Breaking News
August 17, 2018 - Team explores diabetes drug’s ability to treat RSV infection
August 17, 2018 - New imaging technique can spot tuberculosis infection in an hour | News Center
August 17, 2018 - PolyU researchers design new self-fitting scaffold to induce bone regeneration
August 17, 2018 - CartiHeal and LSU Health successfully enroll first two patients in Agili-C IDE pivotal study
August 17, 2018 - Less-invasive options are slowing disease progression in glaucoma patients
August 17, 2018 - Researchers discover new promising target point for cancer and diabetes therapies
August 17, 2018 - Podcast: KHN’s ‘What the Health?’ See you in court!
August 17, 2018 - New mobile phone application enables early detection of cerebral ictus
August 17, 2018 - UK’s leading sight loss charity invites applications from brightest minds in ophthalmic research
August 17, 2018 - Researchers produce artificial placenta model that closely resembles natural organ
August 17, 2018 - FDA Alert: Temporary Total Artificial Heart Companion 2 Driver System by SynCardia Systems: Letter to Health Care Providers
August 17, 2018 - Researchers discover why sepsis from a staph infection causes organ failure
August 17, 2018 - Revealed: The molecular mechanism underlying hypertrophic cardiomyopathy | News Center
August 17, 2018 - Research explores relationship between personal history of infectious fever and cancer risk
August 17, 2018 - Study finds rise in cases of progressive massive fibrosis among U.S. coal miners
August 17, 2018 - NEDBELS project examines impact of neurodiversity concept on legal systems
August 17, 2018 - Seeking solutions to treat scleroderma
August 17, 2018 - Statins may improve conditions of people with rare lung disease
August 17, 2018 - Study finds why some people with brain markers of Alzheimer’s never develop dementia
August 17, 2018 - Life Biosciences contributes $100,000 to fund its biomedical innovation course on aging
August 17, 2018 - Researchers develop a set of health outcome measures for children with complex medical situations
August 17, 2018 - Many Americans Not Being Assessed for Depression
August 17, 2018 - Scientists report setbacks in quest for AIDS cure
August 17, 2018 - Christopher Gardner busts myths about milk | News Center
August 17, 2018 - Bacterial activity in child’s mouth may serve as biomarkers for autism spectrum disorder
August 17, 2018 - Scripps Research scientists uncover new approach for treating thrombocytopenia
August 17, 2018 - Mathematical model shows the influence of human behavior on spread of infectious diseases
August 17, 2018 - Valley Hospital achieves Magnet recognition for fourth consecutive time
August 17, 2018 - Researchers describe link between poor oocyte development and oxidative stress in obese mice
August 17, 2018 - Hospitals battle for control over fast-growing heart-valve procedure
August 17, 2018 - AHA: Home-Delivered Meals Keep Heart Failure Patients Out of Hospital
August 17, 2018 - In Southern Mozambique, only half of people diagnosed with HIV enroll in medical care
August 17, 2018 - Researchers discuss techniques to help combat growing epidemic of obesity
August 17, 2018 - Researchers develop novel statistical method to evaluate gene-to-gene interactions linked with cancer
August 17, 2018 - Island Fertility joins Stony Brook Community Medical to provide comprehensive fertility care
August 17, 2018 - Study shows link between thinning of the retina and early sign of Parkinson’s disease
August 17, 2018 - Digital birth control app gets FDA nod
August 17, 2018 - FDA grants approval for first generic version of epinephrine auto-injector
August 17, 2018 - Federal advisory group publishes recommendations on prevention of acute, chronic pain
August 17, 2018 - 3D-printed human body parts to be used as teaching aids for surgical training
