Breaking News
Ad-Free Version. Upgrade your experience. Save up to 40% More details

BioMarin (BMRN) Files NDA for Vosoritide to Treat Dwarfism

By Zacks Investment ResearchStock MarketsAug 21, 2020 06:30AM ET
BioMarin (BMRN) Files NDA for Vosoritide to Treat Dwarfism
By Zacks Investment Research   |  Aug 21, 2020 06:30AM ET
Saved. See Saved Items.
This article has already been saved in your Saved Items

BioMarin Pharmaceutical (NASDAQ:BMRN) Inc. BMRN filed a new drug application (NDA) for vosoritide to the FDA to treat children with achondroplasia, the most common form of dwarfism for which no drug is approved yet.

The company filed a regulatory application for vosoritide, a once daily injection analog of C-type Natriuretic Peptide (CNP), in the EU in July. The European Medicines Agency (EMA) commenced the review process on Aug 13. The regulatory applications are based on final data from a phase III study, evaluating the efficacy and safety of vosoritide in children (aged 5-14), long-term safety and efficacy data from the ongoing phase II and phase III extension studies and extensive natural history data.

In December 2019, BioMarin announced positive top-line data from the phase III 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.

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

Meanwhile, Ascendis Pharma (NASDAQ:ASND) ASND also has an achondroplasia candidate, TransCon CNP in a phase II study.

Another key candidate in BioMarin’s pipeline is Roctavian ( valoctocogene roxaparvovec), an investigational gene therapy, developed for treating patients with severe hemophilia A. BioMarin had filed a biologics license application (BLA) for Roctavian in December last year. However, in a surprise move, on Aug 19, the FDA issued a complete response letter (CRL) to Roctavian’s BLA ahead of Aug 21 PDUFA date.

The BLA application was based on interim data from an ongoing phase III study and the updated three-year results from a long-term phase I/II study. In a new requirement, the FDA asked for two-year follow-up data on annualized bleed rates from the ongoing phase III study to provide additional evidence of a durable effect. The data is not expected to be available before November 2021.

The CRL came as a massive disappointment to investors and shares crashed more than 35% on Wednesday in response.

This year so far, BioMarin’s shares have declined 11.5% against the industry’s increase of 2.7%.

Investors were expecting the FDA to grant accelerated approval to the drug on the PDUFA date. It was expected that Roctavian, if approved, would be a transformational product as it has the potential to dramatically change the treatment paradigm. However, the CRL now pushes potential approval of Roctavian to 2022.

Zacks Rank and Stocks to Consider

BioMarin currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the biotech sector are Emergent Biosolutions (NYSE:EBS) EBS and Horizon Therapeutics (NASDAQ:HZNP) Public Limited Company HZNP, both with a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Emergent Biosolutions’ earnings per share estimates have moved up 56.4% for 2020 and 44.1% for 2021 over the past 30 days. Share price of the company has increased 143% this year.

Horizon Therapeutics’ earnings per share estimates for 2020 have risen 58% while those for 2021 have gone up by 55.4% over the past 30 days. The stock has risen 107.5% this year so far.

More Stock News: This Is Bigger than the iPhone!

It could become the mother of all technological revolutions. Apple (NASDAQ:AAPL) sold a mere 1 billion iPhones in 10 years but a new breakthrough is expected to generate more than 27 billion devices in just 3 years, creating a $1.7 trillion market.

Zacks has just released a Special Report that spotlights this fast-emerging phenomenon and 6 tickers for taking advantage of it. If you don't buy now, you may kick yourself in 2021.

Click here for the 6 trades >>

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

BioMarin Pharmaceutical Inc. (BMRN): Free Stock Analysis Report

Emergent Biosolutions Inc. (EBS): Free Stock Analysis Report

Horizon Therapeutics Public Limited Company (HZNP): Free Stock Analysis Report

Ascendis Pharma AS (ASND): Free Stock Analysis Report

To read this article on click here.

Zacks Investment Research
BioMarin (BMRN) Files NDA for Vosoritide to Treat Dwarfism

Related Articles

BioMarin (BMRN) Files NDA for Vosoritide to Treat Dwarfism

Add a Comment

Comment Guidelines

We encourage you to use comments to engage with other users, share your perspective and ask questions of authors and each other. However, in order to maintain the high level of discourse we’ve all come to value and expect, please keep the following criteria in mind:  

  •            Enrich the conversation, don’t trash it.

  •           Stay focused and on track. Only post material that’s relevant to the topic being discussed. 

  •           Be respectful. Even negative opinions can be framed positively and diplomatically. Avoid profanity, slander or personal attacks directed at an author or another user. Racism, sexism and other forms of discrimination will not be tolerated.

  • Use standard writing style. Include punctuation and upper and lower cases. Comments that are written in all caps and contain excessive use of symbols will be removed.
  • NOTE: Spam and/or promotional messages and comments containing links will be removed. Phone numbers, email addresses, links to personal or business websites, Skype/Telegram/WhatsApp etc. addresses (including links to groups) will also be removed; self-promotional material or business-related solicitations or PR (ie, contact me for signals/advice etc.), and/or any other comment that contains personal contact specifcs or advertising will be removed as well. In addition, any of the above-mentioned violations may result in suspension of your account.
  • Doxxing. We do not allow any sharing of private or personal contact or other information about any individual or organization. This will result in immediate suspension of the commentor and his or her account.
  • Don’t monopolize the conversation. We appreciate passion and conviction, but we also strongly believe in giving everyone a chance to air their point of view. Therefore, in addition to civil interaction, we expect commenters to offer their opinions succinctly and thoughtfully, but not so repeatedly that others are annoyed or offended. If we receive complaints about individuals who take over a thread or forum, we reserve the right to ban them from the site, without recourse.
  • Only English comments will be allowed.

Perpetrators of spam or abuse will be deleted from the site and prohibited from future registration at’s discretion.

Write your thoughts here
Are you sure you want to delete this chart?
Post also to:
Replace the attached chart with a new chart ?
Your ability to comment is currently suspended due to negative user reports. Your status will be reviewed by our moderators.
Please wait a minute before you try to comment again.
Thanks for your comment. Please note that all comments are pending until approved by our moderators. It may therefore take some time before it appears on our website.
Are you sure you want to delete this chart?
Replace the attached chart with a new chart ?
Your ability to comment is currently suspended due to negative user reports. Your status will be reviewed by our moderators.
Please wait a minute before you try to comment again.
Add Chart to Comment
Confirm Block

Are you sure you want to block %USER_NAME%?

By doing so, you and %USER_NAME% will not be able to see any of each other's's posts.

%USER_NAME% was successfully added to your Block List

Since you’ve just unblocked this person, you must wait 48 hours before renewing the block.

Report this comment

I feel that this comment is:

Comment flagged

Thank You!

Your report has been sent to our moderators for review
Disclaimer: Fusion Media would like to remind you that the data contained in this website is not necessarily real-time nor accurate. All CFDs (stocks, indexes, futures) and Forex prices are not provided by exchanges but rather by market makers, and so prices may not be accurate and may differ from the actual market price, meaning prices are indicative and not appropriate for trading purposes. Therefore Fusion Media doesn`t bear any responsibility for any trading losses you might incur as a result of using this data.

Fusion Media or anyone involved with Fusion Media will not accept any liability for loss or damage as a result of reliance on the information including data, quotes, charts and buy/sell signals contained within this website. Please be fully informed regarding the risks and costs associated with trading the financial markets, it is one of the riskiest investment forms possible.
Continue with Google
Sign up with Email