Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN)
today announced that the U.S. Food and Drug Administration (FDA)
has granted orphan drug designation to ACH-4471 for the treatment
of paroxysmal nocturnal hemoglobinuria (PNH). Furthermore,
Achillion announced today 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 PNH.
“We are very pleased with the receipt of orphan
drug designation from the FDA, and a positive opinion by COMP for
orphan status, for ACH-4471, a first-in-class, oral inhibitor of
complement factor D,” commented Milind Deshpande, Ph.D., President
and Chief Executive Officer of Achillion. “We believe that
modulation of the complement alternative pathway by targeting
factor D, could provide a novel and pharmacologically
differentiated approach to treating diseases of the complement
system, including PNH and C3G.”
The FDA Orphan Drug Designation program provides
incentives for the development of potentially promising drugs to
treat, diagnose or prevent orphan diseases and disorders that
affect fewer than 200,000 people in the U.S. This designation may
provide, under specified conditions, for a seven-year marketing
exclusivity period, as well as certain incentives, including
federal grants, tax credits and a waiver of PDUFA filing fees.
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 Paroxysmal Nocturnal
Hemoglobinuria
PNH is thought to be caused by a mutation
resulting in the absence of receptors normally present on red blood
cells (RBCs) that interact with the alternative pathway (AP). The
AP of the complement system typically functions normally in these
patients but due to the lack of key receptors, known as CD55 and
CD59, on the surface of PNH RBCs, the AP treats these cells as
foreign and destroys them via hemolysis in the circulatory system
(intravascular hemolysis). Factor D is a critical protein of the
AP, including the amplification loop of the complement system, and
it is believed that inhibiting it could control the AP activity.
Furthermore, this mechanism of action represents a potentially
distinct and unique therapeutic approach for controlling both
intravascular hemolysis, and extravascular hemolysis associated
with C5 targeted therapies for PNH.
About the Achillion Alternative Pathway
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 platform 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 paroxysmal nocturnal
hemoglobinuria (PNH), C3 glomerulopathy (C3G), immune
complex-mediated membranoproliferative glomerulonephritis
(IC-MPGN), and geographic atrophy (GA), an advanced form of dry
age-related macular degeneration (dry AMD).
About Achillion
Pharmaceuticals
Achillion Pharmaceuticals, Inc. (NASDAQ:ACHN) is
a science-driven, patient-focused company seeking to leverage its
strengths across the continuum from discovery to commercialization
in its goal of providing better treatments for people with serious
diseases. The company employs a highly-disciplined discovery and
development approach that has allowed it to pursue best-in-class
oral antiviral therapy for chronic hepatitis C (HCV) and build a
platform of potent and specific complement factor D inhibitors for
AP-mediated diseases. Achillion is rapidly advancing its efforts to
become a fully-integrated pharmaceutical company with a goal of
bringing 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,” “may,”
“potential,” and similar expressions to identify such
forward-looking statements. These forward-looking statements also
include statements about: Achillion’s ability to obtain and
maintain orphan drug designation for ACH-4771 and the expected
benefits of orphan drug designation; the potential benefits of, and
potential indications for, Achillion’s compounds that inhibit
factor D, including ACH-4771; and statements concerning Achillion’s
strategic goals, efforts, plans, and prospects. 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:
successfully obtain orphan drug designation from the EC for
ACH-4771; maintain any orphan drug designation once obtained; avail
itself of any of the benefits of orphan drug designation for
ACH-4771, assuming that such designation is successfully obtained
and maintained, because, for example, even if Achillion obtains
orphan drug designation, exclusivity may not prevent the FDA, EMA
or other regulatory authorities from approving competing products;
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, including
completing the patent process such that patents issue with respect
to composition of matter claims for compounds that inhibit
complement factor D activity; demonstrate in any current and future
clinical trials the requisite safety, efficacy and combinability of
its drug candidates; obtain and maintain necessary regulatory
approvals, and the granting of orphan designation does not alter
the standard regulatory requirements and process for obtaining such
approval; establish commercial manufacturing arrangements;
identify, enter into and maintain collaboration and other
commercial agreements with third-parties; compete successfully in
the markets in which it seeks to develop and commercialize its
product candidates and future products; manage expenses; 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 Quarterly Report on Form 10-Q
for the fiscal quarter ended September 30, 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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