- Maralixibat would be the first treatment available for use in
Alagille syndrome, if approved - Completion of the NDA submission
expected in the first quarter of 2021 - Expanded Access Program for
maralixibat now open
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a biopharmaceutical
company focused on the development and commercialization of novel
therapies for debilitating liver diseases today announced that it
has submitted the first portion of its rolling New Drug Application
(NDA) to the U.S. Food and Drug Administration (FDA) for
maralixibat. Maralixibat is a novel, minimally absorbed, orally
administered apical sodium dependent bile acid transporter (ASBT)
inhibitor being evaluated for the treatment of cholestatic pruritus
in patients with Alagille syndrome (ALGS). Mirum expects to
complete the NDA submission in the first quarter of 2021.
Mirum also launched its Expanded Access Program (EAP) for
maralixibat for the treatment of cholestatic pruritus in patients
with ALGS one year of age and older. The EAP is open for
registration in the United States and Canada. Through this program,
physicians can request access to maralixibat for eligible patients
who are not part of an ongoing clinical trial.
“We are thrilled to initiate the rolling NDA submission for
maralixibat, taking us one step closer to making this medicine
widely available for patients with ALGS,” said Chris Peetz,
president and chief executive officer at Mirum. “We believe the
results of our clinical program demonstrate the potential of
maralixibat to transform the treatment of this life-threatening
disease. We are also pleased to launch our Expanded Access Program
for patients with ALGS in the United States and Canada and are
evaluating ways in which we can make maralixibat available for
patients with ALGS in other countries. Additionally, we are
planning to broaden access to maralixibat through our anticipated
Marketing Authorization Application submission for patients with
PFIC2 in Europe later this year.”
About the NDA Submission Maralixibat was previously
granted Rare Pediatric Disease Designation for ALGS and, as such,
may qualify for receipt of a priority review voucher if the NDA is
approved by the FDA. Maralixibat was also granted Breakthrough
Therapy Designation for the treatment of pruritus associated with
ALGS in patients one year of age and older. Maralixibat was granted
Orphan Drug Designation by the FDA for the treatment of patients
with PFIC and ALGS in the United States.
Data from the maralixibat Phase 2 ICONIC study evaluating
patients with ALGS serves as the basis of efficacy for the
submission. Previously presented data from this study in November
2019 are available within the Publications and Presentations
section on Mirum’s website.
Mirum expects to complete the rolling NDA submission in the
first quarter of 2021 and is planning for a potential launch of
maralixibat in ALGS in the second half of 2021.
About the Expanded Access Program The EAP, sometimes
referred to as “compassionate use,” provides a potential pathway
for a patient with an immediately life-threatening condition or
serious disease to gain access to an investigational medicine for
the treatment of that disease outside of a clinical trial when no
comparable or satisfactory alternative therapy options are
available.
The goal of Mirum’s EAP is to provide access to maralixibat for
the treatment of cholestatic pruritus in eligible patients with
ALGS prior to FDA approval of the medication and until maralixibat
is available by prescription.
Requests for expanded access to maralixibat must be made by a
licensed physician. Physicians and patients can learn more about
the maralixibat EAP by visiting the program website at
www.ALGSEAP.com or via
https://clinicaltrials.gov/ct2/show/NCT04530994. Physicians who
would like to request access for their patient can contact
MirumALGS@clinigengroup.com.
For patients with PFIC, access to maralixibat is possible
through our MARCH Phase 3 study, which is currently open to
enrollment. More information can be found at:
https://pfictrial.com. Mirum plans to continually evaluate the need
for expanded access to maralixibat as studies reach enrollment
milestones.
About Maralixibat Maralixibat is a novel, minimally
absorbed, orally administered investigational drug being evaluated
in several rare cholestatic liver diseases. Maralixibat inhibits
the apical sodium dependent bile acid transporter (ASBT), resulting
in more bile acids being excreted in the feces, leading to lower
levels of bile acids systemically, thereby potentially reducing
bile acid mediated liver damage and related effects and
complications. More than 1,600 individuals have received
maralixibat, including more than 120 children who have received
maralixibat as an investigational treatment for Alagille syndrome
(ALGS) and progressive familial intrahepatic cholestasis (PFIC). In
the ICONIC Phase 2b ALGS clinical trial, patients taking
maralixibat had significant reductions in bile acids and pruritus
compared to placebo, as well as reduction in xanthomas and
accelerated growth long-term. In a Phase 2 PFIC study, a
genetically defined subset of BSEP deficient (PFIC2), patients
responded to maralixibat. The FDA has granted maralixibat
Breakthrough Therapy designation for treatment of pruritus
associated with ALGS in patients one year of age and older and for
PFIC2. Maralixibat was generally well-tolerated throughout the
studies. The most frequent treatment-related adverse events were
diarrhea, abdominal pain, and vomiting. Until maralixibat is
approved by the FDA and available for prescribing, the medication
is available to patients with ALGS through Mirum’s expanded access
program. For more information, please visit ALGSEAP.com. For more
information about the Phase 3 study for maralixibat in pediatric
patients with PFIC, visit PFICtrial.com.
About 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 Alagille syndrome 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
About Mirum Pharmaceuticals
Mirum Pharmaceuticals, Inc. is a clinical-stage
biopharmaceutical company focused on the development and
commercialization of a late-stage pipeline of novel therapies for
debilitating liver diseases. The company’s lead product candidate,
maralixibat, is an investigational oral drug in development for
Alagille syndrome (ALGS), progressive familial intrahepatic
cholestasis (PFIC), and biliary atresia. The Company has initiated
a rolling NDA submission for maralixibat in the treatment of
patients with cholestatic pruritus associated with ALGS and expects
to complete the submission in the first quarter of 2021.
Additionally, the company plans to submit a marketing authorization
application to the European Medicines Agency for maralixibat in the
treatment of patients with PFIC2 in the fourth quarter 2020.
The company is also developing volixibat, also an oral
ASBT-inhibitor, in primary sclerosing cholangitis and intrahepatic
cholestasis of pregnancy. For more information, visit
MirumPharma.com. Follow Mirum on Twitter, Facebook and
LinkedIn.
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 results, conduct, progress and
timing of Mirum’s ongoing and planned studies for maralixibat, as
well as Mirum’s Expanded Access Program for maralixibat, the
regulatory approval path for maralixibat and volixibat, and the
potential launch of maralixibat, if approved. 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 “plans,” “will”,
“believes,” “anticipates,” “expects,” “intends,” “goal,”
“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/20200901005437/en/
Investor Contact: Ian Clements, Ph.D. ir@mirumpharma.com
Media Contact: Erin Murphy media@mirumpharma.com
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