European Medicines Agency Recommends Orphan Drug Designation for Relacorilant to Treat Patients with Cushing’s Syndrome
May 02 2019 - 5:05PM
Corcept Therapeutics Incorporated (NASDAQ: CORT), a
commercial-stage company engaged in the discovery and development
of drugs to treat severe metabolic, oncologic and psychiatric
disorders by modulating the effects of the stress hormone cortisol,
today announced that the European Medicines Agency Committee for
Orphan Medicinal Products (COMP) has issued a positive opinion
recommending the approval of orphan drug designation for Corcept’s
proprietary, selective cortisol modulator, relacorilant, for the
treatment of Cushing’s syndrome. The European Commission is
expected to adopt the COMP’s recommendation in May 2019.
Orphan drug designation in the European Union
(EU) provides regulatory and financial incentives for companies to
develop and market therapies to treat serious disorders affecting
no more than five in 10,000 persons in the EU, including ten-year
marketing exclusivity in the EU upon approval, eligibility for
protocol assistance, reduced fees and access to the EU’s
centralized marketing authorization procedure.
To be considered for orphan designation in the
EU, there must be plausible evidence of a drug candidate’s efficacy
and of its potential to confer significant clinical benefit
compared to already-approved treatments. The COMP letter
states, “The sponsor has provided clinical data that demonstrate
that the product can reduce blood pressure and improve control of
hyperglycaemia in patients who were not adequately managed by
currently authorised products. The Committee considered that this
constitutes a clinically relevant advantage.” The medications
ketoconazole, metyropone and pasireotide are approved in the EU for
the treatment of one or more aetologies of Cushing’s syndrome.
Corcept based its submission on data from
relacorilant’s Phase 2 trial, in which patients demonstrated
improvements in the primary endpoints of glucose control and
hypertension, and in a variety of secondary endpoints, including
weight loss, liver function, coagulopathy, cognition, mood, insulin
resistance, and quality of life. Relacorilant was
well-tolerated, with the most common treatment-emergent adverse
events being those associated with reduction in excess cortisol
activity – headache, nausea, back pain and edema – that usually
resolve with continued treatment.
Because relacorilant, unlike Corcept’s
FDA-approved cortisol modulator, Korlym®, does not bind to the
progesterone receptor, patients did not experience vaginal bleeding
caused by uterine thickening, a frequently-reported adverse event
of Korlym. There were also no cases of drug-induced hypokalemia
(low potassium), a leading cause of Korlym
discontinuation.
Relacorilant’s Phase 3 GRACE trial is underway
and is expected to enroll 130 patients at 60 sites in the United
States, Canada, Europe and Israel.
The United States Food and Drug Administration
granted relacorilant orphan drug status for Cushing’s syndrome in
October 2018.
Cushing’s Syndrome
Hypercortisolism, often referred to as Cushing’s
syndrome, is caused by excessive activity of the hormone cortisol.
Endogenous Cushing’s syndrome is an orphan disease that most often
affects adults aged 20-50. In the United States, an estimated
20,000 patients have Cushing’s syndrome, with about 3,000 new
patients being diagnosed each year. Symptoms vary, but most people
experience one or more of the following manifestations: high blood
sugar, diabetes, high blood pressure, upper-body obesity, rounded
face, increased fat around the neck, thinning arms and legs, severe
fatigue and weak muscles. Irritability, anxiety, cognitive
disturbances and depression are also common. Hypercortisolism can
affect every organ system in the body and can be lethal if not
treated effectively.
Corcept Therapeutics Incorporated
Corcept is a commercial-stage company engaged in
the discovery and development of drugs that treat severe metabolic,
oncologic and psychiatric disorders by modulating the effects of
the stress hormone cortisol. Corcept’s approved product, Korlym®,
was the first FDA-approved treatment for patients with Cushing’s
syndrome. Korlym modulates the activity of cortisol at the
glucocorticoid receptor, one of the two receptors to which cortisol
binds. Corcept has discovered a large portfolio of proprietary
compounds that selectively modulate the effects of cortisol but not
progesterone. Corcept owns extensive United States and foreign
intellectual property covering the composition of its selective
cortisol modulators and in the use of cortisol modulators,
including Korlym, to treat a wide variety of serious disorders.
Forward-Looking Statements
Statements in this press release, other than
statements of historical fact, are forward-looking statements,
which are based on our current plans and expectations and are
subject to risks and uncertainties that might cause actual results
to differ materially from those such statements express or imply.
These risks and uncertainties include, but are not limited to, our
ability to generate sufficient revenue to fund our commercial
operations and development programs; the availability of competing
treatments, including generic versions of Korlym; our ability to
obtain acceptable prices or adequate insurance coverage and
reimbursement for Korlym; and risks related to the development of
our product candidates, including regulatory approvals, mandates,
oversight and other requirements. These and other risks are set
forth in our U.S. Securities and Exchange Commission (SEC) filings,
which are available at our website and the SEC’s website. In this
press release, forward-looking statements include those concerning
expectations that the European Commission will adopt the COMP’s
recommendation in May 2019; the regulatory and financial incentives
accompanying orphan drug designation; the progress, timing, design
and results of our development programs, including the GRACE trial;
and the scope and protective power of our intellectual property. We
disclaim any intention or duty to update forward-looking statements
made in this press release.
CONTACT:Christopher S. James,
MDDirector, Investor RelationsCorcept
Therapeutics650-684-8725cjames@corcept.comwww.corcept.com
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