BioMarin Pharmaceutical Inc (BMRN) ニュース

BMRN volatile stock

GRABAR LAW OFFICE INVESTIGATES POTENTIAL DERIVATIVE ACTION ON BEHALF OF CURRENT SHAREHOLDERS OF BIOMARIN PHARMACEUTICAL INC. (NASDAQ: BMRN)
A class action alleging BioMarin misled investors survived a motion to dismiss. For more information see https://grabarlaw.com/the-latest/biomarin-pharma-investigation/

and the CEO gets on a earnings call and lowers guidance for Q4

and that is that

crushed the expectations on EPS

options trading on BMRN is insane

BMRN shorts will be “suprised and burned” by CHMP - will be SO NICE.

Hey Shorts-why didn’t the FDA inspect BMRN Valrox factory in Nevada, while the EMA inspected the BMRN Valrox factory in Ireland 3 months ago? Actions speak louder than words - FDA had no intention of Approving without more data and EMA does - Ask BA CSI Wedbush and Cantor.

BMRN
Cantor Fitzgerald Note on BMRN 8/19- Price Target $125 from $129 - Overweight maintained:

“We think the base business alone (NPV, removing spend for pipeline) is worth $90/sh vs. shares at $76/sh currently. While CRL is a setback, we still see significant growth ahead for BMRN. We see potential for upside from execution across EU Roctavian review, vosoritide (achondroplasia, regulatory submissions accepted), and emerging gene therapy portfolio, coupled with manufacturing expertise. We note that the PKU gene therapy program is expected to dose its first patient this quarter and we think shares reflect limited credit to that program.”

BMRN
Wedbush analyst Liana Moussatos reiterated a Buy rating on BioMarin Pharmaceuticals-
– Research Report) today and set a price target of $136.00. The company’s shares closed last Wednesday at $75.04.

According to tipranks.com, Moussatos is a 5-star analyst with an average return of 12.2% and a 53.4% success rate.

CMA for BMRN - BMRN will receive a “Conditional marketing authorisation“ -for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A-
“The approval of a medicine that address unmet medical needs of patients on the basis of less comprehensive data than normally required. The available data must indicate that the medicine’s benefits outweigh its risks and the applicant should be in a position to provide the comprehensive clinical data in the future.”

https://www.ema.europa.eu/en/glossary/conditional-marketing-authorisation

EMA will ignore Trumps FDA -greater Hemophilia need :
BA Analyst Meachum says we can still see EU
$BMRN
approval in 2020- EMA says 210 days til word on
$BMRN
Hemophilia approval - wow - even with clock stops - any day now : SAN RAFAEL, Calif., Dec. 23, 2019 /PRNewswire/ -- “BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Medicines Agency (EMA) validated the Company's Marketing Authorization Application (MAA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. The MAA review will commence in January 2020 under accelerated assessment.

EMA granted access to its Priority Medicines (PRIME) regulatory initiative in 2017 and recently granted BioMarin's request for accelerated assessment of this MAA-This submission marks the first marketing application submission in Europe for a gene therapy product for any type of hemophilia.
Evaluating a MAA under the EMA centralized procedure can take up to 210 days.”

BioMarin Submits New Drug Application to U.S. Food and Drug Administration for Vosoritide to Treat Children with Achondroplasia

Published: Aug 20, 2020

= “near term growth “- as is pending Hemophilia approval in EU - a larger market for Hemophiia than is the US

https://www.biospace.com/article/releases/biomarin-submits-new-drug-application-to-u-s-food-and-drug-administration-for-vosoritide-to-treat-children-with-achondroplasia/

BMRN
Sorry CitI - BA analyst Geoff Meachum (maintains Buy with $105 price target) and Canaccord Genuity analyst Michelle Gilson say you’re wrong-Gilson maintains a Buy rating on BioMarin Pharmaceutical yesterday and set a price target of $95.00.

According to tipranks.com/, Gilson is a 5-star analyst with an average return of 35.1% and a 63.8% success rate.

