- Positive HTA recommendation for reimbursement
signifies an important milestone towards public funding to treat
eligible patients with this rare liver disorder
- LIVMARLI is the first and only approved
medication to treat cholestatic pruritus in patients with Alagille
syndrome in Canada
Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that
the Canadian Agency for Drugs and Technologies in Health (CADTH)
Canadian Drug Expert Committee (CDEC) has recommended public
reimbursement for LIVMARLI® (maralixibat oral solution) for the
treatment of cholestatic pruritus in patients with Alagille
Syndrome (ALGS), with certain conditions. This announcement follows
Health Canada’s authorization of LIVMARLI in this indication in
2023.
“The positive reimbursement recommendation for LIVMARLI by CADTH
is an important step forward for patients suffering from the
debilitating effects of cholestatic pruritus related to Alagille
syndrome,” said Chris Peetz, chief executive officer at Mirum.
“Accelerating access in all provinces to this meaningful treatment
is critical as it is the first and only approved therapy available
in this setting in Canada. These patients suffer greatly from the
debilitating and disruptive effects of Alagille syndrome, which
often starts in early childhood.”
“CADTH’s recommendation of reimbursement for LIVMARLI provides a
meaningful advancement in the treatment of cholestatic pruritus for
patients with Alagille syndrome. I am pleased that LIVMARLI will
now become more accessible in Canada, where I have seen the
critical need for a treatment to address the itch caused by
cholestasis,” said Dr. Binita Kamath, Director, Hepatology Program
and Senior Associate Scientist at The Hospital for Sick Children
(SickKids), Toronto, Canada.
“This recommendation is incredibly meaningful as it signifies
hope for patients and their families who have suffered from the
disruptive and painful effects of pruritus for far too long,” said
Roberta Smith, president, Alagille Syndrome Alliance and mother of
a daughter with ALGS. “We are grateful to CADTH for ensuring
support of this new and important medication.”
The CADTH recommendation is based on data from the pivotal
ICONIC study including six years of data across the LIVMARLI
clinical program resulting in a robust body of evidence in patients
with cholestatic pruritus in ALGS. Data from ICONIC demonstrated
statistically significant and clinically meaningful reductions in
pruritus compared to placebo, as well as significant reductions in
serum bile acids, both of which were durably maintained over
several years of treatment.
LIVMARLI is also approved for the treatment of cholestatic
pruritus in patients with Alagille syndrome (ALGS) in the U.S. and
Europe, and for Progressive Familial Intrahepatic Cholestasis
(PFIC) in the U.S. and in Europe.
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.
About LIVMARLI® (maralixibat) oral solution
LIVMARLI® (maralixibat) oral solution is an orally administered,
once-daily, ileal bile acid transporter (IBAT) inhibitor and the
only approved medication by the U.S. Food and Drug Administration
for the treatment of cholestatic pruritus in patients with Alagille
syndrome (ALGS) three months of age and older and progressive
familial intrahepatic cholestasis (PFIC) five years of age and
older.
LIVMARLI is also the only approved IBAT inhibitor approved by
the European Commission for the treatment of cholestatic pruritus
in patients with ALGS two months and older, and by Health Canada
for the treatment of cholestatic pruritus in ALGS. For more
information for U.S. residents, please visit LIVMARLI.com.
Mirum has also submitted LIVMARLI for approval in Europe in PFIC
for patients two months of age and older.
LIVMARLI has received Breakthrough Therapy designation for ALGS
and PFIC type 2 and orphan designation for ALGS and PFIC. To learn
more about ongoing clinical trials with LIVMARLI, please visit
Mirum’s clinical trials section on the company’s website.
IMPORTANT SAFETY INFORMATION
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 CHENODAL® (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 five
years of age and older. Mirum has submitted for approval in Europe
for the treatment of PFIC in patients two months of age and older.
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 or liver disease. CHENODAL has received medical necessity
recognition by the FDA to treat patients with cerebrotendinous
xanthomatosis (CTX).
Mirum’s late-stage pipeline includes two investigational
treatments for debilitating liver diseases. Volixibat, an IBAT
inhibitor, is being evaluated in two potentially registrational
studies including the Phase 2b VISTAS study for primary sclerosing
cholangitis and Phase 2b VANTAGE study for primary biliary
cholangitis. Lastly, CHENODAL, has been evaluated in a Phase 3
clinical study, RESTORE, to treat patients with CTX, with positive
topline results reported in 2023.
To learn more about Mirum, visit mirumpharma.com and follow
Mirum on Facebook, LinkedIn, Instagram and Twitter.
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 potential commercial success of
LIVMARLI in Canada and other approved territories and the
additional improvements in disease beyond pruritus. 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,” “intended,”
“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 geopolitical and macroeconomic events , and the other
risks described in Mirum’s filings with the Securities and Exchange
Commission, including its most recent Annual Report on Form 10-K,
as updated by any subsequent Quarter Reports on Form 10-Q. 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20240402976390/en/
Media: Erin Murphy 510-508-6521 media@mirumpharma.com
Investors: Andrew McKibben ir@mirumpharma.com
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