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Ipsen's Alagille Drug Backed By CHMP Again, With New Name

In unusual circumstances, Ipsen has been given the green light for its odevixibat drug for severe itching (pruritus) in patients with rare liver disease Alagille syndrome (ALGS) in the EU – for the second year running.

The once-daily, non-systemic ileal bile acid transport (IBAT) inhibitor was recommended for approval by the EMA's human medicines committee, the CHMP, in July 2023 for this indication under the Bylvay brand. It is already approved in the EU under that name for progressive familial intrahepatic cholestasis (PFIC), another rare disease affecting the liver.

That recommendation came however without the orphan status that can often be the key to unlocking reimbursement decisions in EU member states and also provides 10 years of marketing exclusivity after approval by the European Commission.

Ipsen was unable to convince the EMA that Bylvay qualified for orphan status – in other words, that it addresses a disease that is life-threatening or chronically debilitating and affects less than five in 10,000 patients – given the number of patients targeted under that brand.

Its solution was to refile the drug under a different brand name and now, a year later, odevixibat has a second positive opinion from the CHMP for ALGS under the new Kayfanda brand. Its orphan status remains up in the air, however, with a final decision still pending from the Commission.

ALGS is caused by a mutation in the JAG1 gene that leads to liver damage and jaundice. Itching is widely acknowledged to be one of the most debilitating symptoms of the disease, particularly in young children, and is one of the main drivers for patients to seek a liver transplant.

In the ASSERT trial – which Ipsen says is the world's first and only phase 3 trial completed in patients with ALGS – odevixibat achieved statistically significant and "clinically meaningful" improvements in scratching severity compared to placebo over six months.

The French drugmaker acquired rights to odevixibat when it bought Albireo last year in a $42-per-share deal worth around $950 million.

Previous estimates have suggested that the drug could make sales over $1 billion if it gets approval across PFIC, ALGS, and follow-up indication biliary atresia (BA). It was approved for the ALGS indication by the FDA, as Bylvay, in June 2023 and has orphan status in the US market.

Iqirvo for primary biliary cholangitis

In another uncommon event, Ipsen also picked up a second CHMP recommendation at its latest meeting, this time for Iqirvo (elafibranor), a drug for primary biliary cholangitis (PBC).

The panel gave its blessing to the use of the drug in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as a monotherapy in patients unable to tolerate UDCA.

PBC is a chronic, autoimmune disease in which bile ducts in the liver are gradually destroyed, leading to cirrhosis, and affects around 100,000 people in the US, mainly women, with its prevalence on the rise. It causes debilitating fatigue and pruritus and, if left untreated, can progress to serious liver disease requiring a transplant.

Iqirvo, a dual PPAR alpha/delta agonist, has been shown to reduce levels of biomarkers for PBC in clinical trials, with a trend towards reduced itching. It was approved in the US in June and competes with Intercept Pharma's Ocaliva (obeticholic acid) in the PBC market.

Ipsen acquired rights to the drug from Genfit in 2021 in return for an upfront cash payment of €120 million and up to €360 million in potential milestones after a phase 2 study showed potential as a treatment for PBC. Analysts have suggested that the market for drugs to treat UDCA-refractory PBC could be worth $1.5 billion or more per year.

The drug was previously tested for metabolic dysfunction-associated steatohepatitis (MASH), but failed a phase 3 trial and was abandoned by Genfit for that indication.

"We are delighted to have received CHMP positive opinions for two potential new medicines in rare cholestatic liver diseases, on the same day," said Ipsen's head of R&D Christelle Huguet, who called it "a rare achievement, and one that demonstrates our commitment to addressing the unmet medical needs in these diseases."

LIVMARLI Now Approved In Japan For ALGS And PFIC

- LIVMARLI is the first and only treatment available for cholestatic pruritus in patients with Alagille syndrome and Progressive Familial Intrahepatic Cholestasis in Japan

FOSTER CITY, Calif., March 27, 2025--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that its partner, Takeda Pharmaceutical Company Limited, has received approval by the Japanese Ministry of Health, Labour, and Welfare for LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) in Japan.

"We are thrilled to see LIVMARLI approved as the first and only medication available in Japan for patients living with pruritus related to ALGS and PFIC," said Chris Peetz, chief executive officer at Mirum. "Takeda is a leading global pharmaceutical company with demonstrated success in the development and commercialization of medicines for rare diseases. Under Takeda's leadership, we are confident that LIVMARLI could have a meaningful impact on the lives of patients with ALGS and PFIC in Japan."

Mirum and Takeda entered into an exclusive license agreement in September 2021 for the development and commercialization of LIVMARLI for rare cholestatic pediatric liver diseases in Japan. Under the terms of the agreement, Mirum is eligible to receive a percentage of Takeda's annual net sales.

