Catalyst Biosciences Granted FDA Orphan Drug Designation for Subcutaneous Recombinant Human Factor IX Variant for Treatment o...
September 26 2017 - 8:00AM
-- Orphan drug designation provides marketing
exclusivity if approved by FDA --
Catalyst Biosciences, Inc. (Nasdaq:CBIO) today announced that
the U.S. Food and Drug Administration (FDA) has granted
orphan drug designation (ODD) for CB 2679d/ISU304, Catalyst’s
highly potent subcutaneous next-generation recombinant human Factor
IX variant, for the treatment of hemophilia B.
“Receipt of FDA orphan status will support the
development of this promising subcutaneous treatment option for
individuals with hemophilia B. Notable benefits of ODD include the
potential for seven years of market exclusivity,” said Nassim
Usman, Ph.D., President and Chief Executive Officer of Catalyst.
“Assuming our ongoing clinical development program is successful,
this important designation will bolster the rapid availability of
CB 2679d for subcutaneous prophylaxis in this
life-threatening and chronically debilitating disorder.”
In June 2017, the Company announced that the European
Commission had also granted orphan medicinal product designation to
CB 2679d for hemophilia B. Catalyst and its collaborator, ISU
Abxis, are evaluating CB 2679d in a Phase 1/2 proof-of-concept
study in individuals with severe hemophilia B. The Companies plan
to have interim, top-line results by the end of 2017 and complete
trial results in early 2018.
About Orphan Designation Orphan drug
designation is granted by the FDA to drugs and biologics that are
defined as those intended for the safe and effective treatment,
diagnosis or prevention of rare diseases/disorders that affect
fewer than 200,000 people in the U.S. Orphan drug designation
provides certain incentives which may include tax credits towards
the cost of clinical trials and prescription drug user fee waivers.
If a product that has orphan drug designation subsequently receives
the first FDA approval for the disease for which it has such
designation, the product is entitled to orphan product
exclusivity.
About Factor IX CB 2679d is a next-generation
high-potency coagulation Factor IX variant that is being evaluated
in a Phase 1/2 proof-of-concept clinical trial in South Korea.
Catalyst believes that CB 2679d may allow for subcutaneous
prophylactic treatment of individuals with hemophilia B. Learn
more about Factor IX.
About Hemophilia and Factor Replacement Therapy
Hemophilia, for which there is no cure, is a rare but serious
bleeding disorder that results from a genetic or an acquired
deficiency of a protein required for normal blood coagulation.
Individuals with hemophilia suffer from spontaneous bleeding
episodes as well as substantially prolonged bleeding times upon
injury. Learn more about hemophilia.
About Catalyst Catalyst is a clinical-stage
biopharmaceutical company focused on developing novel medicines to
address hematology indications. Catalyst is focused on the field of
hemostasis, including the subcutaneous prophylaxis of hemophilia
and facilitating surgery in individuals with hemophilia. For more
information, visit www.catalystbiosciences.com.
Forward-Looking StatementsThis press release
contains forward-looking statements that involve substantial risks
and uncertainties. All statements, other than statement of
historical facts, included in this press release regarding our
strategy, the potential uses and benefits of CB 2679d and
development plans for this product candidate are forward-looking
statements. Examples of such statements include, but are not
limited to, statements relating to Catalyst’s clinical trial
timelines, including the anticipated completion of a Phase 1/2
proof-of-concept study for CB 2679d, the plans to disclose interim
top-line results from the Phase 1/2 study by the end of 2017 and
complete trial results by early 2018 and to report results at
upcoming medical conferences, and the potential uses and benefits
of subcutaneously dosed CB 2679d. Actual results or events could
differ materially from the plans and expectations and projections
disclosed in these forward-looking statements. Various important
factors could cause actual results or events to differ materially
from the forward-looking statements that Catalyst makes, including,
but not limited to, the risk that trials and enrollment may be
delayed and may not have satisfactory outcomes, that human trials
will not replicate the results from preclinical studies, that
subcutaneous dosing of CB 2679d may not replicate potency or
duration of blood levels, that potential adverse effects may arise
from the testing or use of Catalyst’s products, including the
generation of antibodies, the risk that costs required to develop
or manufacture Catalyst’s products will be higher than anticipated,
competition, and other factors described in the “Risk Factors”
section of the Company’s most recent Quarterly Report on Form 10-Q
filed with the SEC on August 3, 2017. Catalyst does not assume
any obligation to update any forward-looking statements, except as
required by law.
Contacts: Investors:
Fletcher Payne, CFO, Catalyst Biosciences
650.871.0761
investors@catbio.com
Media:Denise Powell510.703.9491denise@redhousecomms.com
Catalyst Biosciences (NASDAQ:CBIO)
Historical Stock Chart
From Mar 2024 to Apr 2024
Catalyst Biosciences (NASDAQ:CBIO)
Historical Stock Chart
From Apr 2023 to Apr 2024