PRINCETON, NJ, Sept. 06, 2022 (GLOBE NEWSWIRE) -- Medunik USA is pleased to announce the availability of Pheburane® oral pellets, a unique taste-masked formulation of sodium phenylbutyrate (NaPB).
Pheburane® is a prescription medicine, used along with a specific diet, for the long-term management of adults and children with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC) or argininosuccinate synthetase (AS). Pheburane® is not used for the treatment of acute hyperammonemia, which can be life-threatening and requires emergency medical treatment.1
UCDs are rare, chronic, genetic conditions that can be fatal if left untreated and can impact children from the time of birth. UCDs disrupt the body’s urea cycle, and therefore, the body is unable to remove the dangerous buildup of toxic chemicals, particularly ammonia, that are created from the digestion of protein. UCDs of different levels of severity affect one in 35,000 people in the United States or about 28 per one million residents.2 It is estimated that approximately 2,000 people in the U.S. require treatments with NaPB products such as Pheburane® for their UCD.
Pheburane®, a proven therapy which has been available to patients for many years in Europe and Canada, delivers NaPB in the form of very small, coated pellets to help mask the awful taste of NaPB, which may influence whether or not patients will consistently take their treatment regimen, an important feature in order to avoid the serious and life-threatening complications related to the disease.3
This innovative taste-masked formulation of NaPB will be priced similarly to other NaPB products currently on the market and at a significant discount in cost to the most commonly prescribed UCD treatment.4
The most common side effects of Pheburane® include absent/irregular menstrual periods, decreased appetite, body odor, and bad taste or avoiding foods that you ate prior to getting sick (taste aversion).
Pheburane® is measured with a reusable calibrated dosing spoon, provided in each package, and does not require any pills, mixing or oral syringes like other forms of NaPB. Pheburane® can be taken with water, fruit juice or protein-free infant formulas, or sprinkled on a spoonful of apple sauce or carrot puree, and taken with a snack or meal.
“Medunik USA is very proud to make Pheburane® available in the United States,” said Tanya Carro, Executive Vice-President of Medunik USA. “This is an important milestone which reflects our ongoing commitment to improving the health of patients suffering from rare diseases as well as potentially reducing the cost of UCD treatment for many patients as well as for private and government payers.”
Medunik USA offers Pheburane® through its UNIK Support Program – designed to support each unique patient. UNIK Support offers specialized services including a copay savings program, patient care liaison services, mail order pharmacy and other support services. Medunik USA provides Pheburane® and all its added benefits at a similar cost to other NaPB products. Visit Pheburane.com for more information.
Pheburane® pharmacy services in the United States are provided exclusively through Accredo, Evernorth’s specialty pharmacy. Accredo also has reimbursement specialists available to advise about insurance coverage through Medicaid, Medicare and private plans.
About urea cycle disorders (UCDs)
The urea cycle is a process which allows the body to keep and use positive contents of food and filter and remove any toxic substances that can be ingested or created. UCDs are rare genetic disorders that cause errors in this process, allowing toxic substances to build up in the bloodstream, potentially to dangerous and fatal levels. The key marker for UCDs is high levels of ammonia. Removal of ammonia from the bloodstream normally occurs via its conversion to urea, which is then excreted by the kidneys. Ammonia is extremely toxic, particularly to the central nervous system. UCDs can cause catastrophic illness in newborns within 36 to 48 hours of birth despite the infants appearing normal, so they can be discharged from hospital before signs of UCDs develop. UCDs require lifelong monitoring and treatment.2
About Pheburane®
Pheburane® is a taste-masked oral formulation of sodium phenylbutyrate approved by the Food and Drug Administration as adjunctive therapy to standard of care, which includes dietary management, for the chronic management of adult and pediatric patients with UCDs, involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC) or argininosuccinic acid synthetase (AS). Pheburane® is not indicated for the treatment of acute hyperammonemia.1
Pheburane®, developed by Lucane Pharma, is under exclusive distribution in the U.S. through Medunik USA. For further information, visit Pheburane.com.
INDICATION AND IMPORTANT SAFETY INFORMATION
What is Pheburane®?
