How PYRUKYND® was studied
The safety and effectiveness of PYRUKYND was studied in 2 clinical trials
Study 1: Adults with PK deficiency
who were not regularly transfused
Study 2: Adults with PK deficiency
who were regularly transfused
STUDY 1 (ACTIVATE):
Not regularly transfused participants
(defined as receiving 4 or fewer blood transfusions a year)
Study 1 measured:
- Changes in participants’ hemoglobin and hemolysis levels
- Improvements in jaundice, tiredness, and shortness of breath
A response to PYRUKYND was measured by at least a 1.5 g/dL increase in hemoglobin during at least 2 of 3 checkups at weeks 16, 20, and 24.
How was the study set up?
40 participants were selected at random to receive PYRUKYND and 40 participants to receive placebo.
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Weeks 1 to 12 Dose increase period   Based on their hemoglobin levels, participants’ hematologists gradually increased their dose as needed. Weeks 13 to 24 Maintenance dose period  Participants then stayed at the same dose for 12 weeks. | P L A C E B O |
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Weeks 1 to 12 Dose increase period |
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Weeks 13 to 24 Maintenance dose period |
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Ongoing long-term study up to 1.5 years.
All participants in long-term study receive PYRUKYND.
STUDY 2 (ACTIVATE-T):
Regularly transfused participants
(defined as receiving 6 or more blood transfusions a year)
Study 2 measured changes in participants’ need for transfusions
A response to PYRUKYND was measured by at least a 33% reduction in the number of transfusions required during the
final 24-week period.
Transfusion reductions were measured in comparison to the number of RBC units they received in the year before the study.
How was the study set up?
All 27 participants received PYRUKYND. None received placebo.
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Weeks 1 to 16 Dose increase period |
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Weeks 17 to 40 Maintenance dose period |
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Weeks 1 to 16
Dose increase period
Participants’ doses of PYRUKYND were gradually increased over the course of 16 weeks.
Weeks 17 to 40
Maintenance dose period
Participants were then given the same dose of PYRUKYND for the next 24 weeks.
Ongoing long-term study
up to 1.8 years.
The safety of PYRUKYND has been studied up to 6 years in 4 clinical trials in 155 participants. The most common side effects of PYRUKYND were decreases in reproductive hormones in males (estrone and estradiol), increased salt from uric acid (urate), back pain, and joint pain.
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.