Bank America note on BMRN

”Approval still in the cards...just delayed
-perhaps 2 years“
Reiterate Rating: BUY | PO: 105.00 USD

No accelerated approval for $SGMO $PFE on 4/6 WEEK followup - $BMRN has triggered 2 year followup for all- Lead maintained.
https://endpts.com/sangamo-pfizer-are-catching-up-to-biomarin-and-spark-on-a-gene-therapy-for-hemophilia-a-with-a-long-way-to-go/

$BAC On $BMRN

“FDA has requested BioMarin provide 2-year annualized bleed rate (ABR) data for Roctavian to complete the filing package (which BioMarin expects to have in November 2021). 6-month phase 3 data showed 85%/100% reduction in mean/median ABR and 4- year phase 1/2 data showed a 95%/100% reduction, which gives us comfort in the reproducibility in 2-year phase 3 data. Notably, in the phase 1/2 dataset, the 2-year mean bleed rate improvement was better than in year 1 (99% vs. 92%) so we could see the 85% reduction potentially improve. If Roctavian can show anything near this in November 2021, we think BioMarin should have no problem getting approval.”

Ttttttiiiiiimmmmmmmbbbbbeeeerrrrr!!!!!!!

$131.19 and still climbing

93.22

talk about missing the boat, I had an alert go off a couple of weeks ago on a price dip below 69.00

I hadn't been keeping up with BMRN lately so I blew it off

now trading at 84.50

I guess I put that alert on this puppy for a reason

Experiments in juveniles and infants can garner public praise, lower regulatory hurdles, and mask overspending. It creates a unique and problematic bias. In the US, mores are well established in this regard. One cannot question the utility of such treatments without being vilified.

Two catalysts this month for BMRN.
1. PDUFA
2. Hem A data

Here is the info link for upcoming biotech data insert-text-here

Thanks

Out @ $88.50 for a small profit after buying down from a too high entry. Probably a multi-day runner, but changing brokers (leaving Ally Invest which has been terrible) and had to sell.

Could be back in tomorrow if transfer proceeds well.

BMRN

very nice move off of the bottom here. I was holding out to start taking a starter until 80.00... good to hold discipline... but I missed a nice move

According to WhaleWisdom, BMRN is Baker Bro 6th largest holding with 22% increase in shares as of the last filing:

BMRN HEALTH CARE 5,905,673 $ 536,353,000 2.59% 3.45% 6 1,099,190 22% 3.4163% Q2 2012 13F 2017-06-30 2017-08-14

https://whalewisdom.com/filer/baker-bros-advisors-llc#/tabholdings_tab_link

BMRN

Sarepta Therapeutics and BioMarin Pharmaceutical Inc. Announce Execution of a Global Settlement and a License Agreement Resolving Exon Skipping Patent Litigation

Agreement terms resolve global patent proceedings regarding Sarepta’s sale of EXONDYS 51® (eteplirsen) and future Duchenne muscular dystrophy (DMD) exon-skipping products

July 18, 2017 08:30 ET | Source: Sarepta Therapeutics
CAMBRIDGE, Mass. and SAN RAFAEL, Calif., July 18, 2017 (GLOBE NEWSWIRE) --

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a U.S. commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases, and BioMarin Pharmaceutical Inc. (NASDAQ:BMRN), a leading biotechnology company in therapies for rare genetic diseases, announced today that Sarepta and BioMarin executed a license agreement that provides Sarepta Therapeutics with global exclusive rights to BioMarin’s DMD patent estate for EXONDYS 51 and all future exon-skipping products. BioMarin retains the right to convert the license to a co-exclusive right in the event it decides to proceed with an exon-skipping therapy for DMD. In addition, Sarepta and BioMarin executed a settlement agreement, resolving the ongoing worldwide patent proceedings related to the use of EXONDYS 51 and all future exon-skipping products for the treatment of DMD. The effectiveness of the agreements is subject to closing conditions including execution of necessary approvals by Academisch Ziekenhuis Leiden (AZL) by July 24, 2017.

Under the terms of the license and settlement agreements, Sarepta will make a one-time payment of $35 million to BioMarin and certain additional regulatory and commercial milestone payments for exons 51, 45, 53 and possibly on future exon-skipping products.

