Sodium Phenylbutyrate

PRINCETON, NJ, June 4, 2024 /PRNewswire/ - Medunik USA, member of Duchesnay Pharmaceutical Group (DPG) one of the few anchor companies selected by the Government of Canada for its Global Hypergrowth Project, is pleased to announce the expansion of its UNIK Support Program specialty pharmacy network to now include both Accredo Health Group and CVS Specialty. UNIK Support offers specialized services to benefit patients including a copay savings program, patient care liaison services, mail order pharmacy and other support services. In addition, Arkansas has recently added Pheburane® to its Medicaid Preferred Drug List. As a result, 48 State Medicaid programs now cover Pheburane®, including 12 State Medicaid programs that have included Pheburane® as a preferred covered agent for the treatment of certain UCD patients. Additionally, ~80% of commercially insured Americans have access to Pheburane® coverage, of which more than 75% is preferred coverage. Continue Reading Medunik USA’s UNIK Support Program for Pheburane® expands to now include 2 specialty pharmacy options (CNW Group/Medunik USA) Pheburane® is an innovative adjunctive therapy consisting of very small coated oral pellets (about the size of sugar crystals) for the chronic management of adult and pediatric patients with UCDs involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC) or argininosuccinate synthetase (AS). 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 Available in Europe since 2013 and in Canada since 2015, Pheburane® has a proven history in the treatment of UCDs at an international level. Medunik USA launched Pheburane® in the USA in September 2022 and since then has significantly widened access to this important treatment option through strategic alliances and cost-effective pricing policies. The widespread preferred coverage for Pheburane® reflects its value proposition and potential to reduce costs within the healthcare ecosystem by 50-70% relative to other taste-masked/nearly tasteless UCD medications*. "The expanded Pheburane® coverage and affordable pricing underscore our commitment to providing Americans with rare diseases access to effective, fairly priced medicines, thus contributing to improving the lives of those whose needs are too often neglected," said Claude-André Boivin, Director, Strategic Pricing and Business Insights, Duchesnay Pharmaceutical Group. 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. One in 35,000 people in the United States or about 28 per one million residents suffer from UCDs of different levels of severity.2 Medunik USA makes Pheburane® available through its UNIK Support Program designed to support each unique patient. Visit Pheburane.com for more information. 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 , 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. 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, visit . 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; Medunik Canada and Medunik USA, which provide treatments for rare 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 8 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 2 years, in order to accelerate our growth to become an anchor firm in the Canadian economy. Duchesnay Pharmaceutical Group, 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 . References SOURCE Medunik USA

Pictured: Biogen's signage at its headquarters in Massachusetts/iStock, JHVEPhoto Biogen and Ionis Pharmaceuticals on Thursday revealed early results from the Phase I/II ALSpire study, demonstrating that their investigational antisense oligonucleotide BIIB105 failed to significantly lower plasma levels of a neurodegeneration biomarker, or improve clinical outcomes, in amyotrophic lateral sclerosis patients. Given the disappointing outcome, the partners have elected to discontinue the development of BIIB105. ALSpire was a multiple-ascending-dose trial designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal BIIB105 in adult patients with amyotrophic lateral sclerosis (ALS). The study consisted of a six-month placebo-controlled study, followed by a three-year, long-term, open-label extension phase. In Thursday’s announcement, Biogen and Ionis revealed that the antisense oligonucleotide was unable to significantly reduce the levels of plasma neurofilament light chain (NfL), a known indicator of nerve damage and neurodegeneration. BIIB105 also showed no meaningful clinical impact on patient function, breathing and strength. Data from ALSPire’s open-label phase were similar to its initial six-month outcomes, with BIIB105 having no significant impact on NfL or clinical measures. While BIIB105 did significantly reduce levels of the ataxin-2 (ATXN2) protein in patients’ cerebrospinal fluid, this was not enough for Biogen. Stephanie Fradette, head of the company’s neuromuscular development unit, said in a statement that Thursday’s readout “gives us confidence that BIIB105 did not slow the disease process.” Biogen and Ionis will continue to evaluate ALSpire’s data to better understand the “underlying disease process and effects of BIIB105.” The companies will present their analysis at the upcoming European Network to Cure ALS meeting in June 2024. William Blair analyst Myles Minter in an investor note said that the outcome of ALSpire was “obviously disappointing,” though not surprising. “We have maintained the view that the program was interesting but highly risky given known challenges in developing treatments for sporadic ALS and lack of a clear human genetic link for ATXN2,” Minter wrote, adding that BIIB105’s discontinuation adds to the setbacks in the ALS field, following the withdrawal of Amylyx’s Relyvrio (sodium phenylbutyrate and taurursodiol) in April 2024. Biogen on Thursday also announced that it would not exercise its option to license and take charge of the development of another Ionis antisense oligonucleotide candidate, dubbed BIIB121, for the treatment of Angelman syndrome. The announcement comes after Ionis posted positive Phase I/IIa data for BIIB121, also known as ION582, showing that the investigational treatment improved cognition, communication and motor function in patients. “While it is difficult to speculate the exact rationale for Biogen’s decision, particularly without the full dataset, Biogen has previously emphasized plans to reprioritize its early-stage R&D efforts, so we are not particularly surprised by Thursday’s update,” Minter wrote in the investor note, adding that ION582 is a “natural fit” for Ionis, given the biotech’s “focus on rare neurological diseases.” Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.