incidence of miscarriage has been reported in women who received mercaptopurine in the first trimester of pregnancy. Adverse embryo-fetal findings, including miscarriage and stillbirth, have been reported in women who received mercaptopurine after the first trimester of pregnancy. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with PURIXAN and for 6 months after the last dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with PURIXAN and for 3 months after the last dose.

Adverse Reactions

Based on multicenter cooperative group ALL trials, the most common adverse reaction occurring in > 20% of patients was myelosuppression, including anemia, neutropenia, lymphopenia and thrombocytopenia. Adverse reactions occurring in 5% to 20% of patients included anorexia, nausea, vomiting, diarrhea, malaise, and rash. Adverse reactions occurring in 5% to 20% of patients included anorexia, nausea, vomiting, diarrhea, malaise, and rash. Adverse reactions occurring in < 5% of patients included urticaria, hyperuricemia, oral lesions, elevated transaminases, hyperbilirubinemia, hyperpigmentation, infections, and pancreatitis. Oral lesions resemble thrush rather than antifolic ulcerations. Delayed or late toxicities include hepatic fibrosis, hyperbilirubinemia, alopecia, pulmonary fibrosis, oligospermia and secondary malignancies.

USE IN SPECIFIC POPULATIONS
  • Lactation: Because of the potential for serious adverse reactions in the breastfed child, advise women not to breastfeed during treatment with PURIXAN and for 1 week after the last dose.
  • Pediatric Use: Symptomatic hypoglycemia has been reported in pediatric patients with ALL receiving mercaptopurine. Reported cases were in pediatrics less than 6 years or with a low body mass index.
  • Renal Impairment: Use the lowest recommended starting dosage for PURIXAN or increase the dosing interval to every 36 to 48 hours in patients with renal impairment (CLcr less than 50 mL/min). Adjust the dose to maintain absolute neutrophil count (ANC) at a desirable level and for adverse reactions.
  • Hepatic Impairment: Use the lowest recommended starting dosage for PURIXAN in patients with hepatic impairment. Adjust the dose to maintain absolute neutrophil count (ANC) at a desirable level and for adverse reactions.

 

OVERDOSAGE

Signs and symptoms of mercaptopurine overdosage may be immediate (anorexia, nausea, vomiting, and diarrhea) or delayed (myelosuppression, liver dysfunction, and gastroenteritis). Dialysis cannot be expected to clear mercaptopurine. Hemodialysis is thought to be of marginal use due to the rapid intracellular incorporation of mercaptopurine into active metabolites with long persistence.

Withhold PURIXAN immediately if severe or life-threatening adverse reactions occur during treatment. If a patient is seen immediately following an accidental overdosage, it may be useful to induce emesis.

 

Please see complete Prescribing Information.

To report SUSPECTED ADVERSE REACTIONS, contact Rare Disease Therapeutics, Inc., at 1-844-472-7389 or by email at safety@raretx.com, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

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