An Interview with Orphan Medical about Xyrem®
`
`Minneapolis, MN- February i 2, 2001
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`Waiting for Xyrerr~
`
`Persons with namolepsy, especially thee who experience cataplexy, are anxiously awaiting the decision by the Food and Drug
`Administration (FDA) on the approval of Xyr~m® as a treatment for the symptoms of narcolepsy. A decision is expected later this spring,
`as the Xyrem® New Drug Application has been given Pdority Review Status.
`
`As a result, there are a num~-er of questions being raised by those most interested in Xyrem®. Naturally, many of these hava come up in
`our regularly scheduled Narcolepsy Chats, so TalkAboutSleep contacted Orphan Medical, the company developing Xyrem, to get some
`answers. Several individuals from the Orphan Medical corporate statf were kind enough to respond, including CEO John Bullion, Chief
`Operating Officer William Houghton M.D., and Vice President of Marketing Patti Engel.
`
`The interview covered a number of topics, including availability, cost, medical education, and "how it works". We hope you enjoy reading
`this inte~ew and find the information helpful to you.
`
`Availability of Xyren~
`
`TaikAboutSleep: Can you summarize the current status of Xyrem and the NDA? When do you expect the FDA to complete its review?
`With FDA approval, how long will it be before Xyrem will be available for doctors to prescribe?
`
`Orphan Medical, John Bullion, CEO: Orphan Medical.filed the New Drug Application (NDA) on October 2, 2000, and the application was
`granted pdority review status on October 10, 2000. There will also be an advisory committee meeting on March 15, to discuss Xyrem.
`Based on the priodty status, the FDA is expected to provide a decision regarding the NDA by Apdl 2 of this year.
`
`The Company expects that Xyrem will be available for patients within a few weeks following the approval with a full launch campaign and
`education materials for physicians coming a few months after the approval.
`
`TalkAboutSleep: Will Xyrem be available in pharmacies? Can you describe how the distribution of Xyrem will occur?. Is this part of the
`FDA decision process? Or are there other regulatory groups invelved in this process?
`
`Orphan Medical, Patti Engel, Vice President: Xyrem will be aveilable through a specialty distribution system that will utilize a central
`pharmacy that will handle the delivery of medicine to the patients.
`
`To order Xyrem, a physician will write a prescription and fax that to the central pharmacy. That pharmacy ~11 precess~the prescription
`request, call the physician to vedfy the prescription, call the patient to assist them in gaining coverage fi’om their insurance company, and
`then set up a delivery time directly to the patient so that they may receive their medicine.
`
`This system was designed by Orphan Medical with assistance and input from State Scheduling authorities, experts in specialty
`distribution, drug diversion investigators, field law enforcement, narcolepsy patient groups, and pharmacists experienced in dealing with
`Scheduled medicines.
`
`TalkAboutSleep: What progress is being made on the state regulations where GHB is still "outlawed"? Will people who reside in states
`with stricter regulation than the federal rules simply not be able to use Xyrem?
`
`Orphan Medical, Patti Engel, Vice President: The federal law allows for patients to have Xyrem as prescribed by their doctor. As of this
`writing, many states have adopted this stance: a few are waiting for formal FDA approval. Once approved, patients can call 1-888-
`8ORPHAN to determine Xyrem’s status in their particular state.
`
`Pricing and Insurance
`
`TalkAboutSleep: What is the estimated cost to Narcolepsy patients? Is it likely that the drug will be covered by health insurers? Will
`Orphan Medical provide assistance to persons with narcolepsy in getting insurance coverage? Will Xyrem be available free or at a reduced
`cost to low-income individuals without insurance? How will this be don~;?
`
`Orphan Medical, Patti Engel, Vice President: Because the handling requirements are not yet finalized by the FDA, there is not yet a
`price established for ×yrem. That will be determined nearer the time of approval, if approved, Xyrem will likely be covered by many health
`insurance plans and our Reimbursement Services department will be a~ilable to assist patients that are working with their plans to obtain
`coverage.
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`AMNXYR 000007839
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` PAR1033
`IPR of U.S. Patent No. 8,457,988
` Page 1 of 2
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`For patients who are uninsured or need financial assistance to obtain Xyrem, there will be a patient assistance program administered
`through the National Organization for Rare Disorders (NQRD) and they will determine the eligibility for patients based on their financial
`status.
`
`How Does Xyrem Work?
`TatkAboutSleep: Originally Xyrem was expected to be a drug for the control of cataplexy for people who have Narcolepsy. We know from
`personal experiences that it can also he~p alleviate Excessive Daytime Sleepiness (EDS). Does Orphan Medical know exactly how Xyrem
`alleviates cataplexy? Is it a chemical effect or does it occur purely by improving sleep?
`
`Orphan Medical, William Houghton, MD, CO0: -[he ultimate mechanism of action is not known. Many of the biochemical changes in
`the brain have been identified in terms of modulation of levels of important chemicals and neurotransmitter functions in the brain. As well,
`pdmary effects of improved sleep architecture occur that may contribute to why Xyrem reaches its optimal results a~ter 8 to 12 weeks, it
`stands to reason that maintained changes in sleep contribute to improve daytime functioning, but further research into the science of
`narcolepsy is required.
`
`TalkAboutSleep: With the improvement in EDS that many with narcolepsy experience, has Xyrem been tested on those who don~. have
`cataplexy or those with a diagnosis of Idiopathic Hyper~omnolence? If so, have these patients also experienced improvement in their EDS?
`
`Orphan Medical, Will,am Houghton~ MD, (;OO: No, we~ been entirely focused on narcolepsy and entry into the clinical trials has been
`designed to include cataplexy as a diagnosis for narcolepsy. We’ve not investigated Xyrem for the potential use in other sleep disorders.
`
`TalkAboutSleep: Over the years of testing, have any negative side effects of Xyrem been found?
`
`Orphan Medical, William Houghton, MD, CO0: The side effects seen in the tdals have been generally minor in nature and tend to abate
`over time and have included headache, nausea, dizzir~ess, enuresis, and sleepwalking.
`
`Medical Education
`TalkAboutSleep: Unfortunately, so many of the doctors who treat patients with Narcolepsy around the count~ do not keep up with current
`research and are unawar~ of the success so many with Narcolepsy have had with Xyrem. Will Orphan Medical have information available to
`doctors? Will there be an education program for those who may be reluctant to prescribe it?
`
`Orphan Medical, Patti Engel, Vice President: Information on Xyrem will be available to physicians through printed material, participation
`at medical meetings, and through representatives of Orphan Medical that will be able to discuss the details of Xyrem.
`
`There is also a program called the Xyrem Physician Success Program that will ensure that a physician understands the unique nature of
`Xyrem and how the prescription and distribution process works to provide this medicine to the patients that need it in a responsible
`manner.
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`TalkAboutSleep: Thank you, Orphan Medical, f~.r answering these questions about Xyrem® for our audience.
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`AMNXYR 000007840
`
` PAR1033
`IPR of U.S. Patent No. 8,457,988
` Page 2 of 2
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