U.S. markets closed

Biomarin to File Regulatory Applications for Vosoritide in Q3

Zacks Equity Research

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.

BioMarin’s shares have declined 2.1% so far this year compared with the industry’s decrease of 11.6%.

The company’s 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.

BioMarin’s 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 company’s PKU franchise generates the majority of the company’s 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 company’s revenues in 2021. A few other companies are also developing treatments for PKU in early-stage studies, which include Synlogic’s 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).

You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Today's Best Stocks from Zacks

Would you like to see the updated picks from our best market-beating strategies? From 2017 through 2019, while the S&P 500 gained and impressive +53.6%, five of our strategies returned +65.8%, +97.1%, +118.0%, +175.7% and even +186.7%.

This outperformance has not just been a recent phenomenon. From 2000 – 2019, while the S&P averaged +6.0% per year, our top strategies averaged up to +54.7% per year.

See their latest picks free >>

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report
Ascendis Pharma A/S (ASND) : Free Stock Analysis Report
Synlogic, Inc. (SYBX) : Free Stock Analysis Report
Homology Medicines, Inc. (FIXX) : Free Stock Analysis Report
To read this article on Zacks.com click here.
Zacks Investment Research