BioMarin

BioMarin

Biopharmaceutical company based in Marin County, California that develops therapeutics for rare genetic diseases.

BioMarin is a biopharmaceutical company that develops therapeutics for rare genetic diseases. The company focuses on conditions that affect small patient populations, most of whom are children.

BioMarin’s cGMP manufacturing facility is in Novato, California. In 2011 the company acquired a biologics manufacturing plant from Pfizer in Shanbally, Cork, Ireland. BioMarin also operates process development and pilot scale laboratories to evaluate investigational product candidates.

Therapeutic products

  • Palynziq (pegvaliase-pqpz) Injection self-administered enzyme substitution therapy for PKU
  • Kuvan (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution for PKU
  • Brineura (cerliponase alfa) for CLN2 Disease
  • Vimizim (elosulfase alfa) for Morquio A Syndrome
  • Naglazyme (galsulfase) for MPS VI
  • Firdapse (amifampridine phosphate) (only approved in the EU) for LEMS
  • Aldurazyme (laronidase) for MPS I

Clinical pipeline

  • Vosoritide (BMN 111) is designed for the treatment of achondroplasia, the most common form of dwarfism
  • Tralesinidase alfa (BMN 250) for MPS IIIB is an enzyme replacement therapy being developed to treat Sanfilippo B Syndrome

Gene therapy

  • Valoctogene roxaparvovec (valrox) is a gene therapy for hemophilia in clinical trials as of 2019
  • BMN 307 for PKU, plans to file an application for beginning clinical trials for was reported in 2019.

Timeline

June 1, 2017

European Medicines Agency approves Brineura as a treatment for CLN2 disease

April 27, 2017

Food & Drug Administration (FDA) approves Brineura as a treatment for CLN2 disease

People

Name
Role
LinkedIn

Jean-Jacques Bienaimé

Chairman and CEO

Further reading

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Documentaries, videos and podcasts

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Companies

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News

Title
Author
Date
Publisher
Description
Amirah Al Idrus
June 18, 2020
FierceBiotech
Four years on, BioMarin's hemophilia A gene therapy is still going strong--it staved off bleeding episodes in patients with hemophilia A and reduced their reliance on infusions of a blood-clotting factor that they lack.

References

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