Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that
the U.S. Food and Drug Administration (FDA) has extended the review
of the Supplemental New Drug Application (sNDA) for LIVMARLI®
(maralixibat) oral solution for the treatment of cholestatic
pruritus in patients with progressive familial intrahepatic
cholestasis (PFIC). The new Prescription Drug User Fee Act (PDUFA)
date is March 13, 2024.
The FDA extended the PDUFA date to allow time for a full review
of a submission provided in response to an FDA information request,
which the FDA deemed a major amendment. The submission comprised
further analysis of certain clinical information that was
previously provided to the FDA by the company. No additional data
or studies have been requested.
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.
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 the U.S. in
cholestatic pruritus in PFIC patients three months of age and
older, and in Europe, in PFIC for patients two months of age and
older.
LIVMARLI is currently being evaluated in late-stage clinical
studies in other rare cholestatic liver diseases including biliary
atresia. LIVMARLI has received Breakthrough Therapy designation for
ALGS and PFIC type 2 and orphan designation for ALGS, PFIC and
biliary atresia. 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 can cause 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
gastrointestinal bleeding and bone fractures.
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
cholestatic pruritus in patients with Alagille syndrome in the U.S.
(three months and older), in Europe (two months and older), and in
Canada. Mirum has also submitted LIVMARLI for approval in the U.S.
in cholestatic pruritus in PFIC patients three months of age and
older and in Europe in PFIC for 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 three investigational
treatments for debilitating liver diseases. The LIVMARLI
development program includes the Phase 2b EMBARK study for biliary
atresia. Mirum’s second investigational IBAT inhibitor is
volixibat, which 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.
To learn more about Mirum, visit mirumpharma.com and follow
Mirum on Facebook, LinkedIn, Instagram and Twitter.
Forward-Looking Statements
This press release contains “forward-looking statements” as that
term is defined in the Private Securities Litigation Reform Act of
1995. Without limiting the foregoing, these statements are often
identified by the words “look forward to”, “will,” “may”, “might”,
“believes”, “anticipates”, “plans”, “expects”, “intends,”
“potential” or similar expressions. In addition, expressions of
strategies, intentions or plans are also forward-looking
statements. Such forward-looking statements include, but are not
limited to, references to: the updated PDUFA date for the PFIC sNDA
and the timeline for the FDA’s review of Company submitted data.
Because such statements are subject to risks and uncertainties,
actual results may differ materially from those expressed or
implied by such forward-looking statements. Other factors that
might cause such a difference include those discussed in the
Company’s filings with the SEC. 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/20231017111211/en/
Media: Erin Murphy 510-508-6521 media@mirumpharma.com
Investors: Andrew McKibben ir@mirumpharma.com
Sam Martin Argot Partners ir@mirumpharma.com
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