Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a leader in the development of innovative therapies to treat seizure disorders, is proud to announce that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending approval of ZTALMY® (ganaxolone) oral suspension for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD). This groundbreaking therapy will be available for those aged 2-17, with the possibility for continuation to those aged 18 and up – offering patients hope for an improved quality of life.
The European Commission is now reviewing the CHMP’s positive opinion on ZTALMY, a potential treatment for the adjunctive treatment of seizures associated with CDD. If approved, ZTALMY will be the first of its kind to be available to EU citizens across all 27 member states plus Iceland, Norway and Liechtenstein, giving hope to CDD sufferers across the region. The EC is expected to render a final verdict within 67 days of receiving the CHMP recommendation, after which commercialization by Orion Corporation may soon commence.
Marinus Pharmaceuticals has submitted an application to the CDD for the approval of ZTALMY, a breakthrough drug to help reduce major motor seizure frequency in patients with epilepsy. The application is supported by extensive clinical data from a Phase 3, double-blind, placebo-controlled clinical trial, which found that patients treated with ZTALMY experienced a median reduction of 30.7% in 28-day major motor seizure frequency compared to those taking placebo (p=0.0036).
Additionally, in an open-label extension study, those patients taking ZTALMY for at least 12 months saw a median reduction of 49.6%. Furthermore, ZTALMY was found to be safe, effective and well tolerated with the most common side effects being somnolence, pyrexia, salivary hypersecretion and seasonal allergy. The results of the Marigold study were published in The Lancet Neurology in May 2022.
About CDKL5 Deficiency Disorder
CDKL5 deficiency disorder (CDD) is a devastating genetic disorder that is characterized by early-onset, severe seizures that are difficult to control, and impairments to neurodevelopment.2 This disease is caused by a mutation in the cyclin-dependent kinase-like 5 (CDKL5) gene, located on the X chromosome.
This gene produces a protein that plays a vital role in the normal development and functioning of the brain.3 Though this condition is rare, its effects can be devastating for those with CDD and their families.
About Marinus Pharmaceuticals
Marinus Pharmaceuticals is a pharmaceutical company that takes radical steps towards furthering the development of innovative treatments for seizure disorders. Their FDA-approved drug, ZTALMY® (ganaxolone) oral suspension CV, is used to treat seizures associated with CDKL5 deficiency disorder in patients over two years of age.
Ganaxolone, an effective neuroactive steroid GABAA receptor modulator, has many other potential applications – such as treating rare seizure disorders – and is being studied in a Phase 3 trial for tuberous sclerosis complex and refractory status epilepticus. This revolutionary compound is being developed in both intravenous and oral forms, ensuring enhanced accessibility to pediatric and adult patients in both chronic and acute settings.
About Orion Corporation
Orion is on a mission to build wellbeing across the globe! This Finnish pharmaceutical company develops, manufactures, and markets both human and veterinary pharmaceuticals, active pharmaceutical ingredients, and a range of self-care products. With a focus on oncology and pain, Orion’s proprietary products are designed to treat a range of ailments, including cancer, neurological diseases, and respiratory diseases. Their A and B shares are available for trading on Nasdaq Helsinki, giving everyone the opportunity to join in on Orion’s ambitious mission of global health and wellbeing.
Marinus Pharmaceuticals is excited to announce its expectations for the approval or marketing authorization of ZTALMY® (ganaxolone) oral suspension for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients two to 17 years of age.
The company is hopeful that this drug will show potential not only for the treatment of CDD, but for a range of seizure disorders. Marinus has outlined a comprehensive commercial and clinical strategy for ganaxolone, and is eager to see the results of their work as they progress along the development timeline.
Our success in the clinical development of ZTALMY depends on numerous factors, including the potential for regulatory approval from the European Commission. If the EC does not grant marketing authorization on the timeline that we have anticipated, it could have a significant impact on our future performance. Additionally, unexpected delays or outcomes in clinical trials, unexpected changes in regulatory policy, competitive conditions, and other factors can cause our goals and projections to shift.
To top it off, the impact of the COVID-19 pandemic on our business, the medical community, regulators, and the global economy adds an extra layer of uncertainty to our plans. Despite the various risks and challenges we face, we remain steadfast in our mission to bring ZTALMY to patients in need.