August 17, 2018 - U.S. murder, suicide rates climbing again
August 17, 2018 - This is your brain on… roller coasters?
August 17, 2018 - Report discusses whether all newborns should undergo genetic sequencing
August 17, 2018 - UCR receives 2018 Inspiring Programs in STEM Award from INSIGHT Into Diversity magazine
August 17, 2018 - Researchers publish new paper on developing vaccine candidates for Helminthic parasites
August 17, 2018 - Researchers develop new method to diagnose broad range of cancers using malaria protein
August 17, 2018 - Female mosquitoes quickly evolve selective mating behavior when faced with threats
August 17, 2018 - FDA Grants Breakthrough Therapy Designation to Daiichi Sankyo’s FLT3 Inhibitor Quizartinib for Relapsed/Refractory FLT3-ITD AML
August 17, 2018 - Resistance training and exercise motivation go hand-in-hand
August 17, 2018 - A lesson for future doctors: Listen to and learn from your patients
August 17, 2018 - NUS study discovers a bidirectional regulator and shines light on A-to-I RNA editing in cancer cells
August 17, 2018 - Research shows link between high blood levels of omega-3s and better brain function in children
August 17, 2018 - Researchers propose new theory for how rare gene mutations cause Alzheimer’s disease
August 17, 2018 - New project to combat DMD-related fibrosis receives major funding boost
August 17, 2018 - Digital psychiatric therapy can ‘rewire’ the brain in children with ADHD, study shows
August 17, 2018 - Psychologist to assess how the brain maintains precise short-term and long-term memories
August 17, 2018 - Eating white button mushrooms could improve regulation of glucose in the liver
August 17, 2018 - Scientists identify mutational signatures in ovarian cancer
August 17, 2018 - Sun Pharma receives U.S. FDA approval for CEQUA to treat patients with dry eye disease
August 17, 2018 - Teva Announces Updated Indication and Vial Presentation for Granix (tbo-filgrastim) Injection in United States
August 17, 2018 - Study shows DNA methylation related to liver disease among obese patients
August 17, 2018 - Life on the border: Back at Stanford, ready to pitch in
August 17, 2018 - New device for accurately placing hemodialysis catheters on kidney patients
August 17, 2018 - New strategy accelerates, automates process of prototype molecule optimization
August 17, 2018 - Study finds role of autoimmunity in development of COPD
August 17, 2018 - Researchers transform research tool to study neuronal function
August 17, 2018 - Cognitive impairment does not equate to unhappiness in older adults
August 17, 2018 - Peer Comparisons Can Decrease Risky Prescribing Patterns
August 17, 2018 - Susceptible genes identified for childhood chronic kidney disease
August 17, 2018 - Research uncovers miscarriage cause, identifies potential targets for treatment
August 17, 2018 - Bacterial armor could be new target for antibiotics | News Center
August 17, 2018 - FDA expands approval of Vertex’ cystic fibrosis medicine to treat children aged 12 to
August 17, 2018 - Give Your Child a Head Start With Math
August 17, 2018 - Ground-breaking study tests whether rejected livers can be made viable for transplantation
August 16, 2018 - New algorithm could improve diagnosis of rare diseases | News Center
August 16, 2018 - SCHILLER introduces latest generation of ECG device, CARDIOVIT AT-102 G2
August 16, 2018 - Proper treatment, refraining from smoking can reduce heart disease risk from type 2 diabetes
August 16, 2018 - Mount Sinai study could transform treatment for patients with retinal degenerative diseases
August 16, 2018 - Penn researchers develop first mouse model of idiopathic pulmonary fibrosis
August 16, 2018 - Four tips to help prevent fall allergy symptoms
Catalyst Pharmaceuticals Announces Submission of New Drug Application for Firdapse for Treatment of Lambert-Eaton Myasthenic Syndrome

