19 June 2019
ROSEMONT, PA, June 19, 2019 – This World Sickle Cell Day, Medunik USA, a company dedicated to improving the health and quality of life of Americans with rare diseases, highlights the importance of early treatment of sickle cell disease.

“Sickle cell disease is a destructive nemesis. The keys to winning this life-long battle are information and inspiration. Information must be readily available, not only concerning the latest advances and pharmacological modalities, but also the familial genetic predisposition for the disorder. Also, our society as a whole needs inspiration. We must be inspired to take charge and fight for more research and funding for patients so they have a marked decrease of symptoms and a better quality of life,” explains Dr. Corey Hebert, Chief Medical Officer at Dillard University.

Children with sickle cell disease have abnormal red blood cells that are stiff and half-moon-shaped, causing them to become stuck in the blood vessels, blocking blood flow. These blockages cause crippling pain and organ damage that worsens over time and may ultimately lead to premature death.

Currently, the National Institutes of Health (NIH) recommend that sickle cell disease be treated as early as possible. That is why Medunik USA is proud to offer Siklos® (hydroxyurea), the first and only hydroxyurea-based sickle cell disease treatment with a pediatric indication. Siklos® is a prescription medicine that is used to reduce the frequency of painful crises and reduce the need for blood transfusions in children, 2 years of age and older, with sickle cell anemia with recurrent moderate to severe painful crises. Siklos® helps keep red blood cells round and flexible so they travel more easily through the blood vessels. This may help reduce painful crises and complications of the disease, including organ damage.

In addition to its unique pediatric indication, Siklos® tablets help doctors adjust dosing to each child’s body weight and can be dissolved in water for patients who have trouble swallowing them whole.

“Until a cure, other than very risky bone marrow transplants, is found, Medunik USA is dedicated to helping pediatric sickle cell disease patients live the healthier childhoods they deserve, said Tanya Carro, General Manager, Medunik USA.”

Siklos® (hydroxyurea) tablets, for oral use

WHAT IS SIKLOS®?

Siklos® is a prescription medicine that is used to reduce the frequency of painful crises and reduce the need for blood transfusions in 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.

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 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.
IMPORTANT SAFETY INFORMATION

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.

WHO SHOULD NOT TAKE SIKLOS®?
Do not take Siklos® if you are allergic to hydroxyurea or any of the ingredients in Siklos®. See the Medication Guide for a list 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®.
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:
See “What is the most important information I should know about Siklos®?”

Skin ulcers, including leg ulcers 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.
The most common side effects of Siklos® include:

Infections
Headache
Fever
Skin problems (e.g., skin reactions, dry skin, changes in skin and nail color)
Stomach and intestine (gastrointestinal) problems (e.g. nausea, constipation)
Decrease in vitamin D
Weight gain
These are not all the possible side effects of Siklos®.

You are encouraged to report negative side effects of prescription drugs to the FDA at www.fda.gov/medwatch, or 1-800-FDA-1088.

Please read the Full Prescribing Information, including Boxed Warning, Medication Guide and Instructions for Use, at www.siklosusa.com

About Medunik USA

Based in Pennsylvania, Medunik USA works to improve the health and quality of life of Americans living with rare diseases by making orphan drug therapies available in the United States. With strategic partnerships at the global level, the company has critical experience in approval and market access processes as well as the marketing of orphan drug therapies. Medunik USA makes critical medications to treat rare diseases available to American patients who might not otherwise have access to these medications. For more information, visit: www.medunikusa.com.

Contact:

Amy Feldman

Lazar Partners

1 212-867-1779

MedunikUSA@lazarpartners.com