In addition, Sarepta will pay royalties to BioMarin as follows:

Exon-skipping compounds 51, 45, and 53 and possibly on future exon-skipping products: Sarepta will pay BioMarin 5 percent of net sales through the end of 2023 in the United States; and
Exon-skipping compounds 51, 45, and 53 and possibly on future exon-skipping products: Sarepta will pay BioMarin 8 percent of net sales through September 30, 2024 in the European Union and in other countries where certain BioMarin / AZL patents exist.
“Upon their effectiveness, these global license and settlement agreements provide Sarepta worldwide freedom to operate for EXONDYS 51 and our future exon-skipping products,” said Douglas Ingram, Sarepta’s President and Chief Executive Officer. “The resolution of these legal matters provides us with more certainty to fully focus our resources and energy on our crucial mission of developing innovative medicines to improve the lives of those impacted by DMD around the world."

“We are pleased to reach a global settlement and license agreement with Sarepta that fairly recognizes the important innovation by the Leiden University Medical Center and allows patients certainty that this issue will not create a barrier to access,” said G. Eric Davis, BioMarin’s Executive Vice President and General Counsel.

About EXONDYS 51
EXONDYS 51 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. EXONDYS 51 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. Data from clinical studies of EXONDYS 51 in a small number of DMD patients have demonstrated a consistent safety and tolerability profile. The pivotal trials were not designed to evaluate long-term safety and a clinical benefit of EXONDYS 51 has not been established.

Important Safety Information
Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended.

In the 88 patients who received ≥30 mg/kg/week of EXONDYS 51 for up to 208 weeks in clinical studies, the following events were reported in ≥10% of patients and occurred more frequently than on the same dose in Study 1: vomiting, contusion, excoriation, arthralgia, rash, catheter site pain, and upper respiratory tract infection.

There have been reports of transient erythema, facial flushing, and elevated temperature occurring on the day of EXONDYS 51 infusion.

About Sarepta Therapeutics
Sarepta Therapeutics is a U.S. commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases. The company is primarily focused on rapidly advancing the development of its potentially disease-modifying Duchenne muscular dystrophy (DMD) drug candidates. For more information, please visit www.sarepta.com.

About BioMarin Pharmaceutical Inc.
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare disorders. The company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.biomarin.com. Information on BioMarin's website is not incorporated by reference into this press release.

Forward-Looking Statements
This press release contains statements that are forward-looking. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements about the license agreement providing Sarepta with global exclusive rights to BioMarin’s DMD patent estate for EXONDYS 51 and all future exon-skipping products; the settlement agreement resolving the ongoing worldwide patent proceedings related to the use of EXONDYS 51 and all future exon-skipping products for the treatment of DMD; the payments and royalties that Sarepta will be making as part of the settlement and license agreements; the settlement and license agreements providing for Sarepta's worldwide freedom to operate for EXONDYS 51 and Sarepta’s future exon-skipping products; the settlement providing Sarepta with the certainty to fully focus its resources and energy on its crucial mission of developing innovative medicines to improve the lives of those impacted by DMD around the world; and the statement that the patent proceedings between the parties will not create for patients a barrier to access to the innovation by the Leiden University Medical Center.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Known risk factors include, among others: the settlement and license agreements may not become effective if their conditions to effectiveness are not met within the required deadline; the parties may not be able to fulfill their commitments and obligations under the settlement and license agreements; any future claims of infringement by other third parties; the expected benefits and opportunities related to the settlement and license agreements between the parties may not be realized or may take longer to realize than expected due to challenges and uncertainties regarding the sales of EXONDYS 51 and the research and development of future exon-skipping products; Sarepta may experience significant fluctuations in sales of EXONDYS 51 from period to period and, ultimately, Sarepta may never generate sufficient revenues from EXONDYS 51 to reach or maintain profitability or sustain its anticipated levels of operations; Sarepta may never receive regulatory approval to its future exon-skipping products due to a variety of reasons including that the results of additional research may not be consistent with past results or may not be positive or may otherwise fail to meet regulatory approval requirements for the safety and efficacy of product candidates; and even if Sarepta obtains regulatory approvals, it may not achieve any significant revenues from the sale of such products; Sarepta may not have worldwide freedom to operate for EXONDYS 51 and Sarepta’s future exon-skipping products due to future proceedings brought by other parties.