LIVMARLI is currently approved for the treatment of pruritus related to ALGS in more than 40 countries and for PFIC in more than 30 countries worldwide.

About LIVMARLI® (maralixibat) oral solution

LIVMARLI® (maralixibat) oral solution is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration and European Medicines Agency for two pediatric cholestatic liver diseases. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. Three months of age and older and in Europe for patients two months of age and older. It is also approved in the U.S. For the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. For more information for U.S. Residents, please visit LIVMARLI.Com.

LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum's clinical trials section on the company's website.

U.S. IMPORTANT SAFETY INFORMATION

Limitation of Use: LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein.

LIVMARLI can cause side effects, including:

Liver injury. Changes in certain liver tests are common in patients with Alagille syndrome and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In PFIC, this can be serious or may lead to liver transplant or death. Your healthcare provider should do blood tests and physical exams before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen), bloating in your stomach area, loss of appetite or bleeding or bruising more easily than normal.

Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain. Your healthcare provider may advise you to monitor for new or worsening stomach problems including stomach pain, diarrhea, blood in your stool or vomiting. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you.

A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat is common in patients with Alagille syndrome and PFIC but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects.

US Prescribing Information EU SmPC Canadian Product Monograph

About Mirum Pharmaceuticals, Inc.

Mirum Pharmaceuticals, Inc. Is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution, CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets.

LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. In cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum is also initiating the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults.

Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder.

To learn more about Mirum, visit mirumpharma.Com and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X).

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the impact of LIVMARLI on patients in Japan, the ability of Takeda to successfully commercialize in the territory and the size of the Japanese market for LIVMARLI. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "will," "could," "can," "would," "potential," "hope," "opportunity," and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum's business in general, the impact of macroeconomic and geopolitical developments, and the other risks described in Mirum's filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. A further description of risks and uncertainties can be found in Mirum's Quarterly Report on Form 10-K for the quarter ended December 31, 2024 and subsequent filings with the U.S. Securities and Exchange Commission and available at www.Sec.Gov.

View source version on businesswire.Com: https://www.Businesswire.Com/news/home/20250327866446/en/

Contacts

Media Contact:Erin Murphymedia@mirumpharma.Com

Investor Contact:Andrew McKibbenir@mirumpharma.Com

NI Woman On Her Incredible Journey Of Receiving Three Life-saving Transplants

A Co Down woman has opened up on her incredible journey of receiving three life-saving transplants.

Portaferry woman Megan Moore who was born with Alagille Syndrome received a liver and two kidneys.

Alagille Syndrome is a rare genetic disorder affecting multiple organs, including the heart, liver, kidneys and eyes. The condition can cause significant liver damage, structural heart abnormalities and other health complications.

READ MORE: NI woman's warning of common signs after 'healthy dad's' diagnosis

READ MORE: How NI woman's incredible gift changed her brother's life

Megan's life-saving transplant journey began at just six years old when she underwent a liver transplant in 2000.

Fourteen years later, in 2014, she received her first kidney transplant and in June 2024, she underwent a second kidney transplant thanks to a living donor, her aunt.

The Co Down woman said: "Due to my condition, I unfortunately needed a liver transplant when I was six years old. I was feeling okay afterwards, although I did have some complications.

Megan Moore received three life-saving transplants (Image: South Eastern Trust)

"I know my mum and dad were told when I was born that I probably wouldn't make it past a certain age - multiple times.

"When I was fourteen, I required my first kidney transplant and in June 2024, I had my second kidney transplant, which my aunt very kindly donated to me."

Megan recalled the moment she received the life-changing phone call that a liver was available. She told: "It was just before Christmas and my family was with me. I didn't know what to expect. When I got the call, I just sat on the stairs and cried. I was only six years old at the time."

Years later, as a teenager facing the need for a kidney transplant, Megan said: "I felt scared, but having been through a transplant before, I knew what was going to happen and I could deal with it. My daddy had just passed away at the time as well, so it was all very overwhelming."

When doctors informed Megan that she needed a second kidney transplant, her family stepped up. She explained: "Quite a few members of my family volunteered to be tested. My Aunt was the final match and we went through the living donor programme.

Megan and her cousin Grace (Image: South Eastern Trust)

"When I realised she was a match, I cried because I didn't think I would make it to my 30th birthday. There was sunshine at the end of the tunnel. Everybody was so happy for me!"

Expressing her thanks to the medical team who supported her, Megan said: "The Renal Unit staff at the Ulster Hospital are just fantastic. They helped me so much, especially, going through dialysis and getting the transplant while also dealing with my Dad's passing."

Now, Megan is encouraging others to consider organ donation: "Organ donation can change somebody else's life for the better. It gives them another lease of life and gives their family hope. It's the best thing to do and I hope that by sharing my story, more people will be inspired to consider becoming donors."

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