Mirum to host an investor call on September 29,
2021 at 4:30 p.m. ET
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leader in rare
liver disease, today announced that the U.S. Food and Drug
Administration (FDA) has approved LIVMARLI™ (maralixibat) oral
solution for the treatment of cholestatic pruritus in patients with
Alagille syndrome (ALGS) one year of age and older. LIVMARLI, a
minimally absorbed ileal bile acid transporter (IBAT) inhibitor, is
the first and only FDA-approved medication in this rare liver
disease which affects 2,000 to 2,500 children in the United States.
LIVMARLI is now available for prescribing. In conjunction with the
approval, Mirum received a rare pediatric disease priority review
voucher.
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www.livmarli.com
“Children with Alagille syndrome suffer from cholestatic
pruritus, which is serious, unremitting, and debilitating. Their
sleep is disrupted, and they endure bleeding and scarring of the
skin due to unrelenting scratching,” said Binita M. Kamath,
MBBChir, Pediatric Hepatologist, The Hospital for Sick Children
(SickKids), Toronto, Ontario, Canada. “There have been no approved
treatments to date for cholestatic pruritus in Alagille syndrome,
and many children ultimately require major surgical interventions
such as liver transplantation for refractory pruritus. The approval
of LIVMARLI signifies a meaningful shift in the treatment paradigm
for Alagille syndrome and provides hope for the many families who
have lived with persistent itch for far too long.”
ALGS is a rare genetic disorder caused by abnormalities in bile
ducts that can lead to progressive liver disease. Malformed or
reduced bile ducts cause cholestasis, the accumulation of bile
acids in the liver, which leads to inflammation and liver injury,
and prevents the liver from working properly. Cholestasis in ALGS
is associated with pruritus which is among the most common
indications for liver transplant in ALGS.
The approval of LIVMARLI is based on the pivotal ICONIC study as
well as five years of data from supportive studies resulting in a
robust body of evidence in 86 patients with ALGS. Data from ICONIC
demonstrated statistically significant reductions in pruritus, one
of the most common and arduous symptoms associated with the
disease, which was maintained through four years.
“Today is a great day for the Alagille syndrome community with
the approval of a much-needed new treatment option to address one
of the most debilitating effects of this disease,” said Chris
Peetz, president and chief executive officer of Mirum. “We are
grateful to the patients, families, and healthcare professionals
who advanced the research and participated in the LIVMARLI clinical
studies. Today is also a landmark day for Mirum as we take steps
forward in developing potentially life-changing medicines for rare
liver disease.”
“We have had the pleasure of being part of and closely following
the clinical progress of LIVMARLI in many ways. Since the first
study’s initiation more than a decade ago, we have dreamed of
today, seeing LIVMARLI receive FDA approval, marking an incredibly
meaningful milestone for the ALGS community,” said Roberta Smith,
president, Alagille Syndrome Alliance and an ALGS mom. “Until now,
patients have had limited-to-no treatment options to address the
severe and unrelenting itch that significantly impacts both
patients and their families. Additionally, because pruritus
associated with ALGS greatly impacts caregivers, having a strong
support program like Mirum Access Plus to reduce the strain on
families is so important. The ALGS community has been waiting for a
long time for a treatment and we’re so pleased that LIVMARLI is now
available in the United States.”
Mirum Access Plus (MAP)
LIVMARLI will be accessible to patients with a prescription
through Mirum Access Plus (MAP), the company’s patient support
services program and single-source specialty pharmacy. The MAP
program has fully dedicated and experienced coordinators who will
work with healthcare providers and families to provide insurance
coverage and access support, as well as help with financial support
options for LIVMARLI. A dedicated Navigator team will also provide
health education and will connect families to resources and tools
to support their disease. MAP staff are available Monday through
Friday, 8:00 a.m. to 8:00 p.m. ET by calling 855-MRM-4YOU
(1-855-676-4968) or more information, visit www.livmarli.com.
Patients Enrolled in the U.S. Expanded Access Program
For patients who are currently enrolled in the U.S. Expanded
Access Program (EAP), MAP coordinators will assist patients who
wish to continue on LIVMARLI with the conversion to prescription
LIVMARLI. The EAP will remain open for eligible patients with ALGS
in Australia, Canada, the UK and several countries in Europe until
LIVMARLI is approved in the respective country. For more
information about the maralixibat EAP, visit algseap.com.
Investor Conference Call
Mirum will host a conference call to discuss LIVMARLI’s approval
in further detail today, September 29, at 4:30 p.m. ET. The live
webcast and archived event will be available within the Events
section on Mirum’s website. To participate in the live call, dial
(844) 200-6205 (toll free) or (646) 904-5544 (international) and
use the passcode 986568.
About LIVMARLI™ (maralixibat) oral solution
LIVMARLI™ (maralixibat) oral solution is an orally administered,
once-daily, ileal bile acid transporter (IBAT) inhibitor approved
by the U.S. Food and Drug Administration for the treatment of
cholestatic pruritus in patients with Alagille syndrome one year of
age and older. For more information, please visit LIVMARLI.com.
