Catalyst Pharmaceuticals Announces Plans to Resubmit New Drug Application for Firdapse®
- Positive meeting with the
FDAabout the resubmission of an NDA for Firdapse
- Resubmission of NDA for Firdapse on schedule for end of the first quarter
As previously reported, Catalyst is presently conducting a Phase 3 double-blind placebo-controlled clinical trial evaluating Firdapse® for the treatment of congenital myasthenic syndromes (CMS), and Catalyst expects to report top line results from the trial in the second half of this year. Catalyst is currently evaluating its options for the most appropriate and efficient path forward to hopefully include CMS in any approved labeling for Firdapse.
"We remain on track to resubmit our NDA for Firdapse in the first quarter of this year," stated
About Lambert-Eaton Myasthenic Syndrome (LEMS)
Lambert-Eaton Myasthenic Syndrome, or LEMS, is a rare autoimmune disorder, most often characterized by muscle weakness of the limbs. The disease is caused by an autoimmune reaction where antibodies are formed against voltage gated potassium channels in the connection between nerves and the muscles they communicate with. In approximately 50% of cases, LEMS is associated with an underlying malignancy, most commonly small-cell lung cancer, and in some individuals, LEMS is the first symptom of such malignancy. LEMS generally affects the extremities, especially the legs. As the disease most affects the parts of limbs closest to the trunk, difficulties with climbing stairs or rising from a
sitting position are commonly noted. Physical exercise and high temperatures tend to worsen the symptoms. Other symptoms occasionally seen include weakness of the muscles of the mouth, throat, and eyes. Individuals affected with LEMS also may have a disruption of the autonomic nervous system, including dry mouth, constipation, blurred vision, impaired sweating, and/or hypotension.
About Congenital Myasthenic Syndromes (CMS)
Congenital myasthenic syndromes, or CMS, are rare neuromuscular disorders comprising a spectrum of genetic defects and is characterized by fatigable weakness of skeletal muscles with usual onset at or shortly after birth or early childhood; in rare cases symptoms may not manifest themselves until later in childhood or adulthood. The severity and course of the disease are variable, ranging from minor symptoms to
progressive disabling weakness; symptoms may be mild, but sudden severe exacerbations of weakness or even sudden episodes of respiratory insufficiency also occur. Congenital myasthenic syndromes are rare, estimated at one-tenth that of myasthenia gravis, which in itself is rare. Based on currently available information, Catalyst estimates that there are between 1,000 and 1,500 CMS patients in
Catalyst Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, including Lambert-Eaton myasthenic syndrome (LEMS), congenital myasthenic syndromes
(CMS), MuSK antibody positive myasthenia gravis, and infantile spasms. Firdapse® has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the treatment of LEMS and Orphan Drug Designation for LEMS, CMS and myasthenia gravis. Firdapse is the first and only approved drug in Europe for symptomatic treatment in adults with LEMS.
Catalyst is also developing CPP-115 to treat refractory infantile spasms. CPP-115 has been granted U.S. Orphan Drug Designation for the treatment of infantile spasms by the FDA and has been granted E.U. Orphan Medicinal Product Designation for the treatment of West syndrome by the European Commission. In addition, Catalyst is developing a generic version of Sabril® (vigabatrin).
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 (i) whether the results of the LMS-003 trial, combined with the results of the Company's previous Phase 3 trial, will be acceptable to the
Chief Executive Officer
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