National Gaucher Foundation - Gaucher Disease
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Pfizer

FAQ - ELELYSO™ (taliglucerase alfa) for injection

1. What is ELELYSO™?
ELELYSO™ (taliglucerase alfa) for injection is a long-term enzyme replacement therapy (ERT) for adults with a confirmed diagnosis of Type 1 Gaucher disease that was approved by the United States (U.S.) Food and Drug Administration (FDA) on May 1, 2012. ELELYSO is available by prescription only.

ELELYSO is the first FDA-approved plant cell-based ERT indicated for the treatment of Gaucher disease.

2. How does ELELYSO work?
People with Gaucher disease, a rare genetic disorder, lack an enzyme called glucocerebrosidase. This causes an accumulation of a fatty substance called glucocerebroside in macrophage cells. The accumulation of these lipids within the cells can cause symptoms such as enlargement and malfunctioning of the liver, spleen, bone marrow and occasionally, the lung, kidney and intestine. ELELYSO catalyzes the hydrolysis of glucocerebroside molecules within the Gaucher disease cells. In clinical trials, ELELYSO reduced spleen and liver size and improved hemoglobin levels and platelet counts.

3. How is ELELYSO administered?
The recommended dose of ELELYSO is 60 Units/kg of body weight administered once every two weeks as a 60-120 minute intravenous infusion. Physicians can adjust dosages based of achievement and maintenance of each patient’s therapeutic goals. Clinical trials have evaluated doses ranging from 11 Units/kg to 73 Units/kg every other week.

People living with Gaucher disease currently being treated with imiglucerase can be switched to ELELYSO. Those previously treated on a stable dose of imiglucerase are recommended to begin treatment with ELELYSO at that same dose when they switch from imiglucerase to ELELYSO.

ELELYSO infusions can take place at an infusion center, doctor's office, or at a patient’s home.

4. What is the safety profile of ELELYSO?
As with any intravenous protein medicine, like enzyme replacement therapy (ERT), severe allergic reactions (including anaphylaxis) have been observed in patients treated with ELELYSO. If this occurs, your doctor may immediately discontinue ELELYSO. Patients who have experienced anaphylaxis to ELELYSO or another ERT should proceed with caution upon retreatment.

In addition, infusion reactions (including allergic reactions) – defined as a reaction occurring within 24 hours of the infusion – were the most commonly observed reactions to ELELYSO. The most commonly observed infusion reactions were headache, chest pain or discomfort, weakness, fatigue, hives, abnormal redness of the skin, increased blood pressure, back or joint pain, and flushing. Most of these reactions were mild and did not require treatment.

Management of infusion reactions is based on the type and severity of the reaction. Your doctor may manage infusion reactions by temporarily stopping the infusion, slowing the infusion rate, or treating with medications such as an antihistamine and/or a fever reducer. Treatment with antihistamines and/or corticosteroids prior to infusion with ELELYSO may prevent these reactions.

Please see full Prescribing Information enclosed.

Other common adverse reactions observed were upper respiratory tract infections, throat infection, flu, urinary tract infection, and pain in extremities.

As with all therapeutic proteins, including ERTs, there is a possibility of developing antibodies to ELELYSO. However, it is currently unclear whether this has an impact on the clinical response or adverse reactions. Patients with an immune response to other ERTs who are switching to ELELYSO should continue to be monitored for antibodies. Comparison of the frequency of antibodies across ERTs may be misleading.

If you are pregnant, or plan to become pregnant, you should talk to your doctor about potential benefits and risks.

5. What types of support programs does Pfizer provide for people living with Gaucher disease?
Pfizer has developed the Gaucher Personal Support™ (GPS) program, a specialized support program for people living with Gaucher disease taking ELELYSO™ (taliglucerase alfa) for injection. GPS also includes a specialty pharmacy that is the sole provider of ELELYSO.

