Taliglucerase alfa

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Taliglucerase alfa
Names
Trade namesElelyso, Uplyso, others
Clinical data
Drug classEnzyme[1]
Main usesGaucher's disease[1]
Side effectsHeadache, joint pain, tiredness, nausea, itchiness, rash[1]
Pregnancy
category
  • AU: B1
  • US: N (Not classified yet)
Routes of
use
Intravenous infusion
Typical dose60 units/kg[1]
External links
AHFS/Drugs.comMonograph
US NLMTaliglucerase alfa
Legal
Legal status
  • US: ℞-only
  • In general: ℞ (Prescription only)
Pharmacokinetics
Elimination half-life18.9-28.7 minutes
Chemical and physical data
FormulaC2580H3918N680O727S17
Molar mass56638.78 g·mol−1

Taliglucerase alfa, sold under the brand name Elelyso among others, is a medication used to treat Gaucher's disease type I.[1] It is given by gradual injection into a vein.[1] It is used long term.[1]

Common side effects include headache, joint pain, tiredness, nausea, itchiness, and rash.[1] Other side effects may include anaphylaxis.[1] It is a glucocerebrosidase made by recombinant DNA technology and works by replacing this missing enzyme.[1]

Taliglucerase alfa was approved for medical use in the United States in 2012.[1] In Europe it received an orphan designation in 2010 but was declined approval in 2012 due to the similar product velaglucerase alfa having market exclusivity.[2] In the United States it costs about 18,000 USD per dose for a 70 kg person (465,000 USD per year).[3] It is the first plant-made medication to be approved in the United States.[4][5]

Medical uses

Dosage

It is given at a dose of 60 units per kg every two weeks.[1] The typical dose for a 70 kg person is thus 4,200 units.

Each vial has 200 units of taliglucerase alfa.

History

The U.S. FDA New Drug Application (NDA) was granted approval in May 2012, for use in adults.[6][7] The U.S. FDA Supplemental New Drug Application (sNDA) for pediatric use was granted approved in August 2014.[8] In Israel, the Israeli Ministry of Health granted approval in September 2012.[9] In Brazil, the Brazilian Health Surveillance Agency (ANVISA) granted approval in March 2013. In Canada, Health Canada issued a Notice of Compliance in May 2014, for both adults and pediatric patients.[10]

Taliglucerase alfa is made by the Israeli biotherapeutics company Protalix and sold by the American pharmaceutical company Pfizer.[citation needed]

Society and culture

Economics

For 2016, Elelyso was ranked third for pharmaceuticals with the highest cost-per-patient, with an average cost of $483,242 per year.[11]

References

  1. 1.00 1.01 1.02 1.03 1.04 1.05 1.06 1.07 1.08 1.09 1.10 1.11 "Taliglucerase Alfa Monograph for Professionals". Drugs.com. Retrieved 21 September 2021.
  2. "Elelyso". Retrieved 21 September 2021.
  3. "Elelyso Prices, Coupons & Patient Assistance Programs". Drugs.com. Retrieved 21 September 2021.
  4. Kaiser J (April 2008). "Is the drought over for pharming?". Science. 320 (5875): 473–5. doi:10.1126/science.320.5875.473. PMID 18436771. S2CID 28407422.
  5. Maxmen A (2 May 2012). "First plant-made drug on the market". Nature, Biology & Biotechnology, Industry. Retrieved 26 June 2012.
  6. Beitz J. "Elenyso (taliglucerase alfa) NDA Approval" (PDF). Center for Drug Evaluation and Research. U.S. Food and Drug Administration.
  7. "Elenyso (taliglucerase alfa): Highlights of Prescribing Information" (PDF). U.S. Food and Drug Administration.
  8. "Supplement Approval Fulfillment of Postmarketing Requirement" (PDF). U.S. Food and Drug Administration.
  9. "מאגר התרופות (Drug details for Elelyso)". Ministry of Health Israel.[dead link]
  10. "Summary Basis of Decision (SBD): Elelyso". Health Canada. 2014-08-01. Archived from the original on 2014-08-06.
  11. "Medical Pharmacy Trend Report" (PDF). Magellan Rx Management (seventh ed.). 2016. Archived from the original (PDF) on 29 March 2019.

External links

Identifiers: