Medunik USA 重申对 UCD 患者的承诺：Pheburane®（苯丁酸钠）持续供应

, /PRNewswire/ -- Medunik USA, a specialty pharmaceutical company dedicated to providing rare disease patients with access to orphan drugs, and part of Duchesnay Pharmaceutical Group (DPG), is pleased to reaffirm that as the treatment landscape for urea cycle disorders evolves following the announced discontinuation of another treatment option , Medunik USA remains committed to supporting the UCD community through the continued availability of Pheburane^® and its patient support services.

Medunik USA reaffirms its commitment to the UCD community

Pheburane^® is FDA-approved as a safe, effective treatment option for certain Urea Cycle Disorders (UCDs) used along with a specific diet for both adult and pediatric patients. Pheburane^® is an innovative palatable, taste-masked formulation of sodium phenylbutyrate consisting of very small coated oral pellets (about the size of sugar crystals). Pheburane^® is not indicated for the treatment of acute hyperammonemia. The most common side effects associated with sodium phenylbutyrate are menstrual dysfunction, decreased appetite, body odor and bad taste or taste aversion.¹

Urea cycle disorders are rare inherited metabolic conditions that impair the body's ability to eliminate ammonia, which can accumulate to toxic levels and potentially lead to serious health complications. Effective long-term disease management is critical for maintaining patient health and quality of life. Patients should talk with their healthcare provider about the treatment approach that best meets their individual needs.

"Patients living with urea cycle disorders rely on consistent access to treatment to help manage their condition," said Tanya Carro, Executive Vice President, U.S. Entities, DPG. "We want patients, caregivers, and healthcare providers to know that Pheburane^® continues to be available, and our team is ready to support those who may be impacted by changes in the treatment landscape."

In addition to providing access to Pheburane^®, Medunik USA offers the Unik Patient Support Program, which provides one-on-one personalized educational assistance, copay/financial support programs, and other resources for patients and families.

Beyond product availability and tailored patient support program, Pheburane^® is supported by broad payer coverage across the United States. Pheburane^® is covered under Preferred Medicaid Programs in 19 states, while 29 states provide coverage through Medicaid programs with limited or no restrictions. The therapy is also reimbursed under commercial insurance plans that collectively reach approximately 80% of the commercially insured U.S. population.

"At a time when treatment choices for the UCD community are becoming more limited, maintaining access to a proven therapy is more important than ever," said David McLaughlin, Vice President, Sales & Marketing, U.S. Entities, DPG. "The combination of broad insurance coverage, patient assistance programs, and dedicated clinical support reflects our commitment to ensuring that patients living with UCDs can continue to access the treatment and resources they need."

With an estimated 1 in 35,000 people in the United States affected by urea cycle disorders, access to treatment remains a critical component of patient care. The combination of broad Pheburane^® coverage under Medicaid and commercial insurance plans, together with Medunik USA's comprehensive Unik Patient Support Program, helps address barriers to treatment and supports continuity of care for patients and families affected by these rare metabolic disorders.

Additional information about Pheburane^® and the Unik Patient Support Program is available at: https://pheburane.com/.

ABOUT UREA CYCLE DISORDERS (UCDs)

The main purpose of the Urea Cycle is to eliminate toxic ammonia from the blood and make urea, which is then excreted as urine. UCDs are rare genetic disorders that cause errors in this process, allowing high levels of ammonia, the key marker for UCDs, to build up in the bloodstream, potentially to dangerous and fatal levels. 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^®:

sleepiness
tiredness
lightheadedness
vomiting
nausea
headache
confusion

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:

absent or irregular menstrual periods
decreased appetite
body odor
bad taste or avoiding foods that you ate prior to getting sick (taste aversion)

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 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. Through its strategic partnerships, Medunik USA develops and provides Americans suffering from rare disease with access to orphan drugs that are not currently available in the U.S. 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 about Medunik USA, please visit www.medunikusa.com.

ABOUT DUCHESNAY PHARMACEUTICAL GROUP

Duchesnay Pharmaceutical Group (DPG), with its affiliated companies, is headquartered in Blainville, Quebec. The Group consists of six pharmaceutical companies to meet the needs of patients in Canada, the U.S. and abroad. The companies are Duchesnay (Canada) and Duchesnay USA, both dedicated to women's health; Médunik Canada and Medunik USA, which provide treatments for rare and debilitating diseases; and Analog Pharma Canada and Analog Pharma, specializing in orphan generic medications. From its state-of-the-art manufacturing plant, DPG exports its innovative treatments to more than 50 countries.

DPG is one of the eight companies across the country chosen to participate in the Government of Canada's Global Hypergrowth Project. This appointment offers exclusive and personalized support for at least two years, in order to accelerate its growth to become an anchor firm in the Canadian economy.

DPG is the winner of the 2024 Life Sciences Innovation Award by ADRIQ, the Association for the Development of Research and Innovation of Quebec, which recognizes DPG's healthy workplace culture and commitment to pharmaceutical innovation, while DPG president Éric Gervais is the recipient of the 2024 Bernard-Landry Award by ADRIQ which acknowledges his impactful leadership on Quebec's research and innovation ecosystem.

DPG, through its proprietary research and development, and through exclusive partnerships, offers innovative treatments for a variety of medical conditions in women's health, urology, oncology and for rare diseases, plus lower-cost generic medications. DPG 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 to giving back to the community through the support of various charitable organizations.

For more information, please visit https://duchesnaypharmaceuticalgroup.com/en.

References

Pheburane^® (sodium phenylbutyrate) oral pellets [Prescribing Information]. Medunik USA, Inc.
Cleveland Clinic, Urea Cycle Disorder, https://my.clevelandclinic.org/health/diseases/23470-urea-cycle-disorder

SOURCE Medunik USA