The GPS Case Manager is one of the core elements of the GPS program. When a patient begins treatment with ELELYSO, he or she is connected with a case manager who becomes that patient’s primary contact to coordinate his or her personalized support through GPS.

GPS Case Managers and a team of both medical and non-medical professionals are all accessible through the same phone number. The case manager may be able to help with Gaucher disease and disease-related issues by working on behalf of the patient with fully trained and capable reimbursement specialists, nurses, and pharmacists. The GPS program may be able to help with:

  • $0 Co-pay program*
  • Reimbursement and finding financial assistance
  • 24/7 pharmacy and Gaucher disease nursing support
  • Coordinating infusion services including helping arrange at-home infusions (with doctor and insurance approval)

For more information about ELELYSO, please visit www.ELELYSO.com. Patients can call 1.855.ELELYSO (1-855-353-5976) for an information kit about ELELYSO and the GPS program.

6. How will Pfizer help Gaucher patients pay for treatment?
Pfizer’s Gaucher Personal Support (GPS) program offers reimbursement support services for people who may need assistance paying for ELELYSO. GPS features an assistance program for insured, uninsured, and underinsured patients. Most patients will be eligible for $0 out-of-pocket costs for ELELYSO.* The GPS Case Manager can also help find sources of reimbursement assistance through which a patient may qualify. For more information about specific patient qualifications, please see below.

___________________________
*ELELYSO $0 Co-pay Card Terms and Conditions apply. See page 3 for full Terms and Conditions.
This program is not health insurance
The co-pay program is available only through the Gaucher Personal Support program
• For any questions, please call Gaucher Personal Support at 1-855-ELELYSO (1-855-353-5976). Gaucher Personal Support, 17877 Chesterfield Airport Road, Chesterfield, MO 63005
• No membership fees required. Average co-pay savings per patient per year is $2,000. The maximum limit is $10,000 per year or the amount of the co-pay you paid, whichever is less

Please see full Prescribing Information enclosed.

7. Does Pfizer have a protocol in place to address supply issues?
In order to help minimize any future supply disruptions, Pfizer developed the “Supply Continuity Program.” Through this program, Pfizer plans to maintain a rolling 24 months of supply at various stages of production around the world for U.S. patients prescribed ELELYSO. This program was developed to help fill a need within the Gaucher community regarding shortage disruptions.

Terms and Conditions
By using the co-pay program, you acknowledge that you currently meet the eligibility criteria and will comply with the terms and conditions described below:

1. This co-pay program is not valid for prescriptions that are eligible to be reimbursed, in whole or in part, by Medicaid, Medicare or other federal or state healthcare programs (including any state prescription drug assistance programs and the Government Health Insurance Plan available in Puerto Rico [formerly known as “La Reforma de Salud”]).
2. This co-pay program is not valid for prescriptions that are eligible to be reimbursed by private insurance plans or other health or pharmacy benefit programs which reimburse you for the entire cost of your prescription drugs.
3. 100% of prescription co-pays will be covered for majority of patients. Average co-pay savings is $2,000 per year. The maximum limit is $10,000 per year or the amount of the co-pay you paid, whichever is less.
4. You must deduct the value received under this program from any reimbursement request submitted to your insurance plan, either directly by you or on your behalf.
5. This co-pay program is not valid for Massachusetts residents whose prescriptions are covered in whole or in part by third party insurance, or where otherwise prohibited by law.
6. Cannot be combined with any other rebate/coupon, free trial or similar offer for the specified prescription.
7. This program is not health insurance.
8. The co-pay program is available only through the Gaucher Personal Support program. For any questions, please contact Gaucher Personal Support at 1-855-ELELYSO (1-855-353-5976). Gaucher Personal Support, 17877 Chesterfield Airport Road, Chesterfield, MO 63005.
9. Offer good only in the US and Puerto Rico.
10. Pfizer reserves the right to rescind, revoke or amend the program without notice.
11. This co-pay program expires 12/31/2013.

Please see full Prescribing Information enclosed.