Welcome!

News Feed Item

Insys Therapeutics Receives FDA Orphan Drug Designation for Its Pharmaceutical Cannabidiol as a Potential Treatment for Dravet Syndrome, a Rare Form of Epilepsy

PHOENIX, AZ -- (Marketwired) -- 07/02/14 -- Insys Therapeutics, Inc. (NASDAQ: INSY), a specialty pharmaceutical company that is developing and commercializing innovative drugs and novel drug delivery systems, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its pharmaceutical cannabidiol (CBD) for the treatment of Dravet syndrome, a rare pediatric-onset epilepsy.

"There is presently no cure for this catastrophic form of epilepsy, and the significant, unmet need is recognized by the orphan drug designation we received today for our pharmaceutical CBD," said Michael L. Babich, President and Chief Executive Officer. "Our pharmaceutical CBD is an alternative to plant derived cannabinoids, one which we believe will provide significant medical benefits and better address the unmet needs of patients across multiple indications including Dravet syndrome. We expect to file an Investigational New Drug Application (IND) for CBD in the second half of 2014."

Insys has more than seven years of research and development experience in the pharmaceutical cannabinoid space. The company manufactures pharmaceutical dronabinol (THC) and pharmaceutical CBD, both of which are cannabinoids, at its FDA-inspected and Drug Enforcement Administration (DEA) approved facility in Round Rock, Texas. Insys believes it is the only U.S.-based company with the capacity to produce pharmaceutical cannabinoids in scalable quantities.

In addition to Dravet syndrome, Insys plans to develop treatments for another rare form of pediatric epilepsy, Lennox-Gastaut syndrome, for which its pharmaceutical CBD recently received orphan drug designation. Insys is also evaluating the potential use of pharmaceutical CBD in several additional indications, including: adult epilepsy; chemotherapy-induced peripheral neuropathy; addiction in cocaine, heroin and opioids; and glioblastoma. Insys intends to pursue orphan drug designation for other indications that may qualify.

Orphan drug designation is granted by the FDA Office of Orphan Products Development (OOPD) to novel drugs or biologics that treat rare diseases or conditions affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity, as well as certain financial incentives that can help support its development.

About Pharmaceutical CBD
Cannabidiol (CBD) is one of at least 60 active cannabinoids identified in cannabis. CBD is considered to have a wider scope of medical applications than tetrahydrocannabinol (THC), and appears to have anti-convulsive action in animal models.

Insys' pharmaceutical CBD is over 99.5% pure cannabidiol. It is synthesized to be identical to the CBD extracted from cannabis, and is produced in a controlled environment. The FDA issued Drug Master File (DMF) #28255 to Insys for its CBD active pharmaceutical ingredient in May 2014.

About Dravet Syndrome
Dravet syndrome, also known as Severe Myoclonic Epilepsy of Infancy (SMEI), is a rare and catastrophic form of intractable epilepsy that begins in infancy. Initial seizures are most often prolonged events and in the second year of life other seizure types begin to emerge. Development remains on track initially, with plateaus and a progressive decline typically beginning in the second year of life. Individuals with Dravet syndrome face a higher incidence of SUDEP (sudden unexplained death in epilepsy) and have associated conditions, which also need to be properly treated and managed.

About Insys Therapeutics, Inc.
Insys Therapeutics is a specialty pharmaceutical company that develops and commercializes innovative drugs and novel drug delivery systems of therapeutic molecules that improve the quality of life of patients. Using its proprietary sublingual spray technology and its capability to develop pharmaceutical cannabinoids, the company addresses the clinical shortcomings of existing commercial products. The company currently markets two products, Subsys, which is sublingual Fentanyl spray for break through cancer pain, and a generic version of Dronabinol (THC) capsules. The company plans to file a New Drug Application (NDA) for an oral liquid formulation of Dronabinol in the second half of 2014 and believes it is a clinically superior product to current Dronabinol capsules. The company is developing a pipeline of sublingual sprays, as well as pharmaceutical CBD.

Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding our expectations related to filing at least one Investigational New Drug application with the Food and Drug Administration in the second half of 2014 for pharmaceutical CBD, our plan to pursue the development of treatments for Lennox-Gastaut syndrome (LGS) and Dravet syndrome related to epilepsy, the potential benefits of our pharmaceutical CBD therapies to patients and our intention to pursue orphan drug designation for other indications that may qualify for orphan drug status. These forward-looking statements are based on management's expectations and assumptions as of the date of this press release, and actual results may differ materially from those in these forward-looking statements as a result of various factors, many of which are beyond our control. For a description of these risks facing the company, please see the risk factors described in our filings with the United States Securities and Exchange Commission, including those factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2013 and any subsequent updates that may occur in our Quarterly Reports on Form 10-Q. Forward-looking statements speak only as of the date of this press release and we undertake no obligation to update or revise these statements, except as may be required by law.

More Stories By Marketwired .

Copyright © 2009 Marketwired. All rights reserved. All the news releases provided by Marketwired are copyrighted. Any forms of copying other than an individual user's personal reference without express written permission is prohibited. Further distribution of these materials is strictly forbidden, including but not limited to, posting, emailing, faxing, archiving in a public database, redistributing via a computer network or in a printed form.

