All patients photographed may or may not still be on treatment.

How PYRUKYND® may help

What a change in hemoglobin
levels 
means for your PK-deficient 
red blood cells

All patients photographed may or may not still be on treatment.

Explore the areas in which
PYRUKYND may make a difference

HEMOGLOBIN LEVELS
NEED FOR TRANSFUSIONS
IMPACT OF SYMPTOMS
HEMOGLOBIN LEVELS NEED FOR TRANSFUSIONS IMPACT OF SYMPTOMS

Hemoglobin levels

Imagine what PYRUKYND 
can do for your hemolytic anemia

PYRUKYND helps activate the PK enzyme to keep red blood cells (RBCs) from breaking down too quickly and increase your hemoglobin (Hb) levels.

two drops of blood with plus sign inside two drops of blood with plus sign inside

Increased hemoglobin levels 
are 
a sign that anemia is improving

40% of patients

out of the 40 participants in Study 1 (ACTIVATE) responded to PYRUKYND compared to 0% receiving placebo, meaning their Hb increased by 1.5 g/dL or greater during at least 2 of 3 checkups at weeks 16, 20, and 24.

In Study 1 (ACTIVATE), initial increases in average Hb levels were seen as early as 2 weeks after starting treatment, and improvements generally continued over the course of the 6-month period.

PYRUKYND was also shown to slow the process of RBC breakdown (hemolysis), which could help RBCs live longer

Blood tests are used to measure levels of 
hemolysis. In clinical studies, PYRUKYND 
noticeably improved*:

A substance released from RBCs when they break down, which can cause yellowing 
of the skin/eyes (jaundice). During hemolysis, bilirubin levels increase
A protein produced by the liver that the body uses to clear hemoglobin found 
outside of RBCs. During hemolysis, haptoglobin is low
RBCs that are still developing. During hemolysis, reticulocyte levels increase
An enzyme found in RBCs as well as other cells. A high LDH level is a sign of hemolysis, as it indicates that RBCs may be breaking apart

*Participants on PYRUKYND showed a -1.5 mg/dL reduction in bilirubin, a 15.8 mg/dL increase in haptoglobin, a -10% reduction in reticulocytes percentage, and a -71 U/L reduction in LDH.

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Hear how PYRUKYND made a difference for Jim's PK
deficiency symptoms

Watch Jim's Story

Transfusions

Imagine an opportunity for fewer transfusions

Another possibility with PYRUKYND

In some cases, people with PK deficiency receive regular transfusions. In Study 2 (ACTIVATE-T), the need for 
transfusions was evaluated in 27 participants who were regularly transfused.

Patients had the opportunity to 
have fewer transfusions.

33% of patients

experienced a ≥33% reduction in the units transfused 
compared to their previous history.*

experienced a ≥33% reduction in the units transfused
compared to their previous history.*

And some were even 
transfusion free.

22% of patients

achieved transfusion-free status during the fixed-dose period 
of Study 2.*

achieved transfusion-free status during the fixed-dose period 
of Study 2.*

*Out of the 27 participants, 9 experienced a transfusion reduction and 6 became transfusion free.
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Impact of symptoms

A new day with PK deficiency and PYRUKYND

Patients taking PYRUKYND saw improvements in signs and symptoms of PK deficiency

Over the course of the clinical study, the PK Deficiency Diary (PKDD) was used to help track changes in these signs and symptoms of PK deficiency compared to placebo:

Jaundice

Tiredness

Shortness of breath

Treatment experiences and results with PYRUKYND will be different for everyone.

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Understand the safety of PYRUKYND

See the details

Important Safety Information

What is PYRUKYND?

PYRUKYND is a prescription medicine used to treat low red blood cell counts caused by the early breakdown of red blood cells (hemolytic anemia) in adults with pyruvate kinase (PK) deficiency.

It is not known if PYRUKYND is safe and effective in children.