Any of the foregoing risks could adversely affect Sarepta's business, results of operations and the trading price of Sarepta's common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review Sarepta's 2016 Annual Report on Form 10-K and most recent Quarterly Report on Form 10-Q for the quarter ended March 31, 2017 filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Internet Posting of Information
We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Media and Investors:
Sarepta Therapeutics, Inc.
Ian Estepan, 617-274-4052
iestepan@sarepta.com
or
W2O Group
Brian Reid, 212-257-6725
breid@w2ogroup.com
or
Investors:
BioMarin Pharmaceutical Inc.
Traci McCarty, 415-455-7558
Media:
BioMarin Pharmaceutical Inc.
Debra Charlesworth, 415-455-7451
______________________________________________

BMRN

Wrong choice. Hugh volume today and now near the 3 month low on no news and biotech up marginally.

BMRN

In BMRN @ $88.80 today. Hadn't traded it for over 3 years.

A Baker Bro stock with limited up potential:

Consensus Ratings for BioMarin Pharmaceutical (NASDAQ:BMRN) (How are Consensus Ratings Calculated?)
Ratings Breakdown: 6 Hold Ratings, 14 Buy Ratings
Consensus Rating: Buy (Score: 2.70)
Consensus Price Target: $113.61 (24.85% upside)
_______________________________________

https://www.marketbeat.com/stocks/NASDAQ/BMRN/

https://seekingalpha.com/article/4087040-biomarin-hemophila-candidates-looks-impress-community

Always good to see the bulls.

It's a ways off, but Jan 27 will be a good day if/when new drug gets approved. I see that sometimes FDA approves on a date prior to the scheduled date - does anyone know if that sort of news that a decision is going to be made early (ie if approval/CRL) is avail before it actually happens?

BMRN bullish 91.99

optionable

FDA denied BMRN. SRPT is leading now.

BioMarin, Sarepta’s Loss Could Be PTC Therapeutic’s Gain

http://marketexclusive.com/biomarin-sareptas-loss-could-be-ptcs-gain/1419/

Could the FDA Approve Both BioMarin and Sarepta’s DMD Candidates?

http://marketexclusive.com/could-the-fda-approve-both-biomarin-and-sareptas-dmd-candidates/1184/

Two Stocks to Watch This Month in Biotech - article discusses BioMarin http://marketexclusive.com/two-stocks/1128/

Surprised to see you here. Bio-Marin is heading back up FYI ..

Looks like we're all going to be on the sidelines today (BMRN also had positive news from the UK's Health Technology Assessment agency, NICE, today, (http://www.pharmatimes.com/Article/15-11-23/NICE_draft_yes_for_BioMarin_s_Vimizim.aspx) but that will likely be drowned out by the outcome of today's FDA review meeting).

"BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) Announced Today the Stock is Halted

Date : 11/24/2015 @ 7:05AM
Source : GlobeNewswire Inc.
Stock : Biomarin Pharmaceutical Inc. (MM) (BMRN)

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) Announced Today the Stock is Halted


Today : Tuesday 24 November 2015


BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that NASDAQ has halted trading of the company’s stock. The Peripheral and Central Nervous System Drugs Advisory Committee (PCNS) of the U.S. Food and Drug Administration (FDA) is meeting today to review BioMarin’s New Drug Application (NDA) for Kyndrisa, an investigational antisense oligonucleotide drug candidate for the treatment of patients with Duchenne muscular dystrophy amenable to exon 51 skipping.


The PCNS advisory meeting is scheduled for November 24 at 8:00 a.m. to 5:30 p.m. EST. The briefing materials and webcast information can be found on the FDA website at: www.fda.gov/AdvisoryCommittees/Calendar/ucm467180.htm

The Prescription Drug User Fee Act (PDUFA) action date for completion of FDA review of the Kyndrisa NDA is December 27, 2015.

