Medunik USA, a specialty pharmaceutical company dedicated to providing rare disease patients with access to orphan drugs and part of Duchesnay Pharmaceutical Group (DPG), winner of the 2024 Life Sciences Innovation Award by ADRIQ, is pleased to announce that public reimbursement for Siklos (hydroxyurea) under Medicaid Programs is now available in 37 states, with 13 states including it on their Preferred Drug List, while 24 states offer it with limited, or no, restrictions. This important milestone for Medunik USA allows broader access to this essential medicine, benefiting patients suffering from sickle cell disease (SCD).
Siklos has been used worldwide to treat SCD for over 15 years and was approved by the FDA for use in the USA in 2017. This medicine is the only hydroxyurea (HU)-based therapeutic option that offers both flexible dosing and administration, giving patients and caregivers more control over managing their care and shaping a treatment routine that works for them. Patients can choose the best way to take Siklos, whether by swallowing it whole or split based on the dose, or dissolving it in a small amount of water.
Siklos® is the only FDA-approved dissolvable formulation of hydroxyurea that reduces painful crises and blood transfusions for sickle cell patients aged 2 years and older. Siklos has a BOXED WARNING regarding Low Blood Cell Count and Cancer. The most common side effects of Siklos in children include: infections and low white blood cells, and in adults include: infections, headache, and dry skin.
“We are proud to see increased coverage for this important therapy, which represents a significant step forward in the standard of care for patients with sickle cell disease. Siklos provides patients with options on how to best take their HU treatment,” said Tanya Carro, Executive Vice President, U.S. Entities. “With fewer SCD treatment options currently available, it is even more important to empower patients to stay on therapy in a way that fits their lives. That’s what drives long-term adherence and meaningful health outcomes.”
Sickle cell anemia is the most common inherited blood disorder, resulting in the production of abnormal hemoglobin, known as hemoglobin-S, responsible for the sickling of red blood cells. This disease affects nearly 100,000 Americans, decreases life expectancy by 25 to 30 years, induces significant morbidity and, may therefore, reduce quality of life. Most of those affected are of African ancestry; a minority are of Hispanic or southern European, Middle Eastern or Asian Indian descent.
Committed to improving the lives of people living with rare disorders, Medunik USA strives to remove the logistical barriers and to broaden reimbursement pathways to empower patients with SCD and their caregivers. Medunik USA supports patient access by offering a robust assistance program that includes educational resources and savings programs.
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INDICATION AND IMPORTANT SAFETY INFORMATION
SIKLOS is a prescription medicine that is used to reduce the frequency of painful crises and reduce the need for blood transfusions in adults and children, 2 years of age and older, with sickle cell anemia with recurrent moderate to severe painful crises. It is not known if SIKLOS is safe and effective in children less than 2 years of age.
IMPORTANT SAFETY INFORMATION
BOXED WARNING: LOW BLOOD CELL COUNT and CANCER
Low blood cell counts are common with SIKLOS, including low red blood cells, white blood cells, and platelets, and can be severe and life threatening. If your white blood cell count becomes very low, you are at increased risk for infection. Your healthcare provider will check your blood cell counts before and every 2 weeks during treatment with SIKLOS. Your healthcare provider may change your dose or tell you to stop taking SIKLOS if you have low blood cell counts. Tell your healthcare provider right away if you get any of the following symptoms: fever or chills; shortness of breath; body aches; unusual headache; feeling very tired; bleeding or unexplained bruising.
Cancer. Some people have developed cancer, such as leukemia and skin cancer, after taking SIKLOS for a long time. Your healthcare provider will check you for cancer. You should protect your skin from the sun using sunblock, hats, and sun-protective clothing.
WHAT IS THE MOST IMPORTANT INFORMATION YOU SHOULD KNOW ABOUT SIKLOS?
- SIKLOS can harm your unborn baby.
- For females taking SIKLOS who can become pregnant:
- You should talk with your healthcare provider about the risks of SIKLOS to your unborn baby.
- You should use effective birth control during treatment with SIKLOS and for at least 6 months after treatment with SIKLOS.
- Your healthcare provider will perform a pregnancy test before you start treatment with SIKLOS. Tell your healthcare provider right away if you become pregnant or think you may be pregnant.
- For males taking SIKLOS: SIKLOS can affect your sperm. If you have a female sexual partner who can become pregnant, you should use effective birth control during treatment with SIKLOS and for at least 6 months after treatment.
- SIKLOS may cause fertility problems in males. Talk to your healthcare provider if this is a concern for you.
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WHO SHOULD NOT TAKE SIKLOS
Do not take SIKLOS if you are allergic to hydroxyurea or any of the ingredients in SIKLOS.
WHAT SHOULD YOU TELL YOUR HEALTH CARE PROVIDER BEFORE TAKING SIKLOS?
Tell your healthcare provider about all of your medical conditions, including if you:
- have kidney problems or are receiving hemodialysis
- have liver problems
- have human immunodeficiency virus (HIV) or take HIV medicines. Taking SIKLOS with certain HIV medicines can cause serious reactions and may lead to death.
- have increased levels of uric acid in your blood (hyperuricemia)
- have a history of receiving interferon therapy or are currently receiving interferon therapy
- have leg wounds or ulcers
- plan to receive any vaccinations. You should not receive “live vaccines” during treatment with SIKLOS.
- are pregnant or plan to become pregnant. See “What is the most important information I should know about SIKLOS?”
- are breastfeeding or plan to breastfeed. It is not known if SIKLOS can pass into your breast milk. Do not breastfeed during treatment with SIKLOS.
- are using a continuous glucose monitor (CGM) to test your blood glucose. Talk to the healthcare provider that prescribed your CGM about whether it is safe to use while you are taking SIKLOS.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
WHAT ARE THE POSSIBLE SIDE EFFECTS OF SIKLOS?
SIKLOS may cause serious side effects, including:
- Skin ulcers, including leg ulcers, and death of skin tissue (gangrene) have happened in people who take SIKLOS. This has happened most often in people who receive interferon therapy or have a history of interferon therapy. Your healthcare provider will decrease your dose or stop treatment with SIKLOS if you develop any skin ulcers.
- Enlarged red blood cells (macrocytosis). Macrocytosis is common in people who take SIKLOS and can make it difficult to detect a decrease of folic acid. Your healthcare provider may prescribe a folic acid supplement for you.
- Hemolytic Anemia, the fast breakdown of red blood cells, has happened in people who take SIKLOS. Tell your healthcare provider I you develop yellowing of your skin (jaundice) or blood in your urine. Your healthcare provider may do blood tests if you have persistent or worsening anemia not related to sickle cell anemia.
The most common side effects of SIKLOS in children include: infections and low white blood cells.
The most common side effects of SIKLOS in adults include: infections, headache, and dry skin.
These are not all the possible side effects of SIKLOS.
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