LIVMARLI is the only FDA-approved medication to treat
cholestatic pruritus associated with Alagille syndrome.
LIVMARLI is currently being evaluated in late-stage clinical
studies in other rare cholestatic liver diseases including
progressive familial intrahepatic cholestasis and biliary atresia,
of which both have received Breakthrough Therapy designation and
Orphan Drug designation. To learn more about ongoing clinical
trials with LIVMARLI, please visit Mirum’s clinical trials section
on the company’s website.
About Alagille syndrome
Alagille syndrome (ALGS) is a rare genetic disorder in which
bile ducts are abnormally narrow, malformed and reduced in number,
which leads to bile accumulation in the liver and ultimately
progressive liver disease. The estimated incidence of ALGS is one
in every 30,000 people.1 In patients with ALGS, multiple organ
systems may be affected by the mutation, including the liver,
heart, kidneys and central nervous system.2 The accumulation of
bile acids prevents the liver from working properly to eliminate
waste from the bloodstream and, according to recent reports, 60% to
75% of patients with ALGS have a liver transplant before reaching
adulthood.3 Signs and symptoms arising from liver damage in ALGS
may include jaundice (yellowing of the skin), xanthomas
(disfiguring cholesterol deposits under the skin), and pruritus
(itch)2. The pruritus experienced by patients with ALGS is among
the most severe in any chronic liver disease and is present in most
affected children by the third year of life.4
IMPORTANT SAFETY INFORMATION
LIVMARLI can cause serious side effects, including:
Changes in liver tests. Changes in certain liver tests
are common in patients with Alagille syndrome and can worsen during
treatment with LIVMARLI. These changes may be a sign of liver
injury and can be serious. Your healthcare provider should do blood
tests 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) or loss of
appetite.
Stomach and intestinal (gastrointestinal) problems.
LIVMARLI can cause stomach and intestinal problems, including
diarrhea, stomach pain, and vomiting during treatment. 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. FSV deficiency is common in patients with
Alagille syndrome but may worsen during treatment. Your healthcare
provider should do blood tests before starting and during
treatment.
Other common side effects reported during treatment were bone
fractures and gastrointestinal bleeding.
Prescribing information
About Mirum Pharmaceuticals, Inc.
Mirum Pharmaceuticals, Inc. is a biopharmaceutical company
dedicated to transforming the treatment of rare liver diseases.
Mirum’s approved medication is LIVMARLI™ (maralixibat) oral
solution which is approved in the U.S. for the treatment of
cholestatic pruritus in patients with Alagille syndrome one year of
age and older.
Mirum’s late-stage pipeline includes two investigational
treatments for debilitating liver diseases affecting children and
adults. Maralixibat (LIVMARLI), an oral ileal bile acid transporter
(IBAT) inhibitor, is currently being evaluated in clinical trials
for pediatric liver diseases and includes the MARCH Phase 3 study
for progressive familial intrahepatic cholestasis (PFIC) and the
EMBARK Phase 2b study for patients with biliary atresia. In
addition, Mirum has an expanded access program open in Canada,
Australia, the UK and several countries in Europe for eligible
patients with Alagille syndrome.
Mirum has submitted a Marketing Authorization Application to the
European Medicines Agency for maralixibat for the treatment of
cholestatic liver disease in patients with Alagille syndrome.
Mirum’s second investigational treatment, volixibat, also an
oral IBAT inhibitor, is being evaluated in two registrational
studies including the OHANA Phase 2b study for pregnant women with
intrahepatic cholestasis of pregnancy and the VISTAS Phase 2b study
for adults with primary sclerosing cholangitis. Mirum is planning
to launch a Phase 2b study in primary biliary cholangitis later
this year.
To augment its pipeline in cholestatic liver disease, Mirum has
acquired the exclusive option to develop and commercialize gene
therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2,
respectively, from Vivet Therapeutics SAS, following preclinical
evaluation and investigational new drug-enabling studies.
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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 commercialization plans and
expectations for commercializing LIVMARLI in the United States,
estimates of the number of patients impacted by ALGS and who are
appropriate for treatment with LIVMARLI, the potential benefits or
competitive position of LIVMARLI, the timing of ongoing and planned
clinical trials and the regulatory approval process of maralixibat
in other indications and jurisdictions and of volixibat. 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,”
“would,” “potential” 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 the COVID-19 pandemic, 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.
1Danks, et al. Archives of Disease in Childhood 1977
2Johns Hopkins Medicine.
hopkinsmedicine.org/health/conditions-and-diseases/Alagille-syndrome
3Vandriel, et al. GALA EASL 2020; Kamath, et al. Hepatology
Communications 2020
4Elisofon, et al. Journal of Pediatric Gastroenterology and
Nutrition 2010
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version on businesswire.com: https://www.businesswire.com/news/home/20210929005810/en/
Media: Erin Murphy media@mirumpharma.com
Investors: Ian Clements, Ph.D. ir@mirumpharma.com
Sam Martin Argot Partners ir@mirumpharma.com
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