About Duchenne Muscular Dystrophy

Changes in the dystrophin gene (mutations) that lead to the near absence of dystrophin protein result in the most severe form of dystrophin deficient muscular dystrophy, Duchenne muscular dystrophy, also known as just Duchenne. Boys living with Duchenne experience progressive muscle weakness, causing serious medical complications including serious heart or respiratory-related complications, resulting in death in early adulthood.

Primarily affecting boys, Duchenne affects approximately 1 in every 3,500-5,000 male children, making it the most common fatal genetic disorder diagnosed in childhood. There is currently no FDA approved therapy designed specifically to treat Duchenne.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases. The company's portfolio consists of five commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.BMRN.com.
Investors
Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558

Media
Debra Charlesworth
BioMarin Pharmaceutical Inc.
(415) 455-7451"

BMRN W

Inst % Owned = 99%

Now in regards to this, I would think $160 near term.

Thank you

King
I think you are one of the best traders on IHUB

You'll find that I'm not a dumb retail trader like you may think I am.

KING
Interesting how we find the same stocks.

BMRN W

Deutsche, Jeffries, Barclays $156 PT this morning.

$BMRN DD Notes ~ http://www.ddnotesmaker.com/BMRN

bullish

$BMRN recent news/filings

## source: finance.yahoo.com

Thu, 25 Sep 2014 21:24:32 GMT ~ BioMarin (BMRN) Faces Threat of Genericization for Kuvan


read full: http://finance.yahoo.com/news/biomarin-bmrn-faces-threat-genericization-212432661.html
*********************************************************

Thu, 25 Sep 2014 12:35:53 GMT ~ 5 Orphan Drug Biotech Stocks to Buy With Big Upside Potential


read full: http://247wallst.com/healthcare-business/2014/09/25/5-orphan-drug-biotech-stocks-to-buy-with-big-upside-potential/
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Wed, 24 Sep 2014 21:26:00 GMT ~ 5:26 pm Biomarin Pharm confirms that a Paragraph IV Certification Notice Letter was submitted to the FDA in connection with an ANDA to the FDA for approval to market a generic version of KUVAN


read full: http://finance.yahoo.com/news/inplay-briefing-com-055139997.html#bmrn
*********************************************************

Wed, 24 Sep 2014 21:21:32 GMT ~ FDA Receives Paragraph IV Notice Letter for KUVAN(R) (sapropterin dihydrochloride) Tablets

[GlobeNewswire] - SAN RAFAEL, Calif. -- BioMarin Pharmaceutical Inc. today announced that a Paragraph IV Certification Notice Letter was submitted to the US Food and Drug Administration (FDA) in connection with an Abbreviated ...

read full: http://finance.yahoo.com/news/fda-receives-paragraph-iv-notice-212132582.html
*********************************************************

Tue, 23 Sep 2014 12:00:00 GMT ~ BioMarin to Attend Upcoming Investor Conferences

[GlobeNewswire] - - Jefferies Gene Therapy Summit on September 30 in Boston

read full: http://finance.yahoo.com/news/biomarin-attend-upcoming-investor-conferences-120000803.html
*********************************************************


$BMRN charts

basic chart ## source: stockcharts.com



basic chart ## source: stockscores.com



big daily chart ## source: stockcharts.com



big weekly chart ## source: stockcharts.com


$BMRN company information

## source: otcmarkets.com

Link: http://www.otcmarkets.com/stock/BMRN/company-info
Ticker: $BMRN
OTC Market Place: Not Available
CIK code: 0001048477
Company name: BioMarin Pharmaceutical, Inc.
Incorporated In: DE, USA


$BMRN share structure

## source: otcmarkets.com

Market Value: $10,490,904,214 a/o Sep 25, 2014
Shares Outstanding: 147,116,873 a/o Jul 18, 2014
Float: Not Available
Authorized Shares: Not Available
Par Value: 0.001
$BMRN extra dd links

