Mirum Pharmaceuticals Submits European Marketing Authorization Application for Maralixibat in Alagille Syndrome Supported by New Positive Results from Natural History Study Comparison
September 13 2021 - 6:45AM
Business Wire
- Marketing Authorization Application for
treatment of cholestatic disease in Alagille syndrome
submitted.
- Natural history analysis shows significant
improvement in event-free survival and transplant-free
survival.
- PFIC regulatory strategy in Europe to focus
on results of MARCH-PFIC Phase 3 study, expected in second quarter
of 2022.
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the
submission of a Marketing Authorization Application (MAA) for the
treatment of cholestatic liver disease in patients with Alagille
syndrome (ALGS) to the European Medicines Agency (EMA). In
conjunction with the ALGS submission, Mirum’s MAA for progressive
familial intrahepatic cholestasis type 2 (PFIC2) was withdrawn with
plans to re-submit after availability of results from the ongoing
MARCH-PFIC Phase 3 study in a broader set of PFIC sub-types and
with higher doses of maralixibat.
The submission of the ALGS MAA presents an opportunity to
deliver the first ever pharmacologic treatment option for this
devastating disease for which there is significant and urgent unmet
need. This submission follows discussion with the EMA on the
appropriate strategy for seeking marketing approval for both ALGS
and PFIC broadly as soon as possible. The MAA is comprised of the
long-term ICONIC study in patients with ALGS, which showed a
significant improvement on pruritus (p<0.0001) and improvement
on other markers of cholestatic liver disease. The ICONIC data is
supported by a new analysis, which includes an aggregated cohort of
maralixibat-treated patients with ALGS (n=84) compared to a natural
history control cohort, demonstrating a statistically significant
improvement in six-year event-free survival (p<0.0001), with
events defined as biliary diversion surgery, liver transplant,
hepatic decompensation (ascites requiring therapy or variceal
bleeding) or death.
“The six-year event-free survival data, coupled with the
previously presented ICONIC data, provides a catalyst to accelerate
our ALGS submission,” said Chris Peetz, president and chief
executive officer at Mirum. “We feel a tremendous urgency to
advance maralixibat for patients as quickly as possible as its
availability may provide a significant shift in treatment options
for patients living with this unrelenting rare liver disease. The
recent long-term analysis allows us to take this major step forward
in ALGS today and we plan to submit for all PFIC types after
completion of the MARCH-PFIC Phase 3 study.”
In the U.S., the Food and Drug Administration has accepted
Mirum’s new drug application for maralixibat for the treatment of
cholestatic pruritus in patients with ALGS and the company is
preparing for a potential launch following the PDUFA date of
September 29, 2021, if approved by the FDA.
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
effects. 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. In a Phase 2 PFIC study, a genetically defined subset of
BSEP deficient (PFIC2), patients responded to maralixibat with an
increase in transplant-free survival. The U.S. Food and Drug
Administration has granted maralixibat Breakthrough Therapy
designation for the treatment of pruritus associated with ALGS in
patients one year of age and older and for PFIC2. Maralixibat was
shown to have a tolerable safety profile in the studies. The most
frequent treatment-related adverse events were diarrhea and
abdominal pain. Maralixibat has been studied extensively and its
safety database represents the largest database for an ASBT
inhibitor.
Until maralixibat is approved 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 further information about maralixibat’s ongoing
studies in pediatric liver disease, please visit the study
websites: Phase 3 MARCH study for PFIC and Phase 2b EMBARK study
for biliary atresia.
About Mirum Pharmaceuticals, Inc.
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. Mirum’s lead product candidate,
maralixibat, is an investigational oral drug in development for
Alagille syndrome (ALGS), progressive familial intrahepatic
cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA
for maralixibat in the treatment of cholestatic pruritus in
patients with ALGS. The NDA has been accepted for priority review
by the FDA with a PDUFA action date of September 29, 2021.
Additionally, Mirum’s marketing authorization application for the
treatment of cholestatic liver disease in patients with ALGS has
been submitted to the European Medicines Agency. Mirum is also
developing volixibat, also an oral ASBT-inhibitor, in primary
sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and
primary biliary cholangitis. For more information, visit
MirumPharma.com.
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 regulatory approval pathway for
maralixibat and the safety and efficacy of maralixibat. 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20210913005255/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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