Catalyst Pharmaceuticals Announces Submission of New Drug Application for Firdapse for Treatment of Lambert-Eaton Myasthenic Syndrome

image_pdfDownload PDFimage_print

Treatment for Lambert Eaton Myasthenic Syndrome (LEMS); Congenital Myasthenic Syndromes (CMS)

Catalyst Pharmaceuticals Announces Submission of New Drug Application for Firdapse for Treatment of Lambert-Eaton Myasthenic Syndrome

CORAL GABLES, Fla., March 29, 2018 (GLOBE NEWSWIRE) — Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, today announced its submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Firdapse (amifampridine) for the symptomatic treatment of Lambert-Eaton myasthenic syndrome (LEMS).

Last month, Catalyst announced the results of its recent positive Type C meeting with the FDA and reported that the minutes of the meeting received from the Agency reflect the FDA’s advice to Catalyst that its proposed filing package would be sufficient for submission of an NDA for Firdapse. Last year, Catalyst reported positive top-line results from its second Phase 3 clinical trial of Firdapse for the treatment of LEMS, which was conducted under a protocol agreed to by the FDA through the Special Protocol Assessment process.

“We are pleased to reach this regulatory milestone and believe that our NDA submission contains all of the necessary information to satisfy the FDA requirements,” said Patrick J. McEnany, Chairman and Chief Executive Officer of Catalyst Pharmaceuticals, Inc. “This important milestone is the culmination of a strong collaboration and commitment among the patients, physicians and Catalyst employees who have worked diligently to advance Firdapse and to further expand access to an FDA-approved product to all LEMS patients. We look forward to continuing to work with the FDA during the review process and to a potential future launch of Firdapse, if it is approved.”

Catalyst also reported on its decision not to include in this FDA submission those limited types of congenital myasthenic syndromes (CMS) that are considered mechanistically similar to LEMS. Catalyst reported that while it considered adding these indications to this NDA submission, after discussion with the FDA at its Type C meeting and further consideration, it decided not to overcomplicate the review of its NDA submission for LEMS with this second indication. Catalyst is currently conducting a Phase 3 trial evaluating Firdapse for the treatment of CMS, and expects to complete enrollment in this trial before the end of the year (and to report top-line results for its trial in the first quarter of 2019). Assuming the trial is successful, Catalyst plans to seek to add CMS to its label for Firdapse for a much broader population of CMS patients.

The NDA submission announced today addressed the two issues raised in the 2016 Refusal to File letter for the previous Firdapse NDA submission and included all additional information requested by the FDA. Once an NDA is submitted, the FDA has a 60-day filing review period to determine whether the NDA is complete and acceptable for filing, after which time the Agency will notify the company submitting the NDA of its decision. Catalyst plans to communicate the Agency’s decision once Catalyst has been notified.

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 Catalyst Pharmaceuticals

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, spinal muscular atrophy (SMA) type 3 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 the 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).

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 (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 FDA as support for an approval of Firdapse for the treatment of LEMS, (ii) whether the results of the abuse liability studies undertaken by Catalyst will be acceptable to the FDA as support for an approval of Firdapse, (iii) whether the NDA submitted for Firdapse will be accepted by the FDA, and the timing of any such acceptance, (iv) whether the receipt of breakthrough therapy designation for Firdapse will expedite the development and review of Firdapse by the FDA or the likelihood that the product will be found to be safe and effective, (v) whether, if an NDA for Firdapse is accepted for filing, such NDA will be given a priority review by the FDA, (vi) whether Firdapse will ever be approved for commercialization, (vii) whether Catalyst will be the first company to receive an approval for amifampridine (3,4-DAP), giving it 5-year marketing exclusivity for its product, and (viii) those other factors described in Catalyst’s Annual Report on Form 10-K for the fiscal year 2017 and its other filings with the U.S. Securities and Exchange Commission (SEC), could adversely affect Catalyst. Copies of Catalyst’s filings with the SEC are available from the SEC, may be found on Catalyst’s website, or may be obtained upon request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date.

Source: Catalyst Pharmaceuticals, Inc.

Posted: March 2018

Related Articles

Firdapse (amifampridine phosphate) FDA Approval History

Tagged with:

About author

Related Articles