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Advancing the neuroscience frontier to help more patients

For the last two decades, Jazz Pharmaceuticals has focused on transforming the lives of patients and their families by delivering life-changing medicines for individuals with serious neurological and psychiatric conditions. We strive to expand research and development to drive innovation and open paths to new treatment options, while listening to the needs of underserved patient populations that historically may have had little to no options for treatment or symptom management.

This focus has informed our multi-disciplinary approach to finding new solutions for the patients who need them most. Guided by the patient voice and science, we are advancing an industry-leading neuroscience pipeline through in-house scientific research, acquisition opportunities and partnerships with researchers and academia to advance molecules.

Many of the therapies we’ve developed are designed for traditionally underserved patient populations living with lifelong conditions that make everyday life profoundly difficult, such as developmental and epileptic encephalopathies, narcolepsy or idiopathic hypersomnia. We are poised to enter new areas with serious unmet patient need, including movement disorders, like essential tremor and tremor in Parkinson’s disease, and psychiatric conditions, like post-traumatic stress disorder (PTSD).

A Pipeline that Focuses on Patients with High Unmet Need

From the beginning, we’ve tackled rare, hard-to-treat disorders in the face of challenges in several pioneering ways. From the development and launch of the first-ever U.S. Food and Drug Administration (FDA)-approved cannabidiol (CBD) treatment for pediatric seizure disorders to the first-ever FDA-approved treatment for adults with idiopathic hypersomnia, we are driven to deliver innovative medicines to patients with high unmet need. Through our diverse portfolio and pipeline across neuroscience, we apply this innovation-first mentality and patient-focused approach to deliver transformative therapies.

PTSD is experienced by about 7 out of 100 Americans and can have a debilitating effect on a person’s ability to participate in daily life.1 Yet, despite efforts put forth by the biopharmaceutical industry and academic researchers alike, limited progress has been made in treating PTSD, with no new pharmacotherapies approved in over 20 years.

Jazz is taking a different approach by looking into novel mechanisms of action. Currently, we are examining a fatty acid amide hydrolase (FAAH) inhibitor for the treatment of PTSD. Anandamide (AEA) is the brain’s major endogenous (a neurochemical or neurotransmitter made by the brain) cannabinoid. AEA is an important regulator of the activity and function of many neurons, or nerve cells, throughout the brain. Levels of AEA in the brain are normally carefully regulated by the protein, or enzyme, FAAH. There are some illnesses that are associated with higher-than-normal activity of FAAH which causes lower levels of AEA. PTSD is one condition that has been shown to be associated with lower-than-normal levels of AEA.

Through the inhibition of FAAH and the subsequent potential increase in AEA, we aim to address the underlying pathophysiology and symptoms of PTSD. This approach is different from existing therapies that focus on symptom reduction of PTSD. By doing so, we hope to improve the lives of patients who are most severely impacted by the debilitating condition.

Essential tremor (ET) is the most common movement disorder, affecting close to 7 million patients in the US alone.2,3,4 ET most commonly affects the hands and arms, but tremor can also occur in the head, voice and lower limbs.2,5,6 ET can be highly debilitating with significant effects on a patient’s quality of life and activities of daily living, such as eating, dressing, and writing. No novel treatments addressing the symptoms of the disease have been introduced in over 50 years. It is estimated that up to half of patients do not respond to available generic treatments, and over 50% of patients reportedly discontinue medication due to lack of efficacy and/or side effects.7 There is a clear and urgent need for new treatment options for patients living with essential tremor.

Currently, Jazz Pharmaceuticals is exploring the use of a selective modulator of T-type calcium channels to target aberrant brain signals at their source to reduce tremor, for the treatment of moderate-to-severe essential tremor. Many neurologic disorders are characterized by abnormal rhythms in certain regions of the brain mediated by T-type calcium channels, which are important for controlling movement. The mechanism through which our investigational therapy is thought to improve tremor in patients creates the potential for this therapeutic to be studied in other disorders such as tremors associated with Parkinson’s disease.

The Path Ahead

By applying our pioneering approach and deep understanding in sleep and seizure disorders to related disease areas, we are working hard to transform scientific discoveries into novel medicines and redefine what’s possible – in the lives of our patients and in science itself.

References

  1. National Institute of Mental Health. Post-Traumatic Stress Disorder (PTSD). National Institute of Mental Health (NIMH). Accessed February 28, 2023. https://www.nimh.nih.gov/health/statistics/post-traumatic-stress-disorder-ptsd.
  2. Song P, Zhang Y, Zha M, et al. The global prevalence of essential tremor, with emphasis on age and sex: A meta-analysis. J Glob Health. 2021;11:04028. Published 2021 Apr 10. doi:10.7189/jogh.11.04028.
  3. Crawford S, Lally C, Petrillo J, Paskavitz J, Louis E. How many adults in the US have essential tremor? Using data from epidemiological studies to derive age-specific estimates of prevalence. Neurology.2020;94(15 Supplement):4458.
  4. Louis ED, Ottman R. How many people in the USA have essential tremor? Deriving a population estimate based on epidemiological data. Tremor Other Hyperkinet Mov (N Y). 2014;4:259. Published 2014 Aug 14. doi:10.7916/D8TT4P4B.
  5. Essential Tremor Information Page. National Institute of Neurological Disorders and Stroke. https://www.ninds.nih.gov/Disorders/All-Disorders/Essential-Tremor-Information-Page. Modified March 27, 2019. Accessed October 2021.
  6. Bhatia KP, Bain P, Bajaj N, et al. Consensus Statement on the classification of tremors. from the task force on tremor of the International Parkinson and Movement Disorder Society. Mov Disord. 2018;33(1):75-87. doi:10.1002/mds.27121.
  7. Jazz Pharmaceuticals, Inc. Data on file.