Ipsen and Medison Pharma Announce Health Canada Approval of Bylvay(TM) (odevixibat) for the treatment of pruritus due to Progressive Familial Intrahepatic Cholestasis (PFIC)

Bylvay (odevixibat) is the first and only oral treatment available in Canada for the treatment of pruritus due to this rare genetic condition

ZUG, Switzerland and PARIS, Nov. 13, 2023 /PRNewswire/ -- Today, Ipsen (Euronext: IPN) (ADR: IPSEY), a global biopharmaceutical company focused on transformative medicines in oncology, rare disease and neuroscience, and Medison Pharma ("Medison"), a global pharma company focused on providing access to highly innovative therapies to patients in international markets, are pleased to announce the Health Canada approval for Bylvay™ (odevixibat) for the treatment of pruritus in patients aged 6 months or older with Progressive Familial Intrahepatic Cholestasis (PFIC), a progressive and life-threatening liver disease. Bylvay is the first medication approved in Canada for the treatment of pruritus in patients aged six months or older with PFIC.

Medison and Ipsen are part of a multiregional partnership in both Canada and Israel to bring Bylvay, a highly innovative therapy for the treatment of pruritus due to PFIC, offering new hope for patients suffering from this rare disease, and their families.

"At Medison, accelerating patients' access to innovative treatments is leading all that we do, so we are pleased and proud to bring Bylvay to patients in Canada," said Gil Gurfinkel, CEO at Medison Pharma. "We work hard, leveraging our multi-regional platform, to be the partner-of-choice for emerging biotech companies seeking to make their products available in international markets."

"Health Canada's authorization of Bylvay brings an important medicine to Canadians living with PFIC and their families," said Stewart Campbell, Executive Vice President and President of North America, Ipsen. "Bringing new medicines to new markets where unmet need exists illustrates Ipsen's mission to improve patient lives. We are pleased to work with Medison to get Bylvay to prescribers as soon as possible."

"I hope that the availability of Bylvay will immediately change the treatment paradigm for PFIC patients in Canada, providing physicians with a non-surgical option to help care for children with PFIC suffering from pruritus," said Dr. Binita Kamath, Division Head (interim), Gastroenterology, Hepatology and Nutrition, The Hospital for Sick Children in Toronto. "Being involved with the clinical investigation of odevixibat, I have firsthand experience with the value this therapy can offer families – including potentially better outcomes."

"Until today, surgery was the only treatment option available for PFIC patients," said Mr. Walter Perez, President, Progressive Familial Intrahepatic Cholestasis Advocacy and Resource Network and father of a child living with PFIC. "As families like mine know, managing symptoms of PFIC is very challenging. We are extremely pleased that a less invasive treatment option is now available."

About Bylvay™ (odevixibat)

Bylvay is a once-daily, non-systemic ileal bile acid transport inhibitor that acts locally in the small intestine and has minimal systemic exposure. It is approved in the U.S. for the treatment of pruritus in patients three months of age or older with PFIC, where it has orphan exclusivity. Bylvay was first launched as a treatment option for patients with PFIC in the U.S. in 2021, where it is supported by a program designed to assist with access to treatment and patient support. Bylvay also received regulatory approval in the E.U. for the treatment of PFIC in patients aged six months or older. It has launched in over nine countries and has secured public reimbursement across several major markets including Germany, Italy, the U.K., France and Belgium. In June 2023, Bylvay was also approved in the U.S. for the treatment of cholestatic pruritus in patients from 12 months of age with Alagille syndrome.

View full product monograph in Canadahere.

Important Safety Information

Indication

Bylvay™ (odevixibat) is indicated for the treatment of pruritus in patients aged 6 months or older with progressive familial intrahepatic cholestasis (PFIC).

Limitation of use: Bylvay may not be effective in a subgroup of PFIC type 2 patients with specific ABCB11 variants resulting in non-functional or complete absence of the bile salt export pump protein.

Contraindications

Odevixibat is contraindicated in patients who are hypersensitive to this drug or to any ingredient in the formulation, including any non-medicinal ingredient, or component of the container.

Most Serious Warnings and Precautions

Liver test abnormalities: In clinical trials, increased levels of liver enzymes and bilirubin were observed in some patients receiving Bylvay. Assessment of hepatic laboratory tests (alanine aminotransferase, aspartate aminotransferase, gamma-glutamyl transferase, alkaline phosphatase and total bilirubin) is recommended for all patients prior to initiating Bylvay, with monitoring per standard clinical practice.