Important Safety Information

PYRUKYND may cause serious side effects, including:

  • Rapid breakdown of red blood cells (acute hemolysis) has happened after suddenly interrupting or stopping treatment with PYRUKYND. You should not suddenly stop taking PYRUKYND. If you have to stop your treatment with PYRUKYND, your healthcare provider should monitor you closely. Tell your healthcare provider right away if you develop any signs or symptoms of breakdown of red blood cells including yellowing of the skin or the whites of your eyes (jaundice), dark colored urine, dizziness, confusion, feeling tired, or shortness of breath

The most common side effects of PYRUKYND were decrease in reproductive hormone (estrone) in men, increased salt from uric acid (urate) blood test, back pain, decrease in reproductive hormone (estradiol) in men, and joint pain (arthralgia).

These are not all the possible side effects of PYRUKYND. Call your healthcare provider for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Before taking PYRUKYND, tell your healthcare provider about all of your medical conditions, including if you:

  • have liver problems
  • are pregnant or plan to become pregnant. It is not known if PYRUKYND will harm your unborn baby. Tell your healthcare provider right away if you become pregnant or think that you are pregnant during treatment with PYRUKYND
  • are breastfeeding or plan to breastfeed. It is not known if PYRUKYND passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby during treatment with PYRUKYND

Tell your healthcare provider about all the medicines that you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. PYRUKYND and certain other medicines may affect each other causing side effects. PYRUKYND may affect the way other medicines work, and other medicines may affect how PYRUKYND works. Know the medicines you take. Keep a list of them to show your healthcare provider or pharmacist when you get a new medicine.

Please see full Prescribing Information, including Patient Information.

What is PYRUKYND?

PYRUKYND is a prescription medicine used
to treat low red blood cell counts caused
by the early breakdown of red blood cells (hemolytic anemia) in adults with pyruvate kinase (PK) deficiency.

It is not known if PYRUKYND is safe and effective in children.

IMPORTANT SAFETY INFORMATION

PYRUKYND may cause serious side effects including:

  • Rapid breakdown of red blood cells (acute hemolysis) has happened after suddenly interrupting or stopping treatment with PYRUKYND. You should not suddenly stop taking PYRUKYND. If you have to stop your treatment with PYRUKYND, your healthcare provider should monitor you closely. Tell your healthcare provider right away if you develop any signs or symptoms of breakdown of red blood cells including yellowing of the skin or the whites of your eyes (jaundice), dark colored urine, dizziness, confusion, feeling tired, or shortness of breath

Important Safety Information

What is PYRUKYND?

PYRUKYND is a prescription medicine used to treat low red blood cell counts caused by the early breakdown of red blood cells (hemolytic anemia) in adults with pyruvate kinase (PK) deficiency.

It is not known if PYRUKYND is safe and effective in children.

Important Safety Information

PYRUKYND may cause serious side effects, including:

  • Rapid breakdown of red blood cells (acute hemolysis) has happened after suddenly interrupting or stopping treatment with PYRUKYND. You should not suddenly stop taking PYRUKYND. If you have to stop your treatment with PYRUKYND, your healthcare provider should monitor you closely. Tell your healthcare provider right away if you develop any signs or symptoms of breakdown of red blood cells including yellowing of the skin or the whites of your eyes (jaundice), dark colored urine, dizziness, confusion, feeling tired, or shortness of breath

The most common side effects of PYRUKYND were decrease in reproductive hormone (estrone) in men, increased salt from uric acid (urate) blood test, back pain, decrease in reproductive hormone (estradiol) in men, and joint pain (arthralgia).

These are not all the possible side effects of PYRUKYND. Call your healthcare provider for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Before taking PYRUKYND, tell your healthcare provider about all of your medical conditions, including if you:

  • have liver problems
  • are pregnant or plan to become pregnant. It is not known if PYRUKYND will harm your unborn baby. Tell your healthcare provider right away if you become pregnant or think that you are pregnant during treatment with PYRUKYND
  • are breastfeeding or plan to breastfeed. It is not known if PYRUKYND passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby during treatment with PYRUKYND

Tell your healthcare provider about all the medicines that you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. PYRUKYND and certain other medicines may affect each other causing side effects. PYRUKYND may affect the way other medicines work, and other medicines may affect how PYRUKYND works. Know the medicines you take. Keep a list of them to show your healthcare provider or pharmacist when you get a new medicine.

Please see full Prescribing Information, including Patient Information.

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