More than 20 years developing therapies for rare genetic disorders.
Each therapy we pursue is guided by a fundamental understanding of the genetics and underlying biology of the condition it is designed to address. The result: seven first or best-in-class treatments with another four in development.
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Please see full prescribing information including boxed warning for risk of anaphylaxis.
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Please see full prescribing information.
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Please see full prescribing information.
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Please see full prescribing information including boxed warning for risk of anaphylaxis.
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Please see full prescribing information.
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Please see full prescribing information including boxed warning for risk of anaphylaxis.
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Vosoritide (BMN 111) Analog of CNP*AchondroplasiaNDA/MAATarget Indication
Vosoritide is an investigational analog of C-type Natriuretic Peptide under investigation for the treatment of children with achondroplasia, the most common form of skeletal dysplasia.
* Vosoritide has been approved in the European Union and is marketed as VOXZOGO™ (vosoritide). FDA review of the vosoritide NDA is ongoing and it has not been approved for use in the United States.
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Valoctocogene Roxaparvovec (BMN 270) AAV-Factor VIIISevere Hemophilia APhase 3Target Indication
Valoctocogene roxaparvovec is an investigational AAV5 gene therapy under regulatory review for the treatment of severe hemophilia A.
Learn More about Valoctocogene Roxaparvovec ⌃ -
BMN 307 AAV
Gene TherapyPKUPhase 1Target IndicationBMN 307 is an AAV5-phenylalanine hydroxylase (PAH) gene therapy under investigation to determine if it can normalize blood phenylalanine concentration levels in patients with PKU.
Learn More ⌃ -
BMN 331 AAV
Gene TherapyHereditary Angioedema
(HAE)PreclinicalTarget IndicationBMN 331 is a gene therapy candidate in development for Hereditary Angioedema.
Learn More about BMN 331 AAV ⌃ -
BMN 255Chronic Renal DiseasePre-clinicalTarget Indication
BMN 255 is an investigational drug candidate in development for chronic renal disease.
We find tomorrows.
Each of our approved therapies was driven by the goal of resetting the standard of care for the conditions they treat.
Learn More about our products ⌃We take on the biggest challenges in rare disease.
Each drug candidate we pursue is guided by a fundamental understanding of the genetics and underlying biology of the condition it will address.
View Now Our Pipeline ⌃
We are driven by patient need.
We are committed to developing safe and effective therapies and providing those therapies to the patients as quickly as possible.
Compassionate Use Policy about our Compassionate Use Policy ⌃