Limited data are available in PFIC patients with cirrhosis or portal hypertension. Consider discontinuing Bylvay if a patient progresses to portal hypertension, cirrhosis or demonstrated hepatic decompensation.

Diarrhea has been reported as a common adverse reaction when taking Bylvay. Diarrhea may lead to dehydration. Patients should be monitored regularly to ensure adequate hydration during episodes of diarrhea. Interruption of treatment should be considered during acute episodes of diarrhea and/or vomiting that risk dehydration.

Fat-soluble vitamin deficiency: In clinical trials, decreased levels of fat-soluble vitamins A, D, E, and K (measured using international normalized ratio (INR)) and calcium were observed in some patients receiving Bylvay, as well as increased potassium levels. All observed decreases in calcium and increases in potassium were not considered to be clinically significant by the investigators.

Assessment of fat-soluble vitamin levels (Vitamins A, D, E), calcium, potassium and INR are recommended for all patients prior to initiating BYLVAY, with monitoring per standard clinical practice. Consider Bylvay discontinuation for fat-soluble-vitamin deficiency refractory to supplementation.

Other relevant warnings and precautions

• Conditions, medications, or surgical procedures that impair gastrointestinal motility, or enterohepatic circulation of bile acids
• Interaction with lipophilic medicinal products
• Pregnant women
• Women of child-bearing potential
• Nursing women
• Geriatrics - limited information in patients of 65 years

For more information

Please consult the Bylvay Product Monograph at https://www.medisonpharma.com/product-monographs/ for important information relating to adverse reactions, drug interactions and dosing information that has not been discussed in this piece. The Product Monograph is also available by calling 1-800-696-1341.

Bylvay does not require refrigeration and can be swallowed whole as a capsule, or opened and the pellets sprinkled onto soft food, or in liquid. This administration flexibility is a factor of importance for adherence in a pediatric patient population.

Health Canada's approval of Bylvay was supported by data from PEDFIC 1the largest, global, Phase 3 trial ever conducted in PFIC. In PEDFIC 1, a randomized, double-blind, placebo-controlled study, Bylvay met both its pruritus (p=0.004) and serum bile acid (p=0.003) primary endpoints and was well tolerated with low incidence of diarrhea/frequent bowel movements (9.5 per cent of treated patients vs. 5.0 per cent of placebo patients). Bylvay was well tolerated with diarrhea/frequent stools being the most common treatment-related gastrointestinal adverse events. There were no serious treatment-related adverse events.

About Progressive Familial Intrahepatic Cholestasis (PFIC)

PFIC is a group of inherited liver diseases resulting from mutations in genes that impact bile secretion that are progressive and life-threatening. Typical onset of PFIC is in infancy or early childhood with a hallmark feature of this condition being severe and debilitating pruritus (itching), which has been shown to considerably reduce quality of life and can result in the need for liver transplantation.[i], [ii]

The estimated global incidence of PFIC is one in 50,000 to 100,000 people worldwide,[iii] resulting in a designation of this condition as extremely or ultra rare.[iv]

About Medison Pharma

Medison is a global pharma company focused on providing access to highly innovative therapies to patients in international markets. Medison is the first to create an international commercialization platform for highly innovative therapies, helping to save and improve lives by making the best available novel treatments accessible to patients in international markets. Medison has a track record of multi-territorial partnerships with leading pharmaceutical and biotech companies seeking to expand their global reach. To learn more visit www.medisonpharma.com

About Ipsen

Ipsen is a global, mid-sized biopharmaceutical company focused on transformative medicines in Oncology, Rare Disease and Neuroscience. With total sales of €3.0bn in FY 2022, Ipsen sells medicines in over 100 countries. Alongside its external-innovation strategy, the Company's research and development efforts are focused on its innovative and differentiated technological platforms located in the heart of leading biotechnological and life-science hubs: Paris-Saclay, France; Oxford, U.K.; Cambridge, U.S.; Shanghai, China. Ipsen has around 5,400 colleagues worldwide and is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit ipsen.com

For additional information, please contact:

Medison Pharma
Inbal Chen, Corporate Communications
M: +972 54 440 0784
E: inbalc@medisonpharma.com

