Achillion Announces ACH-4471 Receives Positive Opinion for Orphan Drug Designation in the European Union for the Treatment of...
February 26 2018 - 5:05AM
Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN), a
biopharmaceutical company focused on advancing small molecule
factor D inhibitors to modulate the complement alternative pathway,
today announced that the European Medicines Agency (EMA) Committee
for Orphan Medicinal Products (COMP) issued a positive opinion on
ACH-4471 for orphan status in the European Union (EU) for the
treatment of C3 glomerulopathy (C3G).
“We are very pleased that ACH-4471, a
first-in-class, oral inhibitor of complement factor D, has received
a positive orphan status opinion for the treatment of C3G. At
Achillion, we continually work to enhance the awareness of C3G, a
rare renal disorder consisting of dense deposit disease (C3G) and
C3 glomerulonephritis (C3GN) for which there are no approved
therapies,” commented Milind Deshpande, Ph.D., Chief Executive
Officer of Achillion.
Dr. Deshpande further stated, “The mechanism of
action for our factor D inhibitors, highlighted by the safety and
efficacy data observed to date with ACH-4471, we believe represents
a truly novel and targeted approach to potentially treating C3G, a
chronic and devastating disease affecting the kidney. By
specifically targeting factor D, we believe we are targeting the
root cause of disease, which experts attribute to overactivation of
the alternative pathway. By inhibiting factor D, we believe
ACH-4471 may be able to reduce the excessive formation of C3
fragments in the blood which in C3G are continually being deposited
in the kidney leading to impaired function and potentially renal
failure.”
There are estimated to be more than 4,000 C3G
patients in Europe for whom there are no approved therapies.
In the EU, the COMP adopts an opinion on the
granting of orphan drug designation, after which the opinion is
submitted to the European Commission (EC) for a decision on whether
or not to accept the opinion. Orphan drug designation by the EC may
provide for regulatory and financial incentives for companies to
develop and market therapies that treat a life-threatening or
chronically debilitating condition affecting no more than five in
10,000 persons in the EU, and where no satisfactory treatment is
available. In addition to a 10-year period of marketing exclusivity
in the EU after product approval, orphan drug designation provides
incentives for companies seeking protocol assistance from the EMA
during the product development phase, and direct access to the
centralized authorization procedure.
About C3G
C3G is a devastating disease affecting the
kidney for which there is no approved therapy. C3G affects persons
of all ages with men and women equally affected. There are
estimated to be approximately 4,000 C3G patients in the United
States, more than 4,000 in Europe, and greater than 1,000 patients
with this disease in Japan. C3G describes a rare renal disease
characterized by the presence of C3 protein fragments in the
filtering units (glomeruli) of the kidney. These C3 fragment
deposits are thought to be the result of overactivation of the
complement alternative pathway. The chronic deposition of C3
fragments results in inflammation in the glomeruli
(glomerulonephritis) and subsequent permanent renal damage. An
estimated 30-50% of C3G patients will require dialysis or a
transplant within 10 years of diagnosis.
About the Complement Factor D
Platform
Achillion has leveraged its internal discovery
capabilities and a novel complement-related drug development
platform to develop small molecule factor D inhibitor compounds
that target the complement AP. Factor D is an essential serine
protease involved in the AP, a part of the innate immune system.
Achillion's complement portfolio is focused on seeking to advance
small molecule compounds that inhibit factor D and can potentially
be used in the treatment of immune-related diseases in which the AP
plays a critical role. Potential indications currently being
evaluated for these compounds include C3G, immune complex-mediated
membranoproliferative glomerulonephritis (IC-MPGN), and paroxysmal
nocturnal hemoglobinuria (PNH).
About Achillion
Pharmaceuticals
Achillion Pharmaceuticals, Inc. (NASDAQ:ACHN) is
a science-driven, patient-focused biopharmaceutical company seeking
to leverage its believed strengths across the continuum from
discovery through commercialization in its goal of meeting the
needs of people with complement-mediated diseases. The company has
employed a highly-disciplined discovery and development approach
that has allowed it to develop potent and specific complement
factor D inhibitors for AP-mediated diseases. Achillion is rapidly
advancing its efforts to bring potentially life-saving medicines to
patients with rare diseases. More information is available
at http://www.achillion.com.
Cautionary Note Regarding
Forward-Looking Statements
This press release includes forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995 that are subject to risks, uncertainties and
other important factors that could cause actual results to differ
materially from those indicated by such forward-looking statements.
Achillion may use words such as “expect,” “anticipate,” “project,”
“target,” “intend,” “plan,” “aim,” “believe,” “seek,” “estimate,”
“can,” “could” “focus,” “will,” “look forward,” “goal,” and “may”
and similar expressions to identify such forward-looking
statements. These forward-looking statements also include
statements about: the potential for a favorable decision by the EC
granting orphan drug designation for ACH-4471; the expected
benefits of orphan drug designation for ACH-4471 in the EU;
Achillion’s expected plans, timing, data readouts and results from
ongoing and planned clinical trials of ACH-4471, ACH-5228 and
ACH-5548. Among the important factors that could cause actual
results to differ materially from those indicated by such
forward-looking statements are risks relating to, among other
things, Achillion’s ability to: advance the preclinical and
clinical development of its complement factor D inhibitors under
the timelines it projects in current and future preclinical studies
and clinical trials; obtain and maintain patent protection for its
drug candidates and the freedom to operate under third party
intellectual property; demonstrate in any current and future
clinical trials the requisite safety, efficacy and combinability of
its drug candidates; obtain and maintain necessary regulatory
approvals; establish commercial manufacturing arrangements;
identify and enter into collaboration agreements with
third-parties; compete successfully in the markets in which it
seeks to develop and commercialize its product candidates and
future products; manage litigation; raise the substantial
additional capital needed to achieve its business objectives; and
successfully execute on its business strategies. These and other
risks are described in the reports filed by Achillion with the U.S.
Securities and Exchange Commission, including its Annual Report on
Form 10-K for the fiscal year ended December 31, 2017, and any
subsequent SEC filings.
In addition, any forward-looking statement in
this press release represents Achillion's views only as of the date
of this press release and should not be relied upon as representing
its views as of any subsequent date. Achillion disclaims any duty
to update any forward-looking statement, except as required by
applicable law.
Investors & Media:
Glenn Schulman, PharmD, MPH
Executive Director, Investor Relations
Achillion Pharmaceuticals, Inc.
Tel. (203) 752-5510
gschulman@achillion.com
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