Beyond Achondroplasia

Growing together with Clara

A challenging report on Vosoritide


A rare disease encompasses any life-threatening or chronically debilitating disorder or condition which, as the name suggests, is uncommon in the general population. Rare diseases typically exhibit a high level of symptom complexity and as a result they very frequently require special combined treatments. 95% of rare diseases have no available treatment. 1 Having this designation “disease” on it, does not mean exactly to be ill.

Having no treatment available for achondroplasia, many young patients and families are waiting for a quicker progression of BioMarin´s clinical trial on Vosoritide, with high expectations and hope on the efficacy of this new drug.

Nevertheless, is important to keep our feet on the ground.

Another vision of the development of this orphan drug will be presented: The market vision.

Pharmaceutical companies are businesses, usually listed on the stock exchange market. These companies work to develop new drugs for patients and to make money, as any company. But it order to obtain profit, pharma companies have to develop the best products possible. These companies spend several years and millions of euros in the research and developing (R&D) of a new drug.

“BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare disorders and the company’s portfolio consists of five commercialized products and multiple clinical and pre-clinical product candidates”. BioMarin press release

Now, more than ever, BioMarin is taking cautious steps in the progression of the study, in order to get the drug approval by the regulators, FDA and EMA, for market access and then for patient access. And because BioMarin is a company in the financial market, it depends very much on investors, that have extra attention to all the details, to all press releases on data of the clinical trial, all advances and setbacks.

Recently, EP Vantage, an agency that does coverage of the pharmaceutical, biotech and medtech industries, giving fresh angles and insight to both current and future industry triggers, published an article after BioMarin’s last press release, on the 19th October 2016.

In the report (highlights presented next) published by EP Vantage, these financial consultants see the lack of increase in effect with the 30µg/kg dose of vosoritide as a flaw.

Biomarin Comes Up Short With Vosoritide

  1. Pivotal path of phase II data for vosoritide was clarified
  2. The fact that doubling the dose on vosoritide has fallen flat should cause investors to pause for thought.
  3. One year vosoritide emerged as an important Biomarin pipeline asset, with initial phase II results with 15µg/kg per day beating expectations, and setting a high bar for future studies of a 30µg/kg dose 
  4. But then, there was a Double dose setback. The 30µg/kg dose did result in a statistically significant increase in mean growth velocity versus baseline – 46% after six months – but this was numerically no better than dosing at half this level. Two six-month 15µg/kg doses gave 65% and 50% increases, and one of these taken out to 12 months increased growth by 46%.
  5. With the higher dose results flaw, BioMarin presented saying the long-awaited phase III trial, due to start before the end of this year, would use 15µg/kg dosing, over one year and with a placebo group.
  6. A leader investment bank in healthcare said it was unclear what additional information regulators might want (regarding EMA and FDA consultancy for a drug with a orphan designation- drugs for rare diseases are called this way).
  7. An analysts from other pharma company accepted that the theory about children catching up growth was dead in the water

BioMarin is taking extra precautions in preparing for phase III and selecting the primary endpoint (The endpoint of a clinical trial is a pre-defined event: for instance, the occurrence of a disease, the occurrence of a symptom, or a particular laboratory result. Once someone reaches the endpoint, they are generally excluded from further research in the trial.- in EUPATI).

BioMarin is working to deliver a good product to patients, but many doubts are still ahead. Phase III might be very revealing about the outcomes.


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