BioMarin Pharmaceutical Inc. BMRN announced that it is planning to file regulatory applications in the third quarter of 2020, seeking approval for achondroplasia candidate, vosoritide, in the United States and Europe following its meetings with health authorities.

The regulatory applications will be filed based on data from successfully completed phase III study, which evaluated vosoritide as a treatment for achondroplasia, the most common form of disproportionate short stature in humans or dwarfism, in children aged 5 to 14 for 52 weeks. In December 2019, BioMarin announced positive top-line data from the study, which showed that patients treated with the candidate achieved placebo-adjusted change from baseline in growth velocity of 1.6 cm/year after treatment duration of one year.

The filings will be further supported by long-term safety and efficacy from a phase II study and ongoing extension studies on vosoritide in dwarf children.

A potential approval will make the drug the first and only approved treatment for achondroplasia in the United States and Europe. BioMarin estimates that around 25,000 children suffer from this disorder in its commercial territories, which represents decent sales growth opportunity.

Meanwhile, Ascendis Pharma ASND is developing an achondroplasia candidate in a phase II study.

BioMarins shares have declined 2.1% so far this year compared with the industrys decrease of 11.6%.

The companys key drugs, Vimizim and Kuvan, enjoy strong demand trends, which drove its revenues higher in 2019. Its newest product, Palynziq is also witnessing strong commercial uptake in the United States.

Apart from vosoritide, the company has a strong rare disease pipeline with focus on developing gene therapies.

BioMarins lead gene therapy candidate, valoctocogeneroxaparvovec (valrox) is under review in the United States and Europe as a treatment for severe hemophilia A. Decisions from the concerned regulatory authorities are expected in the second half of 2020.

A phase I/II study is evaluating another gene therapy candidate, BMN-307, as a treatment for phenylketonuria (PKU). Please note that Kuvan and Palynziq are also approved for treating PKU.

The companys PKU franchise generates the majority of the companys revenues. Another drug will likely boost the sales of this franchise.

Meanwhile, Kuvan is set to face generic competition as early as October 2020. This will likely dent the companys revenues in 2021. A few other companies are also developing treatments for PKU in early-stage studies, which include Synlogics SYBX small molecule candidate, SYNB1618; Homology Medicines FIXX gene therapy, HMI-102; and Rubius Therapeutics red cell therapeutic candidate, RTX-134. Potential approval to vosoritide may partially offset loss in Kuvan sales.

BioMarin Pharmaceutical Inc. Price

BioMarin Pharmaceutical Inc. Price

BioMarin Pharmaceutical Inc. price | BioMarin Pharmaceutical Inc. Quote

Zacks Rank

BioMarin currently has a Zacks Rank #3 (Hold).

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Biomarin to File Regulatory Applications for Vosoritide in Q3 - Yahoo Finance

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