Catalyst Announces Initiation of an Expanded Access Program to Provide Firdapse at No Cost to U.S. Patients with Lambert-Eaton Myasthenic Syndrome
"As part of our ongoing commitment to the LEMS community, we are making
Firdapse available to those who would like access to this
investigational drug, but are unable to participate in clinical studies
or travel to sites where it is available," said
Patients diagnosed with LEMS or physicians treating such patients can call toll free 1-844-FIRDAPSE (1-844-347-3277) to inquire about this program.
"In previously published clinical trials, amifampridine has been shown
to reduce neuromuscular weakness and symptoms of autonomic dysfunction
in LEMS patients while being well tolerated," said
Expanded access is a mechanism supported by regulatory agencies for
getting investigational treatment to patients who have a life
threatening or severely debilitating disease and who cannot be
satisfactorily treated with an alternative therapy approved by the
About Lambert-Eaton Myasthenic Syndrome (LEMS)
LEMS is an autoimmune neuromuscular disease in which the release of acetylcholine is decreased at the neuromuscular junction, resulting in muscle weakness. Patients with LEMS typically present with fatigue, muscle pain and stiffness that is generally more marked in the proximal muscles, most often muscles of the legs and trunk. Other symptoms may include reduced reflexes, drooping eyelids, facial weakness and swallowing problems. Patients often report a dry mouth, impotence, constipation and light-headedness on standing. On occasion, these problems can be life threatening when the weakness involves respiratory muscles. Approximately 50 percent of patients diagnosed with LEMS have an underlying malignancy, most commonly small cell lung cancer. LEMS is therefore regarded as a paraneoplastic syndrome (a condition that arises as a result of cancer elsewhere in the body).
About Firdapse
Firdapse, amifampridine phosphate or 3,4-diaminopyridine phosphate (3,4-DAP), is a potassium channel inhibitor. By blocking this ion channel, Firdapse increases the nerve repolarization time, which causes an increase in the influx of calcium, thereby causing more acetylcholine to be released, restoring muscle fiber contraction thus relieving muscle weakness caused by LEMS. In addition to LEMS, other potential orphan neuromuscular indications for Firdapse include certain types of Myasthenia Gravis and Congenital Myasthenic Syndrome, among others.
Firdapse has been granted orphan drug and breakthrough therapy
designations by the U.S.
About Catalyst's Commitment to LEMS Patients
Catalyst is committed to bringing all patients diagnosed with LEMS a
quality, safe and effective,
About
Catalyst is also developing a potentially safer and more potent
vigabatrin analog (designated CPP-115) to treat infantile spasms, and
epilepsy, as well as other neurological conditions associated with
reduced GABAergic signaling, like post-traumatic stress disorder and
Tourette syndrome. CPP-115 has been granted U.S. orphan drug designation
for the treatment of infantile spasms by the
Forward-Looking Statements
This press release contains forward-looking statements.
Forward-looking statements involve known and unknown risks and
uncertainties, which may cause Catalyst's actual results in future
periods to differ materially from forecasted results. A number of
factors, including the anticipated timing of the receipt of top-line
results from the double-blind, placebo-controlled portion of the Phase 3
trial of Firdapse, whether historic metrics of patients enrolled in the
trial who complete the run-in phase of the trial and are randomized into
the double-blind, placebo-controlled portion of the trial will continue
to apply, such that at least 36 patients will be randomized into the
double-blind, placebo-controlled portion of the trial from the patients
already enrolled in the trial, whether the Phase 3 trial will be
successful, whether the receipt of breakthrough therapy designation for
Firdapse will expedite the development and review of Firdapse by the
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Media/Investor Contacts
LaVoie Health Science
dconnolly@lavoiehealthscience.com
or
akrause@lavoiehealthscience.com
or
Company
Contact
Patrick J.
McEnany, 305-529-2522
Chief Executive Officer
pmcenany@catalystpharma.com
Source:
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