Company name: BioMarin Pharmaceutical, Inc.
## STOCK DETAILS ##
After Hours Quote (nasdaq.com): http://www.nasdaq.com/symbol/BMRN/after-hours
Option Chain (nasdaq.com): http://www.nasdaq.com/symbol/BMRN/option-chain
Historical Prices (yahoo.com): http://finance.yahoo.com/q/hp?s=BMRN+Historical+Prices
Company Profile (yahoo.com): http://finance.yahoo.com/q/pr?s=BMRN+Profile
Industry (yahoo.com): http://finance.yahoo.com/q/in?s=BMRN+Industry

## COMPANY NEWS ##
Market Stream (nasdaq.com): http://www.nasdaq.com/symbol/BMRN/stream
Latest news (otcmarkets.com): http://www.otcmarkets.com/stock/BMRN/news - http://finance.yahoo.com/q/h?s=BMRN+Headlines

## STOCK ANALYSIS ##
Analyst Research (nasdaq.com): http://www.nasdaq.com/symbol/BMRN/analyst-research
Guru Analysis (nasdaq.com): http://www.nasdaq.com/symbol/BMRN/guru-analysis
Stock Report (nasdaq.com): http://www.nasdaq.com/symbol/BMRN/stock-report
Competitors (nasdaq.com): http://www.nasdaq.com/symbol/BMRN/competitors
Stock Consultant (nasdaq.com): http://www.nasdaq.com/symbol/BMRN/stock-consultant
Stock Comparison (nasdaq.com): http://www.nasdaq.com/symbol/BMRN/stock-comparison
Investopedia (investopedia.com): http://www.investopedia.com/markets/stocks/BMRN/?wa=0
Research Reports (otcmarkets.com): http://www.otcmarkets.com/stock/BMRN/research
Basic Tech. Analysis (yahoo.com): http://finance.yahoo.com/q/ta?s=BMRN+Basic+Tech.+Analysis
Barchart (barchart.com): http://www.barchart.com/quotes/stocks/BMRN
DTCC (dtcc.com): http://search2.dtcc.com/?q=BioMarin+Pharmaceutical%2C+Inc.&x=10&y=8&sp_p=all&sp_f=ISO-8859-1
Spoke company information (spoke.com): http://www.spoke.com/search?utf8=%E2%9C%93&q=BioMarin+Pharmaceutical%2C+Inc.
Corporation WIKI (corporationwiki.com): http://www.corporationwiki.com/search/results?term=BioMarin+Pharmaceutical%2C+Inc.&x=0&y=0

## FUNDAMENTALS ##
Call Transcripts (nasdaq.com): http://www.nasdaq.com/symbol/BMRN/call-transcripts
Annual Report (companyspotlight.com): http://www.companyspotlight.com/library/companies/keyword/BMRN
Income Statement (nasdaq.com): http://www.nasdaq.com/symbol/BMRN/financials?query=income-statement
Revenue/EPS (nasdaq.com): http://www.nasdaq.com/symbol/BMRN/revenue-eps
SEC Filings (nasdaq.com): http://www.nasdaq.com/symbol/BMRN/sec-filings
Edgar filings (sec.gov): http://www.sec.gov/cgi-bin/browse-edgar?action=getcompany&CIK=0001048477&owner=exclude&count=40
Latest filings (otcmarkets.com): http://www.otcmarkets.com/stock/BMRN/filings
Latest financials (otcmarkets.com): http://www.otcmarkets.com/stock/BMRN/financials
Short Interest (nasdaq.com): http://www.nasdaq.com/symbol/BMRN/short-interest
Dividend History (nasdaq.com): http://www.nasdaq.com/symbol/BMRN/dividend-history
RegSho (regsho.com): http://www.regsho.com/tools/symbol_stats.php?sym=BMRN&search=search
OTC Short Report (otcshortreport.com): http://otcshortreport.com/index.php?index=BMRN
Short Sales (otcmarkets.com): http://www.otcmarkets.com/stock/BMRN/short-sales
Key Statistics (yahoo.com): http://finance.yahoo.com/q/ks?s=BMRN+Key+Statistics
Insider Roster (yahoo.com): http://finance.yahoo.com/q/ir?s=BMRN+Insider+Roster
Income Statement (yahoo.com): http://finance.yahoo.com/q/is?s=BMRN
Balance Sheet (yahoo.com): http://finance.yahoo.com/q/bs?s=BMRN
Cash Flow (yahoo.com): http://finance.yahoo.com/q/cf?s=BMRN+Cash+Flow&annual

