BioMarin Pharmaceutical US FDA Approval for ROCTAVIAN On June 30, 2023, BioMarin Pharmaceutical (BMRN) announced that the U.S. FDA has approved ROCTAVIAN™ (valoctocogene roxaparvovec-rvox) gene therapy for adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by . . . This content is for paid subscribers. Please click here to …

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 hemophilia A without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test. The one-time, single-dose infusion is the first approved gene therapy for severe hemophilia . . . This content is for paid subscribers. Please click here to subscribe …

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 successfully treated thanks to discoveries made possible through the information brought in by the unparalleled revolution that allows for a better understanding of the living body mechanisms of actions in health and in diseases. …

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 . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

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) Antisense Proof of Concept and Feasibility EYE ON INVIVA (INVA) AND THERAVANCE BIOPHARMA (TBPH) Sales of The Ellipta Inhalers Started to Pick up Before an expected Boom Not Losing . . . This content …

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 Memorial Day was initiated as Decoration Day after the Civil War in the year 1868 when the Grand Army of the Republic - an organization of Union Veterans in Illinois established it to decorate the graves …

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 the FDA to approve Sarepta’s DMD product eteplirsen. As we see from Biomarin press release, its decision was based on several reasons, including the discussions at the May 2016 Committee for Medicinal Products for Human Use (CHMP) meeting, which clearly …

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 responsibility of marketing cabozantinib abroad and pay $200 million upfront payment to Exelixis. They said: Although the FDA approved Cabometyx (cabozantinib) for advanced renal cell carcinoma (RCC), the drug will …

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 intractable and life-threatening malignant and non-malignant disease. We expect the biotech firms to bring us more cures based on the maturation of genetic engineering processes following a lot of trial and …

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 are waiting FDA approvals for Duchenne Muscular Dystrophy (DMD) Gene Editing Technology Competitors: Cellectis S.A. (CLLS); CRISPR; PTC Therapeutics (PTCT) Firms Using Different Approaches For DMD Treatment (see Below) Zink Finger Tech: Sangamo (SGMO); RNAi …

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 the meeting that took a much longer time than committees’ meetings usually take. At the end of the long session that included several patients’ and patients’ relatives’ testimonies, the FDA Advisory Committee was incapable of voting for …

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 drisapersen in exon 51 skip amenable patients is inconclusive at this time. Therefore, the benefit-risk assessments were not made.” Analysts interpreted the FDA statement as a condemnation of the drug, which does not really tell what the FDA meant …

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 dystrophy (DMD) disables children. Also because, from no treatment at all, now we have several biotech firms developing various drugs based on different scientific strategies. The new approaches have increased in number and have …

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 weeks earlier than reviewing Sarepta (SRPT) DMD drug Eteplirsen. The real reasons for the FDA decision remain inside the FDA mind. No outsider will be allowed to know anything related to NDA reviews. All the talk and …

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 was the sudden unexplicable and unexpected plummeting of the biotech sector. The stocks were flying sky high until modday and suddenly, all the biotech stocks, without exception, took a dive. The second reason was the triumph of …

Prohost Letter #383 The Market - Recent tails about the market and the biotech sector are true thrillers. By true we mean no-fiction. The accounts lack common sense. Professionals' interpretations of the stocks' and of the market's moves are bizarre and misleading. General investors complain only when the stocks' volatility gets out of hand, yet, do not object to the hype, or interferes against the baseless …

The biotechnology industry is proving to be the most innovative and the most growing among Wall Street industries. Individual biotech companies’ achievements complement each other. Some are evolving the sequencing of genes and genetic analysis, while others are excelling in designing drugs based on the tremendous amount of facts they are getting on root causes of the diseases and the interaction of proteins. The biotech …

but first: Here is some good news from  SAREPTA Yesterday we wrote about BioMarin (BMRN) being granted FDA “Rare Pediatric Disease Designation for its Duchenne potential muscular dystrophy (DMD) drug drisapersen.Today, Sarepta Therapeutics (SRPT) also a developer of RNA-targeted therapeutics has been granted the same for its drug eteplirsen, also a potential treatment for DMD patients who are amenable to skipping exon 51. The Rare …

Prohost Letter #378 Part 1 Views and Visions TOP-TIER BIOTECHNOLOGY COMPANIES GILEAD We wrote a lot about Gilead (GILD), doing our best to protect investors against negative gossip that filled the media and aimed at misleading and insulting investors’ intelligence. We never lowered our target based on the perpetrated, fabricated stories, which made many investors lose confidence in Gilead’s strategy and sell their stocks, thus, …