Jazz Pharmaceuticals: Tackling Unmet Medical Needs
Jazz Pharmaceuticals has been busy. Founded in 2003, the international biotechnology company has been methodically building its portfolio of meaningful products that address unmet patient medical needs in focused areas – most notably in sleep medicine and in hematology/oncology.
“Since our founding, we have focused on putting the patient first and this remains our priority as we continue to develop, acquire and commercialize new treatment options for patients with very challenging conditions that no one else is tackling,” says Chairman and CEO Bruce Cozadd.
One of Jazz’s growth driving products – Xyrem® (sodium oxybate) – treats excessive daytime sleepiness and cataplexy, the two primary symptoms associated with narcolepsy, an orphan condition that severely impacts patients’ quality of life. Narcolepsy is a chronic neurological disorder that is highly misunderstood and can take up to 10 year to diagnose. The condition can be extremely debilitating for patients, as it makes even daily routines difficult.
“Living with narcolepsy is so much more than just feeling tired or sleepy, it often makes it difficult for patients to hold down a job or live a normal life” notes Cozadd. “Patients often experience the pervasive need to fall asleep when doing normal daily activities such as driving a car.”
The company also has a drug (JZP-110) in clinical trials for excessive daytime sleepiness in both narcolepsy and obstructive sleep apnea.
“We know that about 25,000 of the 50,000 diagnosed and treated narcolepsy patients, and about five to ten times more diagnosed and treated obstructed sleep apnea patients, don’t get adequate relief for their excessive daytime sleepiness from existing therapies, or cannot tolerate them,” says Cozadd. “Based on two phase II trials, we are optimistic that JZP-110, a wake-promoting experimental therapy, may provide another option for these patients.”
Jazz Pharmaceuticals also markets Erwinaze® (asparaginase Erwinia chrysanthemi), a form of asparaginase therapy used to treat acute lymphoblastic leukemia (ALL), one of the most common pediatric cancers. ALL is often treated with E. coli-derived asparaginase, in combination with other therapies, but some children develop an allergy to E.coli-derived asparaginase. Erwinaze offers this small group of patients an alternative asparaginase treatment, which allows them to complete their course of therapy.
Jazz Pharmaceuticals’ growing R&D pipeline includes a number of products at various stages of development. The company is excited about one of its new products in the EU, Defitelio®▼(defibrotide). Defitelio is the first and only licensed product in the EU to treat severe hepatic veno-occlusive disease (sVOD), a complication for patients receiving stem cell transplants. Jazz is currently in discussions with the FDA regarding the use of defibrotide in the U.S. They are hoping to submit a new drug application in the first half of 2015.
“Patients who undergo stem cell transplants and develop severe VOD often face a hundred-day mortality rate of 80 percent,” says Cozadd. “Defitelio, the only treatment approved for severe VOD, has been shown to significantly increase these patients’ survival. We are optimistic about this product’s potential to impact patients’ lives and we are working hard to get defibrotide approved in the U.S. as well.”
Growth and Expansion
Jazz Pharmaceuticals is an international biopharmaceutical company with offices in Dublin, Ireland (corporate headquarters); Palo Alto, California; Philadelphia, Pennsylvania; Oxford (England); and Villa Guardia (Italy). Jazz Pharmaceuticals has a manufacturing facility in Italy and is building another in Ireland.
“We continue to expand our business to be able to offer more differentiated products that can help improve patients’ outcomes,” says Cozadd. “The success of our core commercial franchises affords us the ability to continue to invest in business development opportunities and in an R&D pipeline focused on future opportunities.”
“In light of our rapid growth, continuing to make Jazz a great place to work remains very important to me. Jazz employees are motivated by what we can do to make a difference in the lives of patients,” says Cozadd.
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