# BioMarin Pharmaceutical (BMRN)

BioMarin Pharmaceutical US FDA Approved ROCTAVIAN BioMarin Pharmaceutical ( BMRN ) announced that the U.S. FDA approved ROCTAVIAN (valoctocogene roxaparvovec-rvox) gene therapy for adults with severe...

Prohost Biotech

Jul 6, 2023

The U.S. FDA Approved BioMarin Pharmaceutical's ROCTAVIAN, the First and Only Gene Therapy for Adults with Severe Hemophilia A

The most important news in biological sciences is the high degree of success the biological revolution can reach. We can feel the great value every time an untreatable, devastating condition is succes...

Prohost Biotech

Jun 30, 2023

BioMarin Pharmaceutical Inc Product VOXZOGO® is the First to Prevent Dwarfism in Europe

Yesterday, under Today’s Highlights , we posted that the U.S. FDA issued a Complete Response Letter ( CRL ) to Gilead Sciences ( GILD ) with regard to the New Drug Application ( NDA ) for filgotinib -...

Prohost Biotech

Aug 31, 2021

The FDA's Complete Response Letters, Creating Investment Opportunities?

Prohost Letter #401 SYNOPSIS PART 1 In this Letter CONTENTS Duchenne Muscular Dystrophy (DMD) FDA Compassionate Approval of Eteplirsen! PART 2 In The Letter Issue #402 CONTENTS EYE ON IONIS (IONS) Ant...

Prohost Biotech

Aug 20, 2020

Prohost Letter #401

Prohost Letter #396 CELEBRATING MEMORIAL DAY - Wish you celebrated the Memorial Day. Memorial Day’s purpose was remembering the people who died while serving in the country’s armed forces. The Memori...

Prohost Biotech

Nov 16, 2016

Prohost Letter #396

Prohost Biotech

Jun 6, 2016

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Biomarin Pharmaceutical’s (BMRN) decision to withdraw its DMD drug Kyndrisa™ (drisapersen) Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) means more than pressuring...

Biomarin Decision Might Add More Stress Than Put Pressure on the FDA to Approve Sarepta Drug

Prohost Biotech

Jun 2, 2016

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EXELIXIS AGAIN They said: Sell EXEL, as no partner is interested in the firm’s FDA approved cabozantinib to date. What really happened: It did not take long for IPSEN to come forward and take the res...

Exelixis Again. The Road to Defeating DMD. Two New Important IPOs

Prohost Letter #389 Happy New Year 2016 Wishing You Good Health Peace of Mind Love And happiness We expect 2016 to be the year that fulfills many of our great expectations about successfully managing...

Prohost Biotech

May 9, 2016

Prohost Letter #389

Why is it Imperative to Learn About Gene Editing? Why Our Article was Delayed? We know well that many are waiting for the Part 2 of the GENE EDITING. The task looked easy but when reaching deeper into...

Prohost Biotech

Jan 2, 2016

Great News and Disappointing Setbacks. Why is Understanding Gene Editing Imperative?

BioMarin Pharmaceutical (BMRN) announced that the FDA has notified it that they had not yet completed their review process and would be unable to take an action by the Prescription Drug User Fee Act (...

Prohost Biotech

Dec 28, 2015

The FDA Delays Reviewing the DMD Drug Kyndrisa. What Happened After Ariad Selected a New CEO?

Prohost Letter #388 TOPICS - 1. AN INDISPENSABLE SHORT INTRODUCTION 2. GENE EDITING 3. GENE THERAPY 4. CANCER IMMUNOTHERAPY 1. GENE EDITING Exon Skipping: Biomarin (BMRN); Sarepta (SRPT); These firms...

Prohost Biotech

Dec 18, 2015

Prohost Letter #388

Prohost Biotech

Dec 2, 2015

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In our yesterday’s article asking about how the FDA advisory committee has to vote regarding Biomarin product Kyndrisa (drisapersen) for Duchenne muscular dystrophy. We got the answer yesterday during...

BioMarin: What Happened During the FDA Committee Meeting?

In our yesterday’s article asking about how the FDA advisory committee has to vote regarding Biomarin product Kyndrisa ( drisapersen) for Duchenne muscular dystrophy. We got the answer yesterday durin...

Prohost Biotech

Nov 25, 2015

Biomarin: Yesterday, FDA Committee Meeting. Adaptimmune: CAR T Approach for Solid Tumors

Prohost Biotech

Nov 25, 2015

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Today, the FDA advisory panel will discuss BioMarin (BMRN) product drisapersen for Duchenne muscular dystrophy based on documents released to the committee by the FDA. The FDA said, “The benefit of dr...

BioMarin: The DMD Drug and the FDA Committee Options

Prohost Letter #387 Topic of the Week DUCHENNE MUSCULAR DYSTROPHY Which Firms Will Get It Right – Deleting DMD Children’s Despair? The topic is important because, first and foremost, Duchenne muscular...

Prohost Biotech

Nov 24, 2015

Prohost Letter #387

Prohost Biotech

Nov 10, 2015

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About Duchenne muscular Dystrophy treatments, bloggers tried to explain the reasons that led the FDA to decide upon reviewing BioMarin’s (BMRN) Duchenne muscular dystrophy (DMD) drug Drisapersen a few...

Sarepta vs. Biomarin: Trying to Make an Educated Guess

PTC Therapeutics’ (PTCT) Duchenne muscular dystrophy drug (DMD) Translama failed to demonstrate a statistically significant improvement in distance traveled over the six-minute walk test, but it has n...

Prohost Biotech

Oct 19, 2015

PTC Therapeutics: Results of DMD Trial Are Good for Approval

Prohost Letter #384 The Assassination of the Biotech Sector The Market (Part 2) On Friday September 18, 2015, the biotechnology sector occupied the news headlines for two different reasons. The first...

Prohost Biotech

Oct 16, 2015

Prohost Letter #384

Yesterday, it was PTC Therapeutics’ (PTCT) good news and now it is Sarepta's (SRPT) turn in in the gene editing group to announce positive news about additional clinical efficacy and safety data from...

Prohost Biotech

Oct 6, 2015

More Successes Achieved in the Gene-Editing Group

Prohost Biotech

Oct 1, 2015