- Pheburane® is a prescription medicine, used along with a specific diet, for the long-term management of adults and children with urea cycle disorders (UCDs), involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC) or argininosuccinate synthetase (AS).
- Episodes of sudden, rapid increase of ammonia in the blood (acute hyperammonemia) may happen in people during treatment with Pheburane®. Pheburane® is not used for the treatment of acute hyperammonemia, which can be life-threatening and requires emergency medical treatment.
Before taking Pheburane®, tell your healthcare provider about all of your medical conditions, including if you:
- have heart problems.
- have kidney or liver problems.
- have diabetes (Pheburane® contains sucrose), or have a history of fructose intolerance, glucose-galactose malabsorption, or sucrose-isomaltase insufficiency.
- are pregnant or plan to become pregnant. It is not known if Pheburane® will harm your unborn baby.
- are breastfeeding or plan to breastfeed. It is not known if Pheburane® passes into your breastmilk. Talk to your healthcare provider about the best way to feed your baby during treatment with Pheburane®.
Tell your healthcare provider about all the medicines you or your child take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
Certain medicines may increase the level of ammonia in your blood or cause serious side effects when taken during treatment with Pheburane®. Especially tell your healthcare provider if you or your child take:
- corticosteroids
- valproic acid
- haloperidol
- probenecid
Know the medicines you take. Keep a list of them to show your or your child’s healthcare provider and pharmacist when you get a new medicine.
What are the possible side effects of Pheburane®?
Pheburane® can cause serious side effects, including:
Nervous system problems (neurotoxicity). Call your healthcare provider right away if you get any of the following symptoms during treatment with Pheburane®:
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Low potassium levels in your blood (hypokalemia). Your healthcare provider will monitor your blood potassium levels during treatment with PHEBURANE and treat if needed.
Conditions related to swelling (edema). Pheburane® contains salt (sodium), which can cause swelling from salt and water retention. Your healthcare provider will decide if PHEBURANE is right for you if you have certain medical conditions that can cause swelling, such as heart failure, liver problems or kidney problems.
The most common side effects of Pheburane® include:
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Your healthcare provider may do certain blood tests to check you or your child for side effects during treatment with Pheburane®.
These are not all the possible side effects of Pheburane®.
Call your doctor for medical advice about side effects.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
Please read the Full Prescribing Information and Patient Information at Pheburane.com.
About Medunik USA
Based in Princeton, New Jersey, Medunik USA is part of the Duchesnay Pharmaceutical Group and 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.
About Duchesnay Pharmaceutical Group
Duchesnay Pharmaceutical Group, with its affiliated companies, is headquartered in Blainville, Quebec. The group consists of five pharmaceutical companies to meet the needs of patients in Canada, the U.S., and abroad. The companies are Duchesnay and Duchesnay USA, both dedicated to women's health; Médunik Canada and Medunik USA, which provide treatments for rare diseases; and Analog Pharma, an American generic drugs company, specializing in authorized generics and orphan drugs. From its state-of-the-art manufacturing plant, Duchesnay can export its innovative treatments to more than 50 countries.
Duchesnay and Medunik, through their proprietary research and development, and through partnerships, offer innovative treatments for a variety of medical conditions in women's health, urology, oncology as well as for rare diseases. The group of companies recognizes the dedication and professionalism of its employees and promotes a positive culture and flexible work environment. It is deeply committed to environmental responsibility and giving back to the community through the support of various charitable organizations. For more information, please visit duchesnay.com, medunik.com and analogpharma.com.
References
1. Pheburane® (sodium phenylbutyrate) oral pellets [Prescribing Information]. Medunik USA, Inc.
2. Cleveland Clinic, Urea Cycle Disorder, https://my.clevelandclinic.org/health/diseases/23470-urea-cycle-disorder
3. Guffon N, Kibleur Y, Copalu W, Tissen C, Breitkreutz J. Developing a new formulation of sodium phenylbutyrate. Arch Dis Child. 2012 Dec;97(12):1081-5. doi: 10.1136/ archdischild-2012-302398. Epub 2012 Aug 31. PMID: 22941860.
4. Medi-Span PriceRx database using usual and maximum daily doses and current WAC prices
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