Elelyso (taliglucerase alfa)
An approved treatment for Gaucher Disease.
The same compound appears under different names depending on the context. Here is how to identify Taliglucerase alfa wherever you encounter it, plus the key facts at a glance.
- Generic name
- Taliglucerase alfa
- Brand name
- Elelyso
- Development code
- prGCD
- Drug class
- Enzyme replacement therapy
- Manufacturer
- Pfizer / Protalix BioTherapeutics
- How it's taken
- Given as an intravenous infusion every 2 weeks at 60 units/kg.
An enzyme replacement therapy for Gaucher disease type 1, produced using a novel plant-cell-based manufacturing process (carrot cells) as an alternative to the mammalian-cell-derived Cerezyme.
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Where Taliglucerase alfa fits
Alternative enzyme replacement therapy for Gaucher disease type 1, produced in plant cells. Provides another option for patients on ERT alongside imiglucerase and velaglucerase alfa.
How Taliglucerase alfa works
Like Cerezyme, Elelyso provides the glucocerebrosidase enzyme to break down accumulated glucocerebroside. The key difference is manufacturing: it's produced in genetically engineered carrot cells rather than Chinese hamster ovary cells, which naturally produces the correct sugar structures for cellular uptake without additional processing.
Mechanism: Plant-cell-derived enzyme replacement therapy providing glucocerebrosidase
Side effects and safety
Elelyso has a boxed warning for severe allergic reactions, including life-threatening anaphylaxis, which can happen early in treatment or after long-term treatment. It should be started in a healthcare setting with emergency equipment available. Common side effects in adults include headache, joint pain, tiredness, nausea, dizziness, belly pain, itching, flushing, vomiting and hives; vomiting was the most common in children. Infusion reactions and antibody development can occur.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Taliglucerase alfa
Given as an intravenous infusion every 2 weeks at 60 units/kg. Each infusion lasts at least 60 minutes. Approved for patients 4 years and older with Type 1 Gaucher disease.
Availability and cost
Only available as the brand-name product.
Plant cell-derived enzyme replacement therapy, the first FDA-approved plant cell-expressed biologic. Alternative manufacturing approach using carrot cell culture.
Help paying for Elelyso
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Copay help
Elelyso Co-pay Program: eligible commercially insured patients may pay as low as $0 per prescription; annual savings up to $15,000.
For: private insurance · source - Free medicine program
Through the Pfizer Patient Assistance Program, ELELYSO may be available at no cost to qualified uninsured or government-insured patients who meet financial criteria.
For: no insurance, Medicare, Medicaid, TRICARE · source - Insurance and case manager help
A GPS Case Manager helps with benefits verification, prior authorization, appeals, insurance changes, and finding assistance programs.
The official page does not say who qualifies. Ask the program. · source
Good to know: Co-pay program excludes Medicare, Medicaid, TRICARE, VA and other government programs. GPS states it does not provide infusion administration support. Call 1-855-ELELYSO.
- From a charity · The Assistance FundGaucher Disease fundOpen
Pays for: Copays, coinsurance, deductibles and other health-related expenses.
The foundation says: “OPEN — Accepting New Patients. TAF is currently accepting new patient enrollments for this program.” - From a charity · TotalAssist (formerly PAN Foundation)Gaucher Disease fundOpen
Pays for: Out-of-pocket costs for approved medications, up to $14,000 per year. Requires health insurance (any kind).
- From a charity · NORD RareCareGaucher Disease Medical Assistance fundWaitlist
Pays for: Medical and medication costs.
The foundation says: “Temporarily Waitlisting” - From a charity · NORD RareCareGaucher Disease Premium Copay Assistance fundWaitlist
Pays for: Insurance premiums and copays.
The foundation says: “Temporarily Waitlisting”
Clinical trial results
Clinical trials showed comparable efficacy to imiglucerase in reducing spleen and liver volume and improving hemoglobin and platelet counts.
Development history
Approved by FDA in May 2012 for adults, expanded to pediatric patients in August 2014. First plant-cell-derived biologic approved by the FDA. Provides manufacturing diversity and an alternative source for enzyme replacement therapy.
Explore Gaucher Disease trials
Other Gaucher Disease treatments
Common questions about Taliglucerase alfa
▸What is Taliglucerase alfa (Elelyso)?
An enzyme replacement therapy for Gaucher disease type 1, produced using a novel plant-cell-based manufacturing process (carrot cells) as an alternative to the mammalian-cell-derived Cerezyme.
▸How does Taliglucerase alfa work?
Like Cerezyme, Elelyso provides the glucocerebrosidase enzyme to break down accumulated glucocerebroside. The key difference is manufacturing: it's produced in genetically engineered carrot cells rather than Chinese hamster ovary cells, which naturally produces the correct sugar structures for cellular uptake without additional processing.
▸What are the side effects of Taliglucerase alfa?
Elelyso has a boxed warning for severe allergic reactions, including life-threatening anaphylaxis, which can happen early in treatment or after long-term treatment. It should be started in a healthcare setting with emergency equipment available. Common side effects in adults include headache, joint pain, tiredness, nausea, dizziness, belly pain, itching, flushing, vomiting and hives; vomiting was the most common in children. Infusion reactions and antibody development can occur.
▸How is Taliglucerase alfa taken?
Given as an intravenous infusion every 2 weeks at 60 units/kg. Each infusion lasts at least 60 minutes. Approved for patients 4 years and older with Type 1 Gaucher disease.
▸Is Taliglucerase alfa FDA approved?
Yes, Taliglucerase alfa (Elelyso) is FDA approved (2012) for the treatment of Gaucher Disease.
▸What makes Elelyso's manufacturing unique?
Elelyso is produced in genetically engineered carrot cells rather than the Chinese hamster ovary (CHO) cells used for Cerezyme. This plant-cell system naturally produces the correct mannose-type sugar structures needed for cellular uptake, without additional processing steps.
▸Was Elelyso the first plant-cell-derived biologic?
Yes. When approved by the FDA in May 2012, Elelyso (taliglucerase alfa) became the first plant-cell-derived biologic approved by the FDA, establishing a new manufacturing paradigm for biological medicines.
▸How does Elelyso compare to Cerezyme?
Clinical trials showed comparable efficacy to imiglucerase (Cerezyme) in reducing spleen and liver volume and improving blood counts. The key difference is the manufacturing process. Both are given as IV infusions every 2 weeks at 60 units/kg.
▸Is Elelyso approved for children?
Yes. Elelyso was approved for adults in May 2012 and expanded to pediatric patients with Type 1 Gaucher disease in August 2014. The current label covers patients 4 years and older.
▸Why might a patient choose Elelyso over other ERTs?
Elelyso provides manufacturing diversity, which is important for supply chain security. During past Cerezyme manufacturing shortages, having alternative ERT sources was critical for Gaucher patients. The plant-cell manufacturing may also have advantages in avoiding mammalian cell contaminants.
▸Who manufactures Elelyso?
Elelyso is manufactured by Protalix BioTherapeutics using their proprietary ProCellEx plant-cell culture system and marketed by Pfizer.