Latest Stories
"Once customers get a year into their IoT deployments, they start to realize that they may have been shortsighted in the ways they built out their deployment and the key thing I see a lot of people looking at is - how can I take equipment data, pull it back in an IoT solution and show it in a dashboard," stated Dave McCarthy, Director of Products at Bsquare Corporation, in this SYS-CON.tv interview at @ThingsExpo, held November 1-3, 2016, at the Santa Clara Convention Center in Santa Clara, CA.
In IT, we sometimes coin terms for things before we know exactly what they are and how they’ll be used. The resulting terms may capture a common set of aspirations and goals – as “cloud” did broadly for on-demand, self-service, and flexible computing. But such a term can also lump together diverse and even competing practices, technologies, and priorities to the point where important distinctions are glossed over and lost.
What happens when the different parts of a vehicle become smarter than the vehicle itself? As we move toward the era of smart everything, hundreds of entities in a vehicle that communicate with each other, the vehicle and external systems create a need for identity orchestration so that all entities work as a conglomerate. Much like an orchestra without a conductor, without the ability to secure, control, and connect the link between a vehicle’s head unit, devices, and systems and to manage the ...
All clouds are not equal. To succeed in a DevOps context, organizations should plan to develop/deploy apps across a choice of on-premise and public clouds simultaneously depending on the business needs. This is where the concept of the Lean Cloud comes in - resting on the idea that you often need to relocate your app modules over their life cycles for both innovation and operational efficiency in the cloud. In his session at @DevOpsSummit at19th Cloud Expo, Valentin (Val) Bercovici, CTO of Soli...
Complete Internet of Things (IoT) embedded device security is not just about the device but involves the entire product’s identity, data and control integrity, and services traversing the cloud. A device can no longer be looked at as an island; it is a part of a system. In fact, given the cross-domain interactions enabled by IoT it could be a part of many systems. Also, depending on where the device is deployed, for example, in the office building versus a factory floor or oil field, security ha...
Amazon has gradually rolled out parts of its IoT offerings in the last year, but these are just the tip of the iceberg. In addition to optimizing their back-end AWS offerings, Amazon is laying the ground work to be a major force in IoT – especially in the connected home and office. Amazon is extending its reach by building on its dominant Cloud IoT platform, its Dash Button strategy, recently announced Replenishment Services, the Echo/Alexa voice recognition control platform, the 6-7 strategic...
"Qosmos has launched L7Viewer, a network traffic analysis tool, so it analyzes all the traffic between the virtual machine and the data center and the virtual machine and the external world," stated Sebastien Synold, Product Line Manager at Qosmos, in this SYS-CON.tv interview at 19th Cloud Expo, held November 1-3, 2016, at the Santa Clara Convention Center in Santa Clara, CA.
Without a clear strategy for cost control and an architecture designed with cloud services in mind, costs and operational performance can quickly get out of control. To avoid multiple architectural redesigns requires extensive thought and planning. Boundary (now part of BMC) launched a new public-facing multi-tenant high resolution monitoring service on Amazon AWS two years ago, facing challenges and learning best practices in the early days of the new service. In his session at 19th Cloud Exp...
Everyone knows that truly innovative companies learn as they go along, pushing boundaries in response to market changes and demands. What's more of a mystery is how to balance innovation on a fresh platform built from scratch with the legacy tech stack, product suite and customers that continue to serve as the business' foundation. In his General Session at 19th Cloud Expo, Michael Chambliss, Head of Engineering at ReadyTalk, discussed why and how ReadyTalk diverted from healthy revenue and mor...
As data explodes in quantity, importance and from new sources, the need for managing and protecting data residing across physical, virtual, and cloud environments grow with it. Managing data includes protecting it, indexing and classifying it for true, long-term management, compliance and E-Discovery. Commvault can ensure this with a single pane of glass solution – whether in a private cloud, a Service Provider delivered public cloud or a hybrid cloud environment – across the heterogeneous enter...
You have great SaaS business app ideas. You want to turn your idea quickly into a functional and engaging proof of concept. You need to be able to modify it to meet customers' needs, and you need to deliver a complete and secure SaaS application. How could you achieve all the above and yet avoid unforeseen IT requirements that add unnecessary cost and complexity? You also want your app to be responsive in any device at any time. In his session at 19th Cloud Expo, Mark Allen, General Manager of...
Financial Technology has become a topic of intense interest throughout the cloud developer and enterprise IT communities. Accordingly, attendees at the upcoming 20th Cloud Expo at the Javits Center in New York, June 6-8, 2017, will find fresh new content in a new track called FinTech.
"We are a leader in the market space called network visibility solutions - it enables monitoring tools and Big Data analysis to access the data and be able to see the performance," explained Shay Morag, VP of Sales and Marketing at Niagara Networks, in this SYS-CON.tv interview at 19th Cloud Expo, held November 1-3, 2016, at the Santa Clara Convention Center in Santa Clara, CA.
@DevOpsSummit taking place June 6-8, 2017 at Javits Center, New York City, is co-located with the 20th International Cloud Expo and will feature technical sessions from a rock star conference faculty and the leading industry players in the world. @DevOpsSummit at Cloud Expo New York Call for Papers is now open.
Keeping pace with advancements in software delivery processes and tooling is taxing even for the most proficient organizations. Point tools, platforms, open source and the increasing adoption of private and public cloud services requires strong engineering rigor - all in the face of developer demands to use the tools of choice. As Agile has settled in as a mainstream practice, now DevOps has emerged as the next wave to improve software delivery speed and output. To make DevOps work, organization...