## HOLDINGS ##
Major holdings (cnbc.com): http://data.cnbc.com/quotes/BMRN/tab/8.1
Insider transactions (yahoo.com): http://finance.yahoo.com/q/it?s=BMRN+Insider+Transactions
Insider transactions (secform4.com): http://www.secform4.com/insider-trading/BMRN.htm
Insider transactions (insidercrow.com): http://www.insidercow.com/history/company.jsp?company=BMRN
Ownership Summary (nasdaq.com): http://www.nasdaq.com/symbol/BMRN/ownership-summary
Institutional Holdings (nasdaq.com): http://www.nasdaq.com/symbol/BMRN/institutional-holdings
Insiders (SEC Form 4) (nasdaq.com): http://www.nasdaq.com/symbol/BMRN/insider-trades
Insider Disclosure (otcmarkets.com): http://www.otcmarkets.com/stock/BMRN/insider-transactions

## SOCIAL MEDIA AND OTHER VARIOUS SOURCES ##
PST (pennystocktweets.com): http://www.pennystocktweets.com/stocks/profile/BMRN
Market Watch (marketwatch.com): http://www.marketwatch.com/investing/stock/BMRN
Bloomberg (bloomberg.com): http://www.bloomberg.com/quote/BMRN:US
Morningstar (morningstar.com): http://quotes.morningstar.com/stock/s?t=BMRN
Bussinessweek (businessweek.com): http://investing.businessweek.com/research/stocks/snapshot/snapshot_article.asp?ticker=BMRN



$BMRN DD Notes ~ http://www.ddnotesmaker.com/BMRN

Don't have PM and haven't been following BioMarin lately. Good luck to any holding through earnings.

BMRN

Great company. If you are in over the last 8-9 days you really are smart money. Me--not in. Just watched it march up looking for a pull back that never came.

BMRN

BioMarin Announces FDA Approval for VIMIZIM(TM) (elosulfase alfa) for the Treatment of Patients With Morquio A Syndrome


Alert



Biomarin (NASDAQ:BMRN)
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BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the U.S. Food and Drug Administration (FDA) has approved VIMIZIM™ (elosulfase alfa) for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).

"The FDA approval of VIMIZIM is an important milestone for BioMarin and for patients with Morquio A syndrome. VIMIZIM is the first and only therapy designed to address the condition at the cellular level, fulfilling a large unmet medical need for patients and their families," said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. "With the approval of VIMIZIM, BioMarin firmly establishes its leadership in advancing therapies to treat MPS diseases. We have developed three therapies to treat three different MPS diseases and continue to build on our extensive scientific and clinical knowledge of lysosomal storage disorders to develop therapies for other rare genetic diseases."

VIMIZIM is an enzyme replacement treatment for Morquio A syndrome, which affects an estimated 3,000 patients in the developed world. The disease occurs as a result of a deficiency of activity in an enzyme involved in glycosaminoglycan (GAG) metabolism. The pervasive and progressive accumulation of GAGs leads to significant morbidities and multisystemic clinical impairments resulting in diminished functional capacity, impaired quality of life, and early mortality. The most common features of the disease are progressive skeletal dysplasia, the need for frequent surgical procedures related primarily to musculoskeletal or respiratory dysfunction, and significant limitations in mobility, endurance, and breathing.

"In clinical trials, VIMIZIM was shown to significantly improve endurance, which possibly could change the course of the disease. As a treating physician, I am encouraged that the therapy has proven to provide clinical benefit, which is not always possible to demonstrate with ultra-rare diseases," said Paul Harmatz, M.D., Associate in Gastroenterology and Nutrition at the Children's Hospital and Research Center in Oakland, California and clinical investigator in the VIMIZIM Phase 3 trial. "The approval of VIMIZIM is an important advance for Morquio A patients and their families and moves treatment beyond supportive care to treating the underlying cause of the disease."

