BioMarin – therapies for rare genetic diseases

BioMarin is a biotech company focusing on the delivery of first-to-market therapies for patients with rare genetic diseases.

BioMarin – therapies for rare genetic diseases


BioMarin is a biopharma company developing and commercializing innovative biopharmaceuticals for serious diseases and medical conditions. BioMarin has already five products on the market:

  • VIMIZIM (elosulfase alfa) for Morquio A syndrome (MPS IVA)
  • Kuvan (sapropterin dihydrochloride) Tablets for phenylketonuria (PKU), developed in partnership with Merck Serono
  • Naglazyme (galsulfase) for MPS VI, a product which BioMarin developed with Genzyme through a 50/50 joint venture
  • Aldurazyme (laronidase) for MPS I
  • Firdapse (amifampridine phosphate), currently approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS)

BioMarin is also developing several candidates products:

  • PEG PAL (PEGylated recombinant phenylalanine ammonia lyase) for the treatment of PKU is currently in Phase 3 clinical development
  • BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor, is currently in Phase 3 clinical development for the treatment of germline BRCA breast cancer
  • BMN 701 is a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA) for the treatment of Pompe disease, currently in Phase 1/2 clinical development
  • BMN 111 (a modified C-natriuretic peptide for the treatment of achondroplasia) is currently in Phase 1 clinical development
  • BMN 190 is a recombinant human tripeptidyl peptidase-1 (rhTPP1) is currently in Phase 1 for the treatment of late-infantile neuronal ceroid lipofuscinosis (CLN2), a form of Batten Disease
  • BMN 270 (an AAV-factor VIII vector) is currently tested for the treatment of hemophilia A
  • BMN 250 is a novel fusion of alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2) for the treatment of MPS IIIB

Founded in 1997, BioMarin is now supported by approximately 1.500 employees worldwide. Management Team is composed by Bryan Lawlis, Kenneth Bate, Alan Lewis, Michael Grey, Richard Meier, Elaine Heron, Dennis Slamon, Jean-Jacques Bienaimé, Robert Baffi, Henry Fuchs, William Young , Pierre Lapalme, Dan Spiegelman, Jeff Ajer, Eric Davis, Daniel Maher, Joshua Grass, Ed Von Pervieux, Philip Lo Scalzo and Richard Ranieri. BioMarin already developed and commercialized five products, a remarkable testament of the company’s dedication to patients with serious, unmet medical needs.

More about BioMarin :

BioMarin – rare genetic diseases – rare genetic disease – therapies for rare genetic disease – biopharmaceutical – VIMIZIM – Kuvan – Naglazyme – Aldurazyme – Firdapse – PEG PAL – BMN 673 – BMN 701 – BMN 111 – BMN 190 – BMN 270 – BMN 250

BioMarin – Bryan Lawlis – Kenneth Bate – Alan Lewis – Michael Grey – Richard Meier – Elaine Heron – Dennis Slamon – Jean-Jacques Bienaimé – Robert Baffi – Henry Fuchs – William Young – Pierre Lapalme – Dan Spiegelman – Jeff Ajer – Eric Davis – Daniel Maher – Joshua Grass – Ed Von Pervieux – Philip Lo Scalzo – Richard Ranieri – elosulfase alfa – Morquio A syndrome – MPS IVA – sapropterin dihydrochloride – Kuvan Tablets -phenylketonuria – PKU – Merck Serono – galsulfase – MPS VI – Genzyme – laronidase – MPS I – amifampridine phosphate – Lambert Eaton Myasthenic Syndrome -LEMS – PEGylated recombinant phenylalanine ammonia lyase – BMN673 – poly ADP-ribose polymerase inhibitor – PARP inhibitor – PARP – germline BRCA breast cancer – BMN701 – insulin-like growth factor 2 – acid alpha glucosidase – IGF2-GAA – Pompe disease – BMN111 – modified C-natriuretic peptide – achondroplasia – BMN190 – recombinant human tripeptidyl peptidase-1 – rhTPP1 – late-infantile neuronal ceroid lipofuscinosis – CLN2 – Batten Disease – BMN270 – AAV-factor VIII vector – hemophilia A – BMN250 – alpha-N-acetyglucosaminidase – NAGLU – IGF2 – MPS IIIB