Ipsen Contacts

Investors

Canadian media inquiries:
Dara Willis, Gage Communications
M: 416-836-9272
E: dwillis@gagecommunications.ca

Ipsen's forward-looking statements

The forward-looking statements, objectives and targets contained herein are based on Ipsen's management strategy, current views and assumptions. Such statements involve known and unknown risks and uncertainties that may cause actual results, performance or events to differ materially from those anticipated herein. All of the above risks could affect Ipsen's future ability to achieve its financial targets, which were set assuming reasonable macroeconomic conditions based on the information available today. Use of the words 'believes', 'anticipates' and 'expects' and similar expressions are intended to identify forward-looking statements, including Ipsen's expectations regarding future events, including regulatory filings and determinations. Moreover, the targets described in this document were prepared without taking into account external-growth assumptions and potential future acquisitions, which may alter these parameters. These objectives are based on data and assumptions regarded as reasonable by Ipsen. These targets depend on conditions or facts likely to happen in the future, and not exclusively on historical data. Actual results may depart significantly from these targets given the occurrence of certain risks and uncertainties, notably the fact that a promising medicine in early development phase or clinical trial may end up never being launched on the market or reaching its commercial targets, notably for regulatory or competition reasons. Ipsen must face or might face competition from generic medicine that might translate into a loss of market share. Furthermore, the research and development process involves several stages each of which involves the substantial risk that Ipsen may fail to achieve its objectives and be forced to abandon its efforts with regards to a medicine in which it has invested significant sums. Therefore, Ipsen cannot be certain that favorable results obtained during preclinical trials will be confirmed subsequently during clinical trials, or that the results of clinical trials will be sufficient to demonstrate the safe and effective nature of the medicine concerned. There can be no guarantees a medicine will receive the necessary regulatory approvals or that the medicine will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Other risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and healthcare legislation; global trends toward healthcare cost containment; technological advances, new medicine and patents attained by competitors; challenges inherent in new-medicine development, including obtaining regulatory approval; Ipsen's ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of Ipsen's patents and other protections for innovative medicines; and the exposure to litigation, including patent litigation, and/or regulatory actions. Ipsen also depends on third parties to develop and market some of its medicines which could potentially generate substantial royalties; these partners could behave in such ways which could cause damage to Ipsen's activities and financial results. Ipsen cannot be certain that its partners will fulfill their obligations. It might be unable to obtain any benefit from those agreements. A default by any of Ipsen's partners could generate lower revenues than expected. Such situations could have a negative impact on Ipsen's business, financial position or performance. Ipsen expressly disclaims any obligation or undertaking to update or revise any forward-looking statements, targets or estimates contained in this press release to reflect any change in events, conditions, assumptions or circumstances on which any such statements are based, unless so required by applicable law. Ipsen's business is subject to the risk factors outlined in its registration documents filed with the French Autorité des Marchés Financiers. The risks and uncertainties set out are not exhaustive and the reader is advised to refer to Ipsen's latest Universal Registration Document, available on ipsen.com.

Logo - https://mma.prnewswire.com/media/1527224/MEDISON_Logo.jpg
Logo - https://mma.prnewswire.com/media/2274745/Ipsen_Logo.jpg

[i] Progressive Familial Intrahepatic Cholestasis Advocacy and Resource Network. Learn About PFIC Disease.https://www.pfic.org/learn-about-pfic-disease/. Accessed October 5, 2023.

[ii] Thompson, R., Arnell, H., et. al. Odevixibat treatment in progressive familial intrahepatic cholestasis: a randomized, placebo-controlled, phase 3 study. Lancet Gastroenterol Hepatol 2022; 7: 830–42.

[iii] Progressive Familial Intrahepatic Cholestasis Advocacy and Resource Network. Learn About PFIC Disease.https://www.pfic.org/learn-about-pfic-disease/. Accessed October 5, 2023.

[iv] Smith, C.I., Bergman, P., Hagey, D. Estimating the number of diseases – the concept of rare, ultra-rare, and hyper-rare. iScience. 2022; Aug 19; 25(8): 104698.

View original content to download multimedia:https://www.prnewswire.com/news-releases/ipsen-and-medison-pharma-announce-health-canada-approval-of-bylvay-odevixibat-for-the-treatment-of-pruritus-due-to-progressive-familial-intrahepatic-cholestasis-pfic-301986006.html

SOURCE Medison Pharma; Ipsen