"We are thrilled that patients with Morquio A syndrome will have access to this potentially life-changing therapy and appreciate BioMarin's commitment to the MPS community and the individuals and their families who are affected by these devastating conditions," said Barbara Wedehase, MSW, CGC, Executive Director of the National MPS Society. "Until now, patients with Morquio A syndrome didn't have a drug treatment option. This approval provides the community with a therapy and with hope."

Shipments of VIMIZIM to the distribution channels will commence immediately, and BioMarin will begin promotion of VIMIZIM in the U.S. immediately. BioMarin has also submitted marketing applications for VIMIZIM in the European Union, Brazil, Australia, Canada, and Mexico.

BioMarin will offer support to patients through its BioMarin Patient & Physician Support (BPPS) team. Through BPPS, patients receive live, personalized support by a specialized case manager who will research insurance coverage and alternative benefit options. BPPS will help patients obtain coverage and minimize out-of-pocket expenses and find alternative financial assistance for treatment. To reach a BPPS case manager, please call, toll-free, 1-866-906-6100 or e-mail bpps@bmrn.com. For more information about VIMIZIM, please visit www.VIMIZIM.com.

Clinical Trial Results

The safety and efficacy of VIMIZIM were assessed in a 24-week, randomized, double-blind, placebo-controlled clinical trial of 176 patients with MPS IVA (MOR-004). The primary endpoint of the trial, change in six-minute walk distance at 24 weeks, was statistically significant in patients receiving weekly infusions of VIMIZIM at the dose of 2 mg/kg with a mean increase of 22.5 meters (p=0.0174) over placebo.

In patients who continued to receive VIMIZIM 2 mg/kg once per week for another 48 weeks (for a total of 72-week exposure), walking ability was sustained to a similar level that was achieved during the first 24 weeks of treatment in the placebo-controlled trial, MOR-004.

Overall, sustained improvements across multiple efficacy measurements and across multiple clinical trials provided evidence of clinical benefit to patients with MPS IVA, a chronic, progressive disease in which clinical deterioration is the expected course.

The adverse events observed in clinical trials were similar to those seen in other enzyme replacement therapies. In the Phase 3 trial, the most common adverse reactions (=10% and a higher incidence than placebo) that occurred were pyrexia, vomiting, headache, nausea, abdominal pain, chills, and fatigue. No new types of adverse reactions were reported in the Phase 3 extension trial. The most common adverse reactions (=10%) observed across pre-marketing clinical trials were similar in type and frequency as those observed in the placebo-controlled trial. Acute reactions requiring intervention were managed by either temporarily interrupting or discontinuing infusion, and administering additional antihistamine, antipyretics, or corticosteroids.

Note to Investors

BioMarin will host a webcast to discuss the VIMIZIM approval Tuesday, February 18, 2014 at 5:00 a.m. PT. Dial-in information for the conference call will be distributed prior to the call.

Interested parties may access a live audio webcast of the conference call via the investor section of the BioMarin website, www.BMRN.com. A replay of the call will be archived on the site for one week following the call.

About VIMIZIM™

VIMIZIM (elosulfase alfa) is a treatment for patients with Morquio A syndrome, or mucopolysaccharidosis IVA (MPS IVA). VIMIZIM is the first enzyme replacement therapy (ERT) designed to target the underlying cause of Morquio A Syndrome – a deficiency in the enzyme N-acetylgalactosamine-6 sulfatase (GALNS). VIMIZIM is intended to provide the exogenous enzyme GALNS that will be taken up into the lysosomes and increase the catabolism of GAGs. Morquio A syndrome is a rare, severely debilitating and progressive disease that previously had no standard accepted treatment other than supportive care.

Not that I believe the BMRN drug would have cured her but still its sad to see that she passed away.

http://www.keyetv.com/news/features/top-stories/stories/austin-woman-battled-cancer